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ES Catheter vs Cryoablation After Pectus Surgery

Participants will be randomized to receive ES catheter or cryoablation for pain management after the Nuss procedure. The goal of this study is to compare the following between the two groups: * Time to achieve short-term physical therapy goals and long-term functional outcomes * Compare immediate and long-term postoperative opioid use * Compare numbness on chest of postoperative day 1 and the return of sensation to baseline * Compare the incidence of neuropathic pain and other complications Participants will receive surveys for up to 12 months postoperatively.

Kristie Geisler - kristie.geisler@cchmc.org

ALL
12 years to 21 years old
NA
This study is NOT accepting healthy volunteers
NCT06682208
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Inclusion Criteria:
* Age 12 - 21 years * History of pectus excavatum * Scheduled for Nuss procedure
Exclusion Criteria:
* Prior pectus repair * Other concomitant surgeries * Chronic pain conditions including Ehlers Danlos Syndrome * Non-English speaking - rationale: the questionnaires used in the study are in English language and the social and cultural factors of pain perception of non-English speaking patients could affect the study power * Severe developmental delays including cognitive (difficulties understanding), motor (cerebral palsy or muscular dystrophy), and speech delays (difficulties communicating) * History of or active renal or liver disease * Major surgery requiring opioids in the last 2 years * Severe respiratory problems (such as obstructive sleep apnea, cystic fibrosis, pulmonary fibrosis, or pneumonia within the last month) * Cardiac conditions including, but not limited to, cyanotic heart disease, hypoplastic left ventricle, arrhythmia, uncontrolled hypertension with ongoing treatment, Kawasaki disease, or cardiomyopathies. Participants with asymptomatic valvular lesions or defects may be included * BMI \>35 * Pregnant or breastfeeding females
PROCEDURE: ES catheter, PROCEDURE: Intercostal nerve cryoablation (INC)
Pectus Excavatum
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Natural History of Cerebral Palsy Prospective Study

The study will describe the natural history of cerebral palsy (CP) as children develop over the first 2 years of life.

Karen L Harpster, PhD, OTR/L - karen.harpster@cchmc.org

ALL
3 months to 24 months old
NCT05576948
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Inclusion Criteria:
* Infants will be included if they have a diagnosis of CP or "at high risk of CP" and under the age of 12 months. * Infants at high risk of CP as defined by any two (any combination) of the following:
• Neuroimaging predictive of a motor disability including the involvement of 1 or more of the following structures: sensori-motor cortex, Basal Ganglia, Posterior Limb of the Internal Capsule, pyramids.
• Trajectory of cramped synchronized general movements (GMs) on General Movement Assessment (GMA) OR absent fidgety GMs at fidgety age
• Hammersmith Infant Neurological Exam (HINE) scores \<57 @ 3months or \<60 @ 6months or \<63 @9 months Infants will also be included if they meet BOTH of the following criteria (even if infants have a HINE score greater than the cut off scores stated above: i. Unilateral brain injury on neuroimaging (magnetic resonance imaging (MRI) or cranial ultrasound (CUS)) predictive of CP ii. Clinical signs of asymmetry
Exclusion Criteria:
* Infants with progressive or neurodegenerative disorders or genetic disorders not associated with CP * Infants with other disability diagnoses e.g. Down Syndrome
Cerebral Palsy
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OMEGA: Outcome Measures in Eosinophilic Gastrointestinal Disorders Across the Ages (OMEGA)

The purpose of this observational study is to find the best measures to define how well a person with eosinophilic disorder is doing. People with EoE, EoG, EoN and EoC normally undergo endoscopy and/or colonoscopy where cells are collected for microscopic analysis. Treatments are then decided based on how the cells look. We are aiming to compare different tissue components such as inflammatory cell types with clinical symptoms. We want to see if scores on standard questionnaires can give us an idea how well the person is doing.

Bliss Magella - bliss.magella@cchmc.org

ALL
3 years and over
This study is NOT accepting healthy volunteers
NCT02523118
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Inclusion Criteria:
* Males or females 3 years of age and older * Mucosal eosinophilia: EoE ≥ 15 eosinophils/HPF in the distal or proximal esophagus EoG ≥ 30 eosinophils/HPF in 5 HPF\'s in the body and/or antrum EoN ≥ 53 eosinophils/HPF in the duodenum and/or ≥ 56 eosinophils/HPF in the jejunum and/or ileum EoC ≥ 84 eosinophils/HPF from the transverse or descending colon and/or ≥ 32 eosinophils/HPF from the rectosigmoid colon or a biopsy from any colonic location with ≥ 100 eosinophils/HPF \- Presence of symptoms is required for patients who are newly diagnosed but not required for patients who were previously diagnosed.
Exclusion Criteria:
* History of intestinal surgery other than G tube placement * Enrolled in a blinded investigational study at the time of the first study visit * Have esophageal stricture (\<3mm) * Have other identifiable causes for eosinophilia (except Inflammatory Bowel Disease): infections, Gastrointestinal (GI) cancer, other GI inflammatory disease (e.g., Ulcerative Colitis or Crohn\'s Disease)
Eosinophilic Gastrointestinal Disorders (EGIDs), Eosinophilic Esophagitis, Eosinophilic Gastritis, Eosinophilic Gastroenteritis, Eosinophilic Colitis
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Biopsychosocial Risk Influences on the Development and Generation of Early-Onset Chronic Pain (BRIDGE Pain)

The purpose of the study is to discover the underlying subtypes of chronic musculoskeletal pain (muscle and joint pain) in youth. Specifically, the researchers are looking for different profiles of brain and immune system signals that affect how the body feels pain, and how these pain profiles are shaped by a person's medical history, psychology, and social environment. The goal is to develop more precise treatments for chronic pain in young people. Primary Aim: The investigators want to identify subtypes of childhood-onset chronic pain (pain lasting more than 3 months) that affects the musculoskeletal system. To do this, the study will enroll teens and young adults (ages 14-26 years) who were diagnosed with chronic pain in childhood (before the age of 18) or have a pediatric rheumatic disease or major surgery that places them at risk for developing chronic pain. The researchers will look at participants' childhood medical histories and treatments, their current pain and other symptoms, sensory tests of pain and immune system markers found in their blood. The investigators will use this information to find different clinical and neuroimmune profiles of chronic pain. They will also be able to identify the profiles of those who are at risk for chronic pain (because of a rheumatic disease or past surgery) but never develop long term pain. Secondary Aim: The study will look at how medical history, treatments, and different lifestyle factors (like sleep and physical activity), and psychosocial factors (mood, coping, social support) influence the pain experience in subgroups of chronic pain identified in the primary aim. By understanding specific behavioral and psychosocial risk and protective factors-the researchers hope to better understand why some young people develop long-lasting pain and others do not and also how better treatments can be developed for those who do have chronic pain.

Megan Kirschman - bridgestudy@cchmc.org

ALL
14 years to 26 years old
This study is NOT accepting healthy volunteers
NCT07602595
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Inclusion Criteria:
* Diagnosis of rheumatic/autoimmune disease/pain condition or initial MSK surgery prior to age 18 * Current age 14 to 26 years * Participant (and parent/legal guardian of participants \< 18 yo) can read and write in English * At least 1 year from diagnosis of pain or rheumatic disease * For individuals with MSK surgical history, at least 1 year after initial surgical intervention * For individuals with rheumatic disease, their disease must be considered inactive
Exclusion Criteria:
* They have active disease (JIA or cSLE) * Other medical conditions (e.g., malignancy, acute injury, surgery, infection) that impact current health status at the time of enrollment * Currently pregnant or breastfeeding
OTHER: Not applicable- observational study
Juvenile Idiopathic Arthritis (JIA), Fibromyalgia, Lupus, Scoliosis, Pectus Excavatum, Chronic Pain, Musculoskeletal Pain
Pediatric Pain, Musculoskeletal Pain
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Study of MRM-3379 in Male Participants With Fragile X Syndrome (BLOOM)

This study is a multicenter, double-blind, randomized, placebo-controlled study to assess the safety and tolerability of 3 doses of MRM-3379 in male participants with Fragile X Syndrome ages 16 to 45 (inclusive). In addition, a parallel cohort of participants ages 13 to \<16 will receive open-label MRM-3379. All participants will participate for 12 weeks of treatment. The study is also intended as a proof-of-concept investigation to evaluate whether MRM-3379 can improve FXS symptoms

Hannah Sachs - Hannah.Sachs@cchmc.org

MALE
13 years to 45 years old
PHASE2
This study is NOT accepting healthy volunteers
NCT07209462
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Inclusion Criteria:
* Willing and able to provide signed informed consent/assent. Where local regulations permit inclusion of participants deemed unable to provide informed consent, a legally authorized representative must provide informed consent on the participant's behalf, and the participant must provide assent if applicable. * Male, 13-45 years of age (inclusive) * Weight ≥30 kg and BMI between 18 and 36 kg/m2 (inclusive) at the screening visit * Diagnosis of FXS with a molecular genetic ≥200 CGG repetitions . * Able to perform the PVT and ORRT of the NIH-TCB * Have a consistent caregiver(s) who is willing and able to be present regularly and reliably with the participant * Able to swallow tablets or capsules
Exclusion Criteria:
History of or current medical condition other than FXS and related issues that would place the participant at higher risk from study participation
DRUG: Low dose of MRM-3379, DRUG: Middle Dose of MRM-3379, DRUG: High dose of MRM-3379, DRUG: Placebo, DRUG: Low dose of MRM-3379 Open-Label
Fragile X Syndrome
Fragile X Syndrome, FXS, FMR, Phosphodiesterase 4, PDE4
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Global Patient Registry to Monitor Long-term Safety and Effectiveness of Increlex® in Children and Adolescents With Severe Primary Insulin-like Growth Factor-1 Deficiency (SPIGFD).

The Increlex® Global Registry is a descriptive, multicenter, observational, prospective, open-ended, non interventional, post-authorisation surveillance registry. The main purpose of this global registry is to collect, analyse and report safety data during and up to at least 5 years after the end of treatment in children and adolescents receiving Increlex® therapy for SPIGFD according to the locally approved product information.

Leah Tyzinski - Leah.Tyzinski@cchmc.org

ALL
2 years to 18 years old
This study is NOT accepting healthy volunteers
NCT00903110
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Inclusion Criteria:
* For US : patients starting or planning to start or currently receiving treatment with Increlex® therapy for severe primary IGF-1 deficiency as defined by the US Increlex® prescribing information or for growth hormone (GH) gene deletion who have developed neutralizing antibodies to GH.For EU : patients starting or planning to start or currently receiving treatment with Increlex® therapy according to the locally approved product information. * Parents or legally authorized representatives if applicable must give signed informed consent before any registry-related activities are conducted. Assent from the subject should also be obtained as appropriate
Exclusion Criteria:
* Subject currently participating in an Increlex® clinical trial * Subject currently participating in any clinical trial for growth retardation * Patient with any contraindication to Increlex® or any condition subject to special warning as per the locally approved label * For US patients, these include patients with hypersensitivity to the active substance or any of the excipients, patients with active or suspected neoplasia and patients with closed epiphyses. * For EU patients: these include patients with hypersensitivity to the active substance or any of the excipients, patients with active or suspected neoplasia or any condition or medical history which increases the risk of benign or malignant neoplasia and patients with closed epiphyses
DRUG: Increlex®
IGF1 Deficiency
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Prevention of Graft Rejection in Hematopoietic Stem Cell Transplant (HSCT) Recipients

The investigators hypothesize that graft rejection after hematopoietic stem cell transplant (HSCT) is primarily driven by interferon gamma, and prophylactic interferon gamma inhibition in high-risk patients will prevent graft rejection. Additionally, knowledge of emapalumab PK/PD and in vitro mechanistic effects of emapalumab in this novel setting will guide optimization of dosing regimens and treatment approaches in future studies.

Jessica Anderson - jessica.anderson@cchmc.org

ALL
EARLY_PHASE1
This study is NOT accepting healthy volunteers
NCT07244419
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Inclusion Criteria:
* All patients undergoing allogeneic HSCT at our institution will be evaluated for graft rejection risk factors. Patients deemed high risk for graft rejection will have 2 or more of the following: mismatched or haploidentical donor, ex vivo t-cell depleted graft, prior history of graft rejection.
Exclusion Criteria:
* Known hypersensitivity to any constituent of the study medication.
DRUG: Emapalumab 3 mg/kg, DRUG: Emapalumab 10 mg/kg
Hematopoietic Stem Cell Transplantation, Graft Failure
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Zoledronate to Prevent Bone Health Complications in Pediatric Hematopoietic Stem Cell Transplant Survivors

The purpose of this pilot study is to investigate the safety and preliminarily assess efficacy of early intervention with zoledronate in high risk pediatric hematopoietic stem cell transplantation (HSCT) patients to prevent the development of bone disease and fractures and reduce potential pain and suffering.

Jessica Anderson - jessica.anderson@cchmc.org

ALL
5 years to 18 years old
PHASE1
This study is NOT accepting healthy volunteers
NCT07375290
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Inclusion Criteria:
* Patients ≥5 and ≤18 years old who are preparing for HSCT with a height-for-age corrected DXA Z-score of \<-2.0 and admitted to a CCHMC inpatient unit. * Patients ≥5 and ≤18 years old recovering from HSCT and who have developed de novo acute or chronic GVHD and are admitted to a CCHMC inpatient unit.
Exclusion Criteria:
* Age \<5 years and \>18 years * Patients with Fanconi anemia or other radiation-sensitive syndromes with increased malignancy risk * history of prior bisphosphonate use * low 25-OH vitamin D levels (\<20 ng/mL) * active febrile illness * uncontrolled infection * Elevated creatinine at the time of enrollment, history or renal failure, or documented low glomerular filtration rate (GFR≤90) * Active bone disease including history of abnormal PTH level for any reason, active bone fracture/healing, or primary disorder of bone development or metabolism. * Women who are pregnant or breast feeding.
DRUG: Zoledronate
Hematopoietic Stem Cell Transplantation
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A Phase 2 Study of Mutant-selective PI3Kα Inhibitor, RLY-2608, in Adults and Children With PIK3CA Related Overgrowth Spectrum and Malformations Driven by PIK3CA Mutation (The ReInspire Study)

This is a 3-part Phase 2 randomized study evaluating the safety and efficacy of the mutant-selective PI3Kα inhibitor, zovegalisib (RLY-2608), in adults and children with PIK3CA Related Overgrowth Spectrum (PROS) and malformations driven by PIK3CA mutation. Part 1 is a dose selection, Part 2 is a basket design with exploratory single-arm cohorts for various subpopulations of participants, and Part 3 is randomized, double-blinded study vs placebo.

Sarah Price - Sarah.price@cchmc.org

ALL
2 years and over
PHASE2
This study is NOT accepting healthy volunteers
NCT06789913
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Key
Inclusion Criteria:
* The participant must have a clinical diagnosis of PROS or a malformation within the ISSVA classification. * One or more documented activating PIK3CA mutation(s) that are targeted by selective PI3Kα inhibitors in lesional tissue and/or cell-free DNA from the lesion or blood. Some participants may be eligible without a documented PIK3CA mutation, with the sponsor's approval, as long as no other genetic driver has been documented. * Lansky (\<16 yo) or Karnofsky (≥16 yo) performance status of ≥50. * Agree to provide archived lesional fluid and/or tissue or be willing to undergo pretreatment lesional biopsy (if considered safe and medically feasible) to assess PIK3CA status. Key
Exclusion Criteria:
* Known hypersensitivity to RLY-2608. * Any factors that increase the risk of QTc prolongation or risk of arrhythmic events * Clinically significant, uncontrolled cardiovascular disease * Received disease-directed therapy prior to the first dose of study drug:
• Systemic therapy or antibody within 5 half-lives of the therapy.
• Local therapy including radiation, surgery, or other procedures within 28 days; lesion(s) must have demonstrated progression after the procedure.
DRUG: RLY-2608, DRUG: Placebo
PIK3CA-Related Overgrowth Spectrum (PROS), Lymphatic Malformations, Vascular Malformations, PIK3CA Mutation, CLOVES Syndrome, Klippel Trenaunay Syndrome, Megalencephaly-capillary Malformation Polymicrogyria Syndrome (MCAP), Vascular Anomalies
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Target Trial Emulation for Pharmacologic Treatment of Neonatal Opioid Withdrawal Syndrome (TreaT NOW)

The goal of this observational study is to learn how two medicines used in routine care-buprenorphine and morphine-affect recovery in newborns (≥36 weeks' gestation) with Neonatal Opioid Withdrawal Syndrome (NOWS). The main questions it aims to answer are: 1. Do infants treated with buprenorphine become medically ready for discharge sooner than those treated with morphine? 2. Does one treatment lead to better overall clinical outcomes than the other? Researchers will compare infants who received buprenorphine with infants who received morphine to see whether one treatment helps babies recover more quickly. Participants will not be asked to do anything. Instead, the study team will collect information already documented in the infant's and mother's medical records securely without any contact or changes to clinical care. No new medicines, procedures, or visits are involved. This study only reviews existing clinical data to better understand which commonly used treatment may support faster recovery for newborns with NOWS.

Traci Beiersdorfer - Traci.Beiersdorfer@cchmc.org

ALL
Not specified
This study is NOT accepting healthy volunteers
NCT07278375
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Inclusion Criteria:

• Infant is ≥ 36 weeks' gestational age
• Infant had antenatal opioid exposure identified by at least one of the following:
• History of maternal opioid use during the second and/or third trimester of pregnancy as noted in the mother's or infant's medical record;
• Positive maternal toxicology screen for opioids during the second or third trimester of pregnancy; and/or
• Positive infant toxicology screen for opioids during the initial hospital stay.
• The infant is being assessed and managed for NOWS at an eligible study site.
• The infant is at risk for pharmacologic treatment for NOWS defined by either of the following: * At least 1 score ≥ 8 if assessed and managed with the Finnegan Neonatal Abstinence Scoring Tool (FNAST) or modification thereof * At least 1 "yes" if assessed and managed with the Eat, Sleep, Console (ESC) care approach
• Infant met all inclusion criteria on or after March 25, 2024.
Exclusion Criteria:

• Infant has major congenital anomalies.
• Infant has neonatal encephalopathy (inclusive of hypoxic ischemic encephalopathy), a metabolic disorder, stroke, intracranial hemorrhage, or meningitis diagnosed prior to the initiation of pharmacologic treatment.
• Infant is receiving respiratory support (any positive pressure or oxygen therapy) at 48 hours of age.
• Infant has undergone major surgical intervention prior to or at 48 hours of age.
• Infant has postnatal opioid exposure prior to the initiation of pharmacologic treatment for NOWS.
• Infant was outborn and pharmacologic treatment was initiated at the transferring hospital.
Neonatal Opioid Withdrawal Syndrome
Neonatal Opioid Withdrawal Syndrome, NOWS
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Zinc Supplementation in Sickle Cell Disease: A Precursor to the Think Zinc for Bones Trial (ZnSCD)

The goal of this short term prospective Phase II study is to compare the effects of two alternate daily doses of zinc (25 and 40 mg/day) in 34 randomly assigned homozygous Sickle Cell Disease (SCD-SS) patients aged 15-40 years old. The main question it aims to answer is: Which biomarkers are most responsive to zinc supplementation, and what is the maximum tolerated zinc dose that induces the desired changes in biomarkers of bone turnover? Participants will be recruited from 6 American Society Hematology Research Collaborative SCD Centers. Eligible SCD subjects will be invited to participate in the 16-week study, involving 2 baseline blood draws 4 weeks apart, followed by a 12-week zinc intervention. The findings from this study will be used to determine the dosage of zinc to be used in a larger, future study on the long term impact of zinc supplementation on bone health in SCD-SS.

Amy Shova - Amy.Shova@cchmc.org

ALL
15 years to 40 years old
PHASE2
This study is NOT accepting healthy volunteers
NCT06260891
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Inclusion Criteria:
* Age: ≥ 15.0 to ≤ 40.0 years * Diagnosis: SCD-SS or SCD-S Beta zero Thalassemia, in steady state (defined as a minimum of 2 weeks days following pain crisis) * Male or Female
Exclusion Criteria:
* Taking any supplement containing zinc and unable/willing to stop for 3 months prior to study start * 25-Hydroxy Vitamin D \< 20 ng/mL * On chronic transfusion therapy (defined as \>8 Transfusions/year) and iron overloaded (defined as liver iron concentration \> 7 mg/g OR average serum ferritin \>4000 ug/L) * Unable swallow pills or take daily supplement as instructed * Currently participating in another investigational drug trial * Prior diagnosis of chronic kidney disease (eGFR \< 30 mL/min/1.73m2)
DRUG: 25 mg/day zinc, DRUG: 40 mg/day zinc
Sickle Cell Disease
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A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections

The purpose of this study is to demonstrate the efficacy and evaluate the safety and tolerability of mavorixafor in participants with congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorders who are experiencing recurrent and/or serious infections as assessed by demonstrating its clinical benefit and increasing levels of circulating neutrophils.

Joan Moore - Joan.Moore@cchmc.org

ALL
12 years and over
PHASE3
This study is NOT accepting healthy volunteers
NCT06056297
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Inclusion Criteria:
* Diagnosis of congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorder ≥6 months prior to the screening visit that is not attributable to medications, active or recent infections or malignancy. * Congenital Neutropenia, including but not limited to these classifications:
• Isolated with a permanent (non-cyclic) presentation, for example, elastase, neutrophil expressed (ELANE), colony stimulating factor 3 receptor (CSF3R), C-X-C chemokine receptor 2 (CXCR2), Wiskott-Aldrich syndrome (WAS)
• Associated with extra-hematologic manifestations, for example, Barth syndrome, Cohen syndrome, glucose-6-phosphatase catalytic subunit 3 (G6PC3), Kostmann disease
• Associated with metabolic disorders, for example, glycogen storage disease 1b (GSD1b)
• Shwachman-Diamond syndrome * Acquired Primary Neutropenia
• Chronic idiopathic neutropenia
• Primary autoimmune neutropenia. Other chronic neutropenia (CN) disorders that may be eligible for enrollment can be clarified and approved upon discussion with study Medical Monitor. * Have an ANC \<1000 cells/µL during screening (single ANC value from hematology) and confirmed trough mean ANC (mean value of multiple ANC measurements over 6 hours) at baseline visit, with no clinical evidence of systemic infection. * Prior history of recurrent and/or serious infections during the 12 months preceding the screening visit (that is, suffering sequelae of chronic neutropenia), as defined by having at least 2 infections in the last 12 months that meet the following criteria: * Infection requiring the use of antibiotics (intravenous \[IV\]/oral); OR * Infection requiring a visit to healthcare facility (including but not limited to emergency room visit, urgent care facility, primary care physician's office, or in-patient hospitalization); AND for all potential participants: * Infections considered by the Investigator to be likely related to the potential participant's CN disorder. * Participants who are on G-CSF or other active background therapy must have been receiving these therapies during the previous 12 months while continuing to suffer from infections, be on a stable dose and dosing schedule for ≥4 weeks prior to screening visit and remain on this dose and dosing schedule throughout the study (unless ANC \>10,000 cells/µL for ≥4 weeks). * Participants must be willing to keep their G-CSF or other background therapy doses/regimens stable (other than for safety reasons) for the duration of the study. Key
Exclusion Criteria:
* A diagnosis of secondary neutropenia including those due to:
• Hypersplenism
• Infection
• Malignancy
• Autoimmune disease, for example, systemic lupus erythematosus, rheumatoid arthritis, inflammatory bowel disease, graft-versus-host disease, thyroid disease
• Nutritional deficiency, for example, vitamin B12, folic acid, copper, caloric malnutrition
• Drug-induced cause, for example, chemotherapy, clozapine, antiretrovirals, antibiotics, monoclonal antibodies. * A diagnosis of any of the following:
• Aplastic anemia
• Warts, hypogammaglobulinemia, infections, and myelokathexis (WHIM) syndrome
• Certain CNs, including but not limited to these classifications are excluded:
• Isolated with a cyclic presentation, for example, elastase, neutrophil expressed (ELANE)
• Associated with immune dysregulation, for example, common variable immunodeficiency (CVID), autoimmune lymphoproliferative syndrome (ALPS), familial hemophagocytic lymphohistiocytosis, Chédiak-Higashi syndrome, GATA-binding protein 2 (GATA2) deficiency syndrome
• Associated with bone marrow failure, for example, Fanconi anemia, Diamond-Blackfan anemia
• Neutropenia associated with a Duffy-null phenotype (formerly known as benign ethnic neutropenia). However, a participant with an autosomal dominant pathogenic variant in a gene associated with CN on a Duffy-null background may be eligible for inclusion * A medical or personal condition that may potentially compromise the safety of the participant, may preclude the participant's successful completion of the clinical study, or could, in the opinion of the Investigator or the Medical Monitor, interfere with the objectives of the study. * Received more than 1 dose of mavorixafor in the past. * Received C-X-C chemokine receptor 4 (CXCR4) antagonist (other than mavorixafor) in the past 6 months. * Participants taking pegylated-G-CSF unless they have a diagnosis of congenital neutropenia confirmed at screening. * Participant is currently taking or has taken other investigational drug \<30 days prior to the screening visit or 5 half-lives, whichever is longer. Note: Other protocol-defined inclusion and exclusion criteria may apply.
DRUG: Mavorixafor, DRUG: Placebo
Neutropenia
Mavorixafor, Chronic neutropenia, Chronic idiopathic neutropenia, Severe Congenital Neutropenia (SCN), C-X-C chemokine receptor 4 (CXCR4), Congenital and acquired neutropenia, Autoimmune disease, Cohen syndrome, Barth syndrome, ELANE, G6PC3, GSD1b, Kostmann disease, Shwachman-Diamond syndrome, Autoimmune neutropenia (AIN)
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ACTEMRA® for the Treatment of Pediatric Adamantinomatous Craniopharyngioma

ACTEMRA (tocilizumab) is an IL-6 receptor antagonist used for the treatment of adult Rheumatoid Arthritis as well as Polyarticular (PJIA) and Systemic (SJIA) Juvenile Idiopathic Arthritis. In this Phase II, the drug will be used to treat pediatric patients diagnosed with recurrent Adamantinomatous Craniopharyngioma including patients who have undergone surgery and/or radiation therapy.

- cancer@cchmc.org

ALL
1 year to 39 years old
PHASE2
This study is NOT accepting healthy volunteers
NCT05233397
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Inclusion Criteria:

• Age: Patients must be ≥ 12 months and ≤ 39 years of age at the time of study enrollment.
• Diagnosis: Patients with histologically-confirmed adamantinomatous craniopharyngioma (ACP) Histologic confirmation of ACP may be made on solid tumor or, if no solid tumor can be safely obtained, cyst fluid with classic ACP characteristics of thick, cholesterol-rich, greenish-brown liquid in the context of imaging features consistent with craniopharyngioma, including lobulated, cystic/solid mass with calcifications that originates in the sellar/suprasellar region.
• Disease Status: Patients must have measurable disease. * Stratum 1: Patients with progressive or recurrent ACP who demonstrate cystic and/or solid recurrence or progression at least 6 months post completion of radiation therapy * Stratum 2 (CLOSED): Patients with measurable ACP who have undergone surgery but have NOT previously undergone irradiation (but may have received prior systemic or intracystic therapy). Progressive disease is allowed but not required.
• Performance Level: Karnofsky ≥ 50% for patients \> 16 years of age and Lansky ≥ 50 for patients ≤ 16 years of age (See Appendix I). Note: Neurologic deficits in patients with CNS tumors must have been stable for at least 7 days prior to study enrollment. Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.
• Prior Therapy: Patients must have recovered or stabilized from the acute toxic effects of prior treatments * Biologic (anti-neoplastic agent): At least 7 days must have elapsed after the last (systemic or intracystic) dose of a biologic agent. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur. The duration of this interval must be discussed with the study chair * Immunotherapy: At least 42 days after the completion of any type of systemic immunotherapy, e.g. tumor vaccines. * Monoclonal antibodies: At least 21 days after the last dose of a monoclonal antibody. * Radiation therapy: Patients must have had their last (conventional or hypofractionated) fraction of: a) Focal irradiation \> 6 months prior to enrollment and b) No prior craniospinal irradiation is permitted. * Corticosteroids: Patients receiving dexamethasone must be on a stable or decreasing dose for at least 1 week prior to enrollment * Myelosuppressive systemic therapy: At least 21 days must have elapsed after the last systemic myelosuppressive therapy. * Surgery: At least 6 weeks must have elapsed since major or intermediate surgery. Major surgery includes major craniotomy for tumor resection or cyst fenestration, organ resection, exploratory laparotomy. Intermediate procedures include ventriculoperitoneal shunt placement, stereotactic brain biopsy and intraventricular catheter placement. Minor procedures that are not excluded include skin biopsy/incision and drainage, bone marrow aspirate, and central venous catheter placement. Ommaya aspirations and Lumbar Punctures are considered minor procedures..
• Organ Function Requirements Adequate Bone Marrow Function Defined as: * Peripheral absolute neutrophil count (ANC) ≥1000/mm3 * Platelet count ≥100,000/mm3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment) * Hemoglobin \>8 g/dL (may be transfused) Adequate Renal Function Defined as: * Creatinine clearance or radioisotope GFR \> 70ml/min/1.73 m2 or * A serum creatinine based on (Schwartz et al. J. Peds, 106:522, 1985) age/gender as follows: 1 to \< 2 years: maximum serum creatinine 0.6 mg/dL for males and females. 2 to \< 6 years: maximum serum creatinine 0.8 mg/dL for males and females. 6 to \< 10 years: maximum serum creatinine 1.0 mg/dL for males and females. 10 to \< 13 years: maximum serum creatinine 1.2 mg/dL for males and females. 13 to \< 16 years: maximum serum creatinine 1.5 mg/dL for males and 1.4 mg/dL for females. ≥ 16 years: maximum serum creatinine 1.7 mg/dL for males and 1.4 mg/dL for females. Adequate Liver Function Defined as: * Total bilirubin within normal institutional limits * AST (SGOT) ≤ 2.5 × institutional upper limit of normal * ALT (SGPT) ≤ 2.5 × institutional upper limit of normal Adequate Neurologic Function Defined as: * Patients with neurological deficits should have deficits that are stable for a minimum of 1 week prior to enrollment. * Patients with current seizure disorders may be enrolled if seizures are well-controlled on antiepileptic therapies.
• Informed Consent: All patients and/or their parents or legally authorized representatives must sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines.
Exclusion Criteria:

• Pregnancy or Breast-Feeding: Pregnant or breast-feeding women will not be entered on this study due to unknown risks of fetal and teratogenic adverse events as seen in animal/human studies. Pregnancy tests must be obtained in girls who are post-menarchal. Males or females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method for at least 90 days after discontinuation of drug for females and at least 60 days for males. For females of childbearing potential, agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive methods (bilateral tubal ligation, male sterilization, hormonal contraceptives that inhibit ovulation, hormone-releasing intrauterine devices, and copper intrauterine devices; hormonal contraceptive methods must be supplemented by a barrier method) and agreement to refrain from donating eggs are required. For males of reproductive potential, agreement to remain abstinent (refrain from heterosexual intercourse) or use a condom, and agreement to refrain from donating sperm.
• Gastrointestinal Disease: Patients with a history of serious gastrointestinal disease, including inflammatory bowel disease or gastrointestinal perforation
• Concomitant Medications * Corticosteroids: Patients receiving corticosteroids who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible. * Investigational Drugs: Patients who are currently receiving another investigational drug are not eligible. * Anti-cancer Agents: Patients who are currently receiving other anti-cancer agents are not eligible.
• Study Specific: * Patients who have an uncontrolled infection are not eligible. * Patients who have received any live or attenuated vaccinations within three months prior to start of therapy are not eligible. * Any significant concurrent medical or surgical condition that would jeopardize the patient's safety or ability to complete the study, including, but not limited to, disease of the nervous, renal, hepatic, cardiac (such as symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia), pulmonary, or endocrine system * Patients who have a history of Human Immunodeficiency Virus, Hepatitis B Virus, Hepatitis C Virus or Tuberculosis infection are not eligible. * Patients who have received a prior solid organ transplantation are not eligible. * Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible. * Patients who have a history of alcohol, drug, or chemical abuse within 6 months of screening. * Patients who have had major or intermediate surgery within the last 6 weeks or who have concerns for poor postsurgical wound healing. * Patients who have a history of allergic reactions attributed to compounds of similar chemical or biologic composition to tocilizumab and its excipients are not eligible.
DRUG: Tocilizumab
Adamantinomatous Craniopharyngioma, Recurrent Adamantinomatous Craniopharyngioma
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Continued Pressure for Alveolar Protection (CPAP Trial) (CPAP)

The objective of the CPAP Trial is to test whether extending CPAP until 34 weeks' PMA or for at least 2 additional weeks compared to weaning to a nasal canula will decrease the likelihood of bronchopulmonary dysplasia or death at 36 weeks' PMA.

Traci Beiersdorfer - traci.beiersdorfer@cchmc.org

ALL
Up to 7 month(s) old
NA
This study is NOT accepting healthy volunteers
NCT07417111
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Inclusion Criteria:
* Gestational age \<29 weeks at birth * PMA \<32 weeks at study entry * On treatment with CPAP without a rate in FiO2 \<0.25 and PEEP of 4-5 cmH2O * Meet stability criteria: * If previously intubated must be extubated ≥ 72 hours * \<3 self-resolving apneas (≤ 20 s) and/or bradycardia (\<100 bpm) in any hour over previous 6 hours * No episodes of apnea or bradycardia requiring intervention (oxygen/stimulation/bag and mask) for 24 hours * Parents/legal guardians consent for enrollment
Exclusion Criteria:
* Major malformation * Neuromuscular condition that affects respiration * Terminal illness * Decision to withhold or limit support * Too sick to participate in opinion of Attending physician * Clinical shock, sepsis * Planned surgery during study period
DEVICE: CPAP, DEVICE: Nasal Cannula
Bronchopulmonary Dysplasia (BPD)
Continuous Positive Airway Pressure, CPAP
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Evaluating Additive Effects of Including Canines in Regulating Together

The primary objective is to evaluate the potential additive effect of animal-assisted intervention (AAI) on a manualized behavioral treatment targeting emotion dysregulation (ED) in children with autism spectrum disorder (ASD). Aim 1: Evaluate whether Regulating Together-Canine demonstrates earlier and greater improvement in emotion dysregulation than Regulating Together-Standard. Aim 2: Evaluate if Regulating Together-Canine increases child engagement and learning compared to Regulating Together-Standard. Exploratory Aim: Explore association of physiological arousal (via heart rate tracking) with emotion dysregulation, treatment engagement, and learning.

Carrie Fassler - carrie.fassler@cchmc.org

ALL
8 years to 15 years old
NA
This study is NOT accepting healthy volunteers
NCT05803343
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Inclusion Criteria:
* Concern of emotion dysregulation (ED) as measured by a score of 6 or greater on the Emotion Dysregulation Inventory-Reactivity (EDI-R) * Diagnosis of autism spectrum disorder (ASD) * Diagnosis confirmed by an experienced ASD clinician and further supported by scoring in the range for ASD on the Autism Diagnostic Observation Schedule (ADOS-2) * A Full Scale Intelligence Quotient score of 65 or greater on the Weschler Abbreviated Scale Intelligence (WASI-II) * English is the primary language * Family willing to keep prescribed medication stable over the course of the study period
Exclusion Criteria:
* Participant has a phobia toward or is allergic to canines * Participant has a history of aggression toward animals * Participant has had any physical aggression toward other children outside the home in the past 2 weeks that resulted in injury * Presence of comorbid major neuropsychiatric illness warranting other treatment approaches as determined by the study clinician(s) including substance use disorders, psychotic disorders/schizophrenia, and bipolar disorder, among others * Presence of any major sensory impairment that would limit participating in the material including blindness or uncorrected hearing loss * A legal guardian is not available to provide informed consent
BEHAVIORAL: Regulating Together-Canine, BEHAVIORAL: Regulating Together-Standard
Autism Spectrum Disorder, Emotion Regulation
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Use of Hyperpolarized Xenon Gas for Lung Imaging in Children and Adults (HPXeMR)

The goal of this study is to evaluate the usefulness of hyperpolarized (HP) 129Xe (xenon) gas MRI for regional assessment of lung function in a normal population of children and adults and in adults and also in children with respiratory compromise due to a variety of diseases.

Carrie Stevens - Carrie.Stevens@cchmc.org

ALL
6 years and over
PHASE1
This study is also accepting healthy volunteers
NCT02272049
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Inclusion Criteria:
* Ages 6 and up * Participant must be able to hold breath for up to 16 seconds
Exclusion Criteria:
* History of heart defect * Pregnancy or positive pregnancy test * History of uncontrolled asthma defined for this study as requiring use of rescue inhaler ≥ 2 times in past month * Symptoms of respiratory infection (loose or productive cough or wheeze), chest tightness, or sinus infection within past week * Baseline oximetry at MRI visit of less than 95% on room air or less than 95% on a previously prescribed dosage of oxygen delivered by nasal cannula * Participant is claustrophobic and unable to tolerate the imaging. * Standard MRI exclusions (metal, implants)
DRUG: Hyperpolarized 129 Xenon
Respiratory Disorders
Respiratory
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MASA Valve Early Feasibility Study (MVEFS)

The MASA Valve Early Feasibility Study (MVEFS) multi-site interventional clinical trial within the United States of America with each center following a common protocol.The objective of the trial is to evaluate the safety and probable benefit of MASA Valve in the indicated subset of patients requiring Right Ventricular Outflow Tract Reconstruction (RVOTR). As an early feasibility study, the purpose is determine the feasibility of success of the device in order to gather early data towards a future pivotal study and/or regulatory clearance submission.

Joshua Freytag - Joshua.Freytag@cchmc.org

ALL
0 years to 22 years old
NA
This study is NOT accepting healthy volunteers
NCT05452720
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Inclusion Criteria:

• At least one of the following: Right Ventricular to Pulmonary Artery mean gradient \> 35mm Hg, moderate or severe Pulmonary regurgitation (≥3+), or clinical indication for replacement of their native or prosthetic pulmonary valve with a prosthesis.
• Age \< 22 years
• Patient is geographically stable and willing to return for 1 year follow-up for the trial.
• Patient's legal guardian should be willing to provide informed consent (IC) at the hospital location where they are being enrolled.
• The patient, and the patient's parent / legal representative where appropriate, and the treating physician agree that the subject will return for all required post-procedure follow up visits and the subject will comply with clinical investigation plan required follow-up visits.
Exclusion Criteria:

• Patient is in need of or has presence of a prosthetic heart valve at any other position
• Patient has a need for concomitant surgical procedures (non-cardiac)
• Patients with previously implanted pacemaker (including defibrillators) or mechanical valves
• Patient has an active bacterial or viral infection or requiring current antibiotic therapy (if temporary illness, patient may be a candidate 4 weeks after discontinuation of antibiotics)
• Patient has an active endocarditis
• Leukopenia, according to local laboratory evaluation of white blood cell count
• Acute or chronic anemia, according to local laboratory evaluation of hemoglobin Patients can be transfused to meet eligibility criteria
• Thrombocytopenia, defined as Platelet count \< 150,000/mm3 Patients can be transfused to meet eligibility criteria
• Severe chest wall deformity, which would preclude placement of the PV conduit
• Known hypersensitivity to anticoagulants and antiplatelet drugs and to the device materials
• Immunocompromised patient defined as: autoimmune disease, patients receiving immunosuppressant drugs or immune stimulant drugs
• Patient has chronic inflammatory / autoimmune disease
• Need for emergency cardiac or vascular surgery or intervention
• Major or progressive non-cardiac disease (liver failure, renal failure, cancer) that has a life expectancy of less than one year
• Currently participating, or participated within the last 30 days, in an investigational drug or device study
• Alcohol or drug abuse as defined by DSM IV-TR criteria for substance abuse - this includes the illicit use of cannabis within the last 12 months
• Patient has medical, social or psychosocial factors that, in the opinion of the Investigator, could have impact on safety or compliance
DEVICE: Surgical Right Ventricular Outflow Tract Reconstruction
Tetrology of Fallot, Pulmonary Stenosis, Truncus Arteriosus, Transposition of Great Vessels, Pulmonary Atresia, Ross Procedure
Right Ventricular Outflow Tract Reconstruction, Pulmonary Valve, MASA Valve, Pulmonary Valve Replacement
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Donor-Derived Viral Specific T-cells (VSTs) (VSTs)

In this research study, the investigators want to learn more about the use of donor-derived viral specific T-cells (VSTs) to treat viral infections that occur after allogeneic stem cell transplant. A viral specific T cell is a T lymphocyte (a type of white blood cell) that kills cells that are infected (particularly with viruses). Allogeneic means the stem cells come from another person. These VSTs are cells specially designed to fight the virus infections that can happen after a bone marrow transplant. The investigators are asking people who have undergone or will undergo an allogeneic stem cell transplant to enroll in this research study, because viral infections are a common problem after allogeneic stem cell transplant and can cause significant complications including death. Stem cell transplant reduces a person's ability to fight infections. There is an increased risk of getting new viral infections or reactivation of viral infections that the patient has had in the past, such as cytomegalovirus (CMV), Epstein-Barr virus (EBV), adenovirus (ADV), BK virus (BKV), and JC virus. There are anti-viral medicines available to treat these infections, though not all patients will respond to the standard treatments. Moreover, treatment of viral infections is expensive and time consuming, with families often administering prolonged treatments with intravenous anti-viral medications, or patients requiring prolonged admissions to the hospital. The medicines can also have side effects like damage to the kidneys or reduction in the blood counts, so in this study the investigators are trying to find an easier way to treat these infections.

Jamie Wilhelm - Jamie.Wilhelm@cchmc.org

ALL
1 month(s) and over
PHASE1
This study is NOT accepting healthy volunteers
NCT02048332
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Inclusion Criteria:
* Recipient must be at least 21 days after stem cell infusion * Clinical status must allow tapering of steroids to 0.5mg/kg prednisone or other steroid equivalent * Recipient must have achieved engraftment with ANC ≥ 500
Exclusion Criteria:
* Active acute GVHD grades II-IV * Uncontrolled bacterial or fungal infection * Uncontrolled relapse of malignancy requiring treatment with chemotherapy * Infusion of ATG or alemtuzumab within 2 weeks of VST infusion
BIOLOGICAL: Viral specific VST Infusion
Allogeneic Stem Cell Transplant, Viral Infection, Viral Reactivation
Epstein-Barr Virus (EBV), Adenovirus (ADV), t-cells, donor, transplant, children, cytomegalovirus (CMV), BK virus (BKV)
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Biomarker and Renal Angina Validation to Assess Heart-Kidney Outcomes After Amino Acid Therapy (BRAVE-HEART)

The goal of the BRAVE-HEART study is to learn if an amino acid infusion can reduce the risk of developing acute kidney injury after cardiac surgery in children. The main questions it aims to answer are: 1. Does an amino acid infusion decrease the number of participants with acute kidney injury? 2. Does an amino acid infusion decrease the number of days that participants are on a ventilator after cardiac surgery? Researchers will compare amino acids to a placebo (a look-alike substance that contains no drug) to see if amino acids decrease the number of participants with acute kidney injury. Participants will receive an amino acid or placebo infusion for up to 72 hours starting during cardiac surgery and only while in the operating room or the intensive care unit.

Kelli Krallman, RN, BSN, MS - kelli.krallman@cchmc.org

ALL
Up to 18 years old
PHASE2
This study is NOT accepting healthy volunteers
NCT07212595
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Inclusion Criteria:
* Expected to be at high risk of developing acute kidney injury after cardiac surgery based on Age, The Society of Thoracic Surgeons-European Association for Cardio-Thoracic Surgery (STAT) score, and anticipated cardiopulmonary bypass time * Age less than or equal to 18 years * Weight greater than or equal to 5 kilograms
Exclusion Criteria:
* Preoperative extracorporeal organ support * History of chronic kidney disease * Known or suspected inborn errors of amino acid metabolism * Known hypersensitivity to amino acids * Aspartate Aminotransferase (AST) or Alanine Aminotransferase (ALT) \> 3 times the upper limit of normal for age/gender * Preterm infants less than 6 months of age who were born at less than 36 weeks gestational age * Anuria at the time of randomization * Expected use of total parental nutrition (TPN) within the first 72 hours post-operatively
DRUG: Amino Acid infusion, DRUG: Lactated ringers solution
Acute Kidney Injury, Mechanical Ventilation
Cardiac Bypass, Pediatric, Acute Kidney Injury, Amino Acids
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Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia

This 3-part, Phase 1/2 study is designed to characterize the safety, tolerability, and pharmacological activity (as assessed by biomarker measurements) and to determine the selected dose of mRNA-3927 in participants with genetically confirmed propionic acidemia (PA). After establishing a dose with an acceptable safety and pharmacodynamic (PD) response for participants ≥1 year of age in Part 1, participants will be enrolled in Part 2 (which will serve as the pivotal study) to allow for determination of the efficacy, safety, and PD of mRNA-3927. Part 3 will evaluate the safety, efficacy and PD response of mRNA-3927 in infants (\<1 year of age).

Laurie Bailey - laurie.bailey@cchmc.org

ALL
PHASE1
This study is NOT accepting healthy volunteers
NCT04159103
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Inclusion Criteria:
Participants ≥1 year of age are eligible to be included in the study only if all of the following criteria apply: * ≥ 8 years of age at the time of consent/assent if enrolled as 1 of the first 2 participants in Part 1. * ≥1 year of age at the time of consent/assent if enrolled after the first 2 participants in Part 1. * Confirmed diagnosis of PA based on diagnosis by molecular genetic testing via central laboratory (PCCA and/or PCCB mutations). * Part 2 only: At least one documented MDE in the 12-month period before consent. Participants \<1 Year of Age : * Identification by newborn screening shortly after birth or having suspected PA by presenting with a spectrum of metabolic symptoms, and having a sibling diagnosed with PA. Participant may enter the Screening Period while awaiting genetic testing results, provided that all other eligibility criteria are met but would not be enrolled until diagnosis of PA is confirmed. * For infants in the neonatal intensive care unit (NICU) only: ≥37 weeks gestational age at the time of birth without other conditions/comorbidities that in the opinion of the Investigator may interfere with the interpretation of study results. * Body weight ≥3 kilograms (kg) at Screening. * At least 1 documented PA-related event prior to Screening defined as the following criteria: * Clinical signs of metabolic deterioration consistent with PA (for example, vomiting, not feeding well/poor suck, heavy breathing, lethargy, absence of proper perfusion, abnormal movements including bicycling, abnormal tone, low body temperature, seizure\[s\]), OR * Meeting the criteria of MDE definition, OR * Evidence of laboratory abnormalities as evidenced by at least one of the following: * Metabolic acidosis with elevated anion gap. * Acute hyperammonemia. * Neutropenia or thrombocytopenia.
Exclusion Criteria:
Participants of all ages are excluded from the study if during Screening any of the following criteria apply: * Any individual with laboratory abnormalities considered to be clinically significant (for example, markedly out of range, associated with clinical symptoms) in the Investigator or Sponsor's opinion that could interfere with or limit the participation in the study. * Estimated glomerular filtration rate (eGFR) \<30 milliliters (mL)/minute/1.73 square meter (m\^2) for participants of all ages receiving chronic dialysis. * History of organ transplantation or planned organ transplantation during the period of study participation. * Corrected QT interval (QTc) \>480 milliseconds (ms) using Bazett's correction. * Grade 3 or 4 heart failure according to the Modified Ross Heart Failure Classification for Children or the New York Heart Association Classification. * Pregnant or breastfeeding. * Other clinically significant conditions that in the Investigator's opinion could interfere with the safety of the participant, the interpretation of study results, or limit the participation in the study.
BIOLOGICAL: mRNA-3927
Propionic Acidemia
mRNA-3927, Propionic Aciduria, Metabolism, Inborn Errors, Genetic Diseases, Inborn Amino Acid Metabolism, Inborn Errors, Acidosis, Acid-Base Imbalance, Metabolic Diseases, Organic Acidemias, Moderna, mRNA
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ADHD PreSMA Response Inhibition Therapy

ADHD children have abnormal inhibitory control, meaning they have trouble stopping themselves from doing something they should not do. This ability to control involves an area in the brain called the pre-supplementary motor area (pre-SMA). Scientists have previously shown that the pre-SMA is abnormal in ADHD patients. In this study, we will use Transcranial Magnetic Stimulation (TMS) to stimulate the pre-SMA and determine the effects on measures that are related to inhibitory control.

Karlee Migneault - karlee.migneault@cchmc.org

ALL
12 years to 17 years old
NA
This study is NOT accepting healthy volunteers
NCT06325813
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Inclusion Criteria:

• ADHD diagnosis
• Ages 12-17 years
• Stimulant use is allowed but must be discontinued 24 hours prior to and during days of TMS visit
Exclusion Criteria:

• Medical conditions contraindicated or associated with altered TMS risk profile, including history of intracranial pathology, epilepsy or seizure disorders, traumatic brain injury, brain tumor, stroke, intellectual disability, cerebral palsy, neurodegenerative conditions, hearing impairment, metallic objects in the head or any other serious medical condition
• Presence of any implanted medical devices (e.g., ports, shunts, stimulators, cochlear implants)
• For biological females who are post-menarche, current pregnancy based on urine pregnancy test.
• Baseline problem of hearing impairment or chronic tinnitus
• Any clinically significant finding on brain MRI
• History of DSM-5 conduct disorder, major depressive disorder, bipolar disorder, obsessive compulsive disorder, anxiety disorder, psychotic disorder
• Non-stimulant medication(s) for ADHD (e.g., alpha2 adrenergic agonist, atomoxetine, tricyclics)
• Neuroleptic/antipsychotic medication(s)
• Inability to undergo MRI
• Active suicidality, history of suicidality, or high risk for suicide as assessed by a study physician
• Substance abuse or dependence within the past year, based on 1) separate screening process of asking parent/guardian and participant, and 2) positive urine drug screen. Exception will be made for positive urine drug screen due to prescribed ADHD medication
DEVICE: Active repetitive TMS, DEVICE: Sham repetitive TMS
Attention Deficit Hyperactivity Disorder
ADHD, Transcranial Magnetic Stimulation
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Hydroxyurea Exposure Limiting Pregnancy and Follow-Up Lactation (HELPFUL)

The purpose of this research study is to document and understand the effects of hydroxyurea exposure for women with SCD and their babies, during both gestation and lactation.

Teresa Latham - Teresa.Latham@cchmc.org

ALL
This study is NOT accepting healthy volunteers
NCT04093986
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Inclusion Criteria:
* Medical records or data available from previous clinical care prior to June 20, 2019 of pregnant females with SCD, including women who miscarried, had a still birth, or completed labor at any gestational stage, with any hydroxyurea exposure during either pregnancy and/or while breastfeeding. * Medical records or data available from previous clinical care prior to June 20, 2019 about pregnancy and breastfeeding outcomes, both for babies with hydroxyurea exposure and other babies by these same women.
Exclusion Criteria:
* Unavailable medical records or lack of information about hydroxyurea exposure.
OTHER: Chart Review, OTHER: Survey
Sickle Cell Disease, Sickle Cell Anemia
Hydroxyurea
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Suicide Treatment Alternatives for Teens (START)

Quasi-Randomized trial to compare inpatient care versus outpatient crisis intervention clinic. This study plans to enroll up to 1,000 participants across 4 sites in a 5 years period.

Katelyn Armstrong - katelyn.armstrong@cchmc.org

ALL
12 years to 18 years old
NA
This study is also accepting healthy volunteers
NCT04089254
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Inclusion Criteria:

• Adolescents that are 12 through 17 years old (including 17 year olds who will turn 18 years old during the course of the study).
• Are brought to the Emergency Department (ED) due to suicidal thoughts or behaviors
• Require a higher level of care (OCIC or Inpatient) indicated by clinician determination and a CHRT-SR score of 15 to 52.
• The presence of a legal guardian
• Capable of giving signed informed consent/assent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
Exclusion Criteria:

• Adolescents with suicidal thoughts that place themselves at a serious imminent risk of suicide based on clinical judgment.
• Adolescents who require 24 hour/day supervision but no adult can provide 24 hour/day supervision outside of the hospital
• Adolescents without the ability to read and answer survey questions
• Adolescents that are non-English speaking due to the scales and surveys that are used for this study only being available in English.
BEHAVIORAL: Inpatient Psychiatry, BEHAVIORAL: Outpatient Crisis Intervention Clinic
Suicidal Ideation
Suicide, Suicidal, Adolescent
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Markers of Trajectory in Pediatric CRPS

Complex Regional Pain Syndrome (CRPS) is a severe and complex chronic pain condition in children. Many psychosocial factors impact its development and recovery. CRPS has a strong central component, which is reflected by structural and functional changes in the brain. However, the interaction between these cerebral changes and trajectory of recovery has been seldom investigated to date. Furthermore, interactions between cerebral changes and psychosocial factors, which might affect trajectory of recovery, are unknown. The aim of this study is to identify the psychosocial factors and cerebral changes that predict the trajectory of recovery from CRPS. Children between the ages of 10 and 17 years will be enrolled with one of their parents or legal guardians for this study. Three populations will be recruited: patients with CRPS undergoing treatment at the Functional Independence Restoration Program (FIRST), patients with CRPS undergoing treatment at the Pain Management Center and matching healthy controls. Participants will undergo three sessions: the first session will be scheduled immediately before or as soon as possible at the beginning of the patients' treatment; the second session will take place at the end of the patients' treatment; the last session will be scheduled six months post-treatment. The timing of the sessions of the healthy participants will follow a schedule similar to the FIRST patients. Each session will last approximately three hours and include acquisition of psychosocial, psychophysical, and brain imaging data in the child participants, as well as acquisition of psychosocial data in the parent participants.

Geraldine Schulze - geraldine.schulze@cchmc.org

ALL
10 years to 17 years old
This study is also accepting healthy volunteers
NCT03838107
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Inclusion Criteria:
All Children: * Age between 10 and 17 years old * Fluent in English Inpatients: * Diagnosis of CRPS * Former unsuccessful treatment for CRPS * Scheduled for or beginning the usual inpatient treatment for CRPS at the FIRST clinic at CCHMC. Outpatients: * Diagnosis of CRPS * Scheduled for or beginning the usual outpatient treatment for CRPS at the pain management clinic Healthy children: \- No diagnosis of chronic pain. Parents: * Fluent in English * Child participating in the study
Exclusion Criteria:
All child participants: * Weight/size incompatible with MRI scanner * Identification of brain, neurologic, or severe psychiatric abnormalities beyond those normally associated with chronic pain. * Documented developmental delays or impairment * Any MRI contra-indication, including * Braces, stents, clips, pace-maker or other metal implants affecting the safety of the participants in the scanner and/or the quality of the images * pregnancy * claustrophobia
BEHAVIORAL: observation and measure of trajectory of recovery in CRPS
Complex Regional Pain Syndromes
pediatric complex regional pain syndrome, fMRI, psychosocial, quantitative sensory testing, markers
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A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)

This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients with hematologic malignancies and other indications.

Jamie Wilhelm - jamie.wilhelm@cchmc.org

ALL
This study is NOT accepting healthy volunteers
NCT01351545
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Inclusion Criteria:
* Disorders affecting the hematopoietic system that are inherited, acquired, or result from myeloablative treatment * Signed informed consent (and signed assent, if applicable) obtained prior to study enrollment * Pediatric and adult patients of any age
Exclusion Criteria:
* Patients who are receiving only licensed CBUs * Cord blood transplant recipients at international transplant centers * Patients who are enrolled on another IND protocol to access the unlicensed CBU(s) * Patients whose selected unlicensed CBU(s) will be more than minimally manipulated
DRUG: A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs)
Hematologic Malignancies, Inherited Disorders of Metabolism, Inherited Abnormalities of Platelets, Histiocytic Disorders, Acute Myelogenous Leukemia (AML or ANLL), Acute Lymphoblastic Leukemia (ALL), Other Acute Leukemia, Chronic Myelogenous Leukemia (CML), Myelodysplastic (MDS) / Myeloproliferative (MPN) Diseases, Other Leukemia, Hodgkin Lymphoma, Non-hodgkin Lymphoma, Multiple Myeloma/ Plasma Cell Disorder (PCD), Inherited Abnormalities of Erythrocyte Differentiation or Function, Disorders of the Immune System, Autoimmune Diseases, Severe Aplastic Anemia
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Targeted Pediatric High-Grade Glioma Therapy

The goal of this study is to perform genetic sequencing on brain tumors from children, adolescents, and young adult patients who have been newly diagnosed with a high-grade glioma. This molecular profiling will decide if patients are eligible to participate in a subsequent treatment-based clinical trial based on the genetic alterations identified in their tumor.

- cancer@cchmc.org

ALL
12 months to 39 years old
This study is NOT accepting healthy volunteers
NCT05839379
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Inclusion Criteria:

• Age: Patients must be ≥12 months and ≤39 years of age at the time of enrollment onto this screening protocol.
• Diagnosis: Patients with newly diagnosed HGG, including DIPG are eligible. Diagnosis must have histologic confirmation from biopsy or resection. The diagnosis of HGG must have been confirmed by pathology review at the local site. For the diagnosis of DIPG, patients must have a tumor with pontine epicenter and diffuse involvement of at least 2/3 of the pons, with histopathology consistent with diffuse WHO grade 2-4 glioma (eg, diffuse astrocytoma, anaplastic astrocytoma, glioblastoma, H3K27-altered diffuse midline glioma). For all other tumors, histologic grade must be WHO grade 3-4.
• Disease Status: There are no disease status requirements for enrollment. * Measurable disease is not required. Patients without measurable disease are eligible. * Patients with metastatic/disseminated or multifocal disease or gliomatosis cerebri are eligible. * Patients with a primary spinal tumor are eligible. * Patients with secondary, radiation related HGG are eligible.
• Prior Therapy for HGG: Surgery, radiation, and/or dexamethasone are permissible. Temozolomide concurrent with radiation is permissible. Prior administration of avastin/bevacizumab is allowed (individual treatment arms have different washout period requirements, check individual arm eligibility). No other prior anticancer therapy for HGG will be allowed. * Participants screening for assignment to TarGeT-L may not have received radiation. Timing from surgery to start of RT: For patients who have started RT, radiation must have started \<42 days from definitive surgery or biopsy, however it is strongly recommended patients start RT within 31 days from definitive surgery (if patient had two surgeries, radiation must have started within 31 days from second surgery).
• Tumor Sample Availability OR results from previous molecular profiling/targeted sequencing * If a patient screens through OPTION #1, tumor sample in addition to normal comparator tissue (peripheral blood, saliva, or buccal swab) must be submitted for comprehensive molecular screening at the time of screening enrollment. * If a patient screens through OPTIONS #2 or #3, results from previously performed molecular profiling must be submitted following enrollment. It is highly recommended that results be uploaded within 7 days of enrollment (if results are available at time of enrollment) or within 7 days of results becoming available (if pending at time of enrollment) to allow adequate time for central review.
• Informed Consent: All patients and/or their parents or legally authorized representatives must sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines.
• Enrollment timeline: Patients are eligible to enroll on the TarGeT-SCR anytime between diagnosis and the following specific timepoints post completion of RT (if relevant) * Patients screening through OPTION #1 are eligible to enroll anytime between diagnosis and 10 days post RT (if completing RT). * Patients screening through OPTIONS #2 or #3 are eligible to enroll anytime between diagnosis and 21 days post RT (if completing RT). * Participants screening for TarGeT-L (lorlatinib) are eligible to enroll on TarGeT-SCR anytime between diagnosis and 31 days post definitive surgery (to allow time for molecular review). However, it is important to note the following: * For treatment protocols that include targeted therapy administered concurrently with RT, patients must start treatment within 10 calendar days of starting RT. * For treatment protocols that only include maintenance/adjuvant therapy (no systemic therapy given concurrently with radiation), patients must start treatment by 35 days post RT #SCREENING OPTIONS * OPTION1: Molecular screening through CONNECT TarGeT Clinical Testing Laboratories * OPTION2: Molecular screening through a national comprehensive tumor profiling program * OPTION3: Clinically validated targeted sequencing or focused profiling
Exclusion Criteria:
-Tumors that do not meet HGG and DIPG diagnoses specified above
High Grade Glioma, Diffuse Intrinsic Pontine Glioma, Anaplastic Astrocytoma, Glioblastoma, Glioblastoma Multiforme, Diffuse Midline Glioma, H3 K27M-Mutant, Metastatic Brain Tumor, WHO Grade III Glioma, WHO Grade IV Glioma
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Epilepsy Journey-An Executive Functioning Intervention for Teens With Epilepsy

The goal of this multi-site clinical trial is to determine the effectiveness of two components of a web-based intervention (Epilepsy Journey) to improve executive functioning in adolescents with epilepsy. The two components include web-based modules and problem-solving telehealth sessions with a therapist focused on executive functioning. This trial aims to answer the following questions: 1. Which components of Epilepsy Journey (web-based modules or telehealth sessions with a therapist) are essential for improving executive functioning in adolescents with epilepsy? 2. Which components of Epilepsy Journey (web-based modules or telehealth sessions with a therapist) are essential for improving quality of life in adolescents with epilepsy? Participants will be randomly assigned to one of four groups: 1) Epilepsy Journey web-based modules and telehealth sessions, 2) Epilepsy Journey web-based modules only, 3) telehealth sessions with a therapist only, or 4) treatment as usual. Participants will: * Independently review Epilepsy Journey web-based modules focused on executive functioning skills (\~15-30 minutes) and/or have weekly telehealth sessions (\~30-45 minutes) with a therapist for 14 weeks. * Complete measures of executive functioning (parent and teen-report) and quality of life (teen-report) at the start of the study, 14-, 26-, and 66- weeks after randomization. The NIH toolbox will be completed at the start of the study and 26-weeks after randomization. Additional measures will also be collected.

Stacy Buschhaus - stacy.buschhaus@cchmc.org

ALL
13 years to 17 years old
NA
This study is NOT accepting healthy volunteers
NCT06608966
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Inclusion Criteria:

• Age between 13-17 years at the time of enrollment
• Child lives at home with primary caregiver and is enrolled in school (excluding summer breaks).
• Confirmed diagnosis of epilepsy with seizures that are categorized as either generalized or focal in onset. Epilepsy is defined as: 1) At least two unprovoked seizures occurring more than 24-hours apart; or 2) One unprovoked seizure and a probability of further seizures similar to the general recurrence risk after two unprovoked seizures.
• Primary language of English
• Screening Inclusion: On the parent-reported Behavior Rating Inventory of Executive Function-2nd edition (BRIEF-2), have executive functioning deficits defined as at least 2 subclinical (60\ • Parent/legal guardian(s) willing to sign an IRB approved informed consent
• Participant willing to sign an Institutional Review Board approved assent
Exclusion Criteria:

• Parent or clinician-reported history in the adolescent of:
• developmental delay (e.g., autism spectrum disorder, pervasive development disorder, history of services for developmental delay or intellectual impairment in the past 5 years, known IQ\<70)
• severe mental illness (e.g., schizophrenia, bipolar disorder, eating disorder within the past 12 months, depression with active suicidal ideation or suicidal ideation/intent in the past 3 months)
• prior (3-months) or current history of trauma and/or stressor-related disorders (e.g. PTSD)
• recent or current significant medical disease (i.e., cardiovascular, hepatic, renal, gynecologic, musculoskeletal, metabolic or endocrine)
• brain injury or brain tumor; and/or
• epilepsy surgery
• any other medical and/or psychological condition that takes treatment precedence over the study intervention
• Clinician-reported diagnosis in the adolescent of
• epilepsy whose seizures are categorized only as either unknown onset or unclassified onset (defined as insufficient information to determine onset)
• epilepsy currently being treated at the time of enrollment by 3 or more antiseizure medications (ASMs) (excluding rescue medication use)
• epilepsy with a history of failure to achieve seizure freedom despite adequate use of 4 different anti-seizure medications
• a confirmed or suspected epileptic encephalopathy (e.g., electrical status epilepticus in sleep, Landau Kleffner syndrome, West syndrome)
• a confirmed or suspected progressive and degenerative disorder (e.g., mitochondrial disorders, metabolic disorders, autoimmune disorders)
• one or more episodes of status epilepticus within the 24 weeks prior to enrollment; and/or
• treatable causes of seizures, for example identified etiologies including metabolic, neoplastic, or active infectious origin.
• non-epileptic event/seizures
• Adolescents currently on the ketogenic diet
• Participation in a trial of an investigational drug or device within 30 days prior to screening
BEHAVIORAL: Epilepsy Journey web-based modules, BEHAVIORAL: Telehealth with a therapist
Epilepsy in Children, Executive Dysfunction
adolescents, executive functioning, seizures, epilepsy, behavioral trial, web-based intervention
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AWARE: Management of ADHD in Autism Spectrum Disorder

This study is a pragmatic clinical trial examining the comparative effectiveness of two stimulant medications (methylphenidate and amphetamine) in the treatment of ADHD in children and adolescents with autism. Using a sequential, multiple assignment randomization trial (SMART) design the study will not only assess these two medications but also the role of an increasingly popular class of ADHD medication, the alpha-2 agonists. Findings from this study will help improve clinicians' approach to medication selection and reduce the repeated trials of multiple medications that are current standard care.

Courtney Herberger - Courtney.Herberger@cchmc.org

ALL
4 years to 17 years old
PHASE4
This study is NOT accepting healthy volunteers
NCT05916339
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Inclusion Criteria:

• Participant and/or legal caregiver must be willing and able to give informed consent/assent for participation in this study.
• Participant and/or legal caregiver must be willing and able (in the Investigator's opinion) to comply with all study requirements.
• Participant must be between 4 and 17 years of age (inclusive) at time of enrollment.
• Participant must have a confirmed diagnosis of ASD based on supportive evidence (e.g. referring physician's report, medical records, such as ADOS or CARS, etc.).
• Participant must have the ability to consistently take medication (via pill, liquid or mixed with food/liquid).
• Participant must have a confirmed diagnosis of ADHD (based upon DSM-5 criteria and supportive evidence).
• Participant must have a consistent reporter (e.g., parent) who spends regular time with the child.
• Participant can be on other psychotropic medications (selective serotonin reuptake inhibitor (SSRI), atypical antipsychotic, anticonvulsant) if dose has been stable for \> 4 weeks prior to consent with no plans for a dose change during the study.
• It has been at least 7 days since the participant last took an ADHD medication and the presiding clinician believes this to be a sufficient amount of time.
• Caregiver must be sufficiently fluent in English or Spanish to be able to complete questionnaires relevant to this study.
Exclusion Criteria:

• Participant has taken ADHD medication within the past 7 days.
• Participant is not stable on other medications (\< 4 weeks).
• Any other risk factor that might prevent patient from safely taking the study medications. * There are no inclusion/exclusion criteria based upon participant IQ. We will include individuals across the entire range of cognition, just as practitioners are asked to treat ADHD in children with ASD across the entire IQ range.
DRUG: Randomization to either Amphetamine (AMP) class of stimulant medication or Methylphenidate (MPH) class of stimulant medication, DRUG: Randomization to either Alpha 2 agonist class of medication or alternate class of stimulant.
ADHD, Autism Spectrum Disorder
ADHD, Autism, ASD
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NANT 2021-01 Phase II STING (Sequential Temozolomide, Irinotecan, NK Cells and GD2 mAb) Trial

This is a phase II study looking at patient response to treatment with the combination dinutuximab, temozolomide, irinotecan, and GM-CSF.

- cancer@cchmc.org

ALL
1 year to 31 years old
PHASE2
This study is NOT accepting healthy volunteers
NCT06450041
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Inclusion Criteria:
* Patients must be ≥ 1 year and ≤31 years of age at the time of enrollment on the study. * Patients must have a diagnosis of neuroblastoma either by histologic verification of neuroblastoma and/or demonstration of tumor cells in the bone marrow with increased urinary catecholamines. * Patients must have high-risk neuroblastoma according to COG risk classification at the time of study registration. Patients whose disease was initially considered low or intermediate risk but then reclassified as high-risk neuroblastoma prior to enrollment also meet this criteria. * Patients must have at least ONE of the following: 1\) Recurrent/progressive disease after the diagnosis of high risk neuroblastoma at any time prior to enrollment - regardless of response to frontline therapy. (Note that this excludes patients initially considered low or intermediate risk that progressed to high risk disease but have not progressed after the diagnosis of high risk neuroblastoma). 2\) If no prior history of recurrent/progressive disease since the diagnosis of high-risk neuroblastoma, 2a) Refractory disease: A best overall response of no response/stable disease since diagnosis of high-risk neuroblastoma AND after at least 4 courses of induction therapy. 2b) Persistent disease: A best overall response of partial response since diagnosis of high-risk neuroblastoma AND after at least 4 courses of induction therapy * Patients must have at least ONE of the following (lesions may have received prior radiation therapy as long as they meet the other criteria listed below) based on institutional assessment: 1\) Bone Sites
• a) MIBG avid tumors: patients must meet one of the following criteria: a. Patients with recurrent/progressive or refractory disease: i. Must have at least one MIBG avid bone site on planar imaging OR ii. Must have \> 2 avid bone lesions on SPECT. iii. A biopsy is not required unless the above imaging criteria are not met. b. Patients with persistent disease: i. If a patient has 3 or more MIBG avid sites by planar or SPECT imaging (including soft tissue and/or bone), then no biopsy is required. ii. If a patient has only 1 or 2 MIBG avid sites by planar or SPECT imaging (including soft tissue and/or bone) then biopsy confirmation of neuroblastoma and/or ganglioneuroblastoma in at least one MIBG avid site present at the time of enrollment is required. Bone lesions may be biopsied at any time point prior to enrollment. 1b) For MIBG non-avid tumors, patients must have biopsy confirmation of neuroblastoma and/or ganglioneuroblastoma from a lesion at any time prior to enrollment of at least one site (with or without FDG-PET uptake). 2\) Bone Marrow Any amount of tumor cells in the bone marrow (including neuroblasts, mature and maturing ganglion cells) done at the time of study enrollment based on routine morphology and/or immunohistochemistry in at least one sample from bilateral aspirates and biopsies. 3\) Soft Tissue Sites 3a) At least one soft tissue lesion that meets criteria for a TARGET lesion as defined by:
• SIZE: Lesion can be accurately measured in at least one dimension with a longest diameter ≥ 10 mm, or for discrete lymph nodes ≥ 15mm on short axis. Lesions meeting size criteria will be considered measurable.
• In addition to size, a lesion needs to meet ONE of the following criteria except for patients with parenchymal CNS lesions which will only need to meet size criteria:
• For MIBG avid tumors: lesion must be MIBG avid and meet one of the following criteria:
• For patients with recurrent/progressive or refractory disease: i. No biopsy is required
• For patients with persistent disease: i. If a patient has 3 or more MIBG avid sites by planar or SPECT imaging (including soft tissue and/or bone), then no biopsy is required. ii. If a patient has only 1 or 2 MIBG avid sites by planar or SPECT imaging (including soft tissue and/or bone), then biopsy confirmation of neuroblastoma and/or ganglioneuroblastoma in at least one MIBG avid site present at the time of enrollment is required. Soft tissue lesions may be biopsied at any time point prior to enrollment. b. For MIBG non-avid tumors, patient must have biopsy confirmation of neuroblastoma and/or ganglioneuroblastoma at any time prior to enrollment from soft tissue lesion (with or without FDG uptake) present at time of enrollment. 3b) At least one non-target soft tissue lesion that is not measurable, but had a biopsy positive for neuroblastoma and/or ganglioneuroblastoma at any time prior to enrollment OR is MIBG avid on planar imaging. * Patients must have a Lansky (≤ 16 years) or Karnofsky (\> 16 years) score of ≥ 50 (Appendix I). Note: Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score. * Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to study registration. * Patients must not have received the therapies indicated below after disease evaluation or within the specified time period prior to registration on this study as follows:
• Myelosuppressive chemotherapy: must not have received within 2 weeks prior to registration.
• Biologic anti-neoplastics- agents not known to be associated with reduced platelet or ANC counts (including retinoids): must not have received within 7 days prior to registration.
• Monoclonal antibodies: must not have received last dose within 14 days of registration and resolution of all toxicities.
• Cellular Therapy (e.g. modified T cells, NK cells, dentritic cells etc.): must not have received within 3 weeks and resolution of all toxicities.
• Radiation: must not have received small port radiation within 7 days prior to registration, large field radiation within 12 weeks, and 131I-MIBG therapy or other radiopharmaceutical within 6 weeks.
• Hematopoietic Stem Cell Transplant- none following myeloblative therapy within 6 weeks
• Any other investigational agents (covered under another IND within 14 days
• Strong inducers or inhibitors of CYP3A4 * Hematologic Function: NOTE: No short acting hematopoietic growth factors within 7 days of blood draw documenting eligibility and no long-acting hematopoietic growth factors within 14 days of blood draw documenting eligibility
• Absolute Neutrophil count ≥750/µL
• Platelet count ≥ 75,000/µL, transfusion independent (no platelet transfusions within 7 days of blood draw documenting eligibility) Patients with known bone marrow metastatic disease will be eligible for study as long as they meet hematologic function criteria above. * Renal Function Patients must have adequate renal function defined as age-adjusted serum creatinine ≤1.5 ULN for age * Liver Function
• Total bilirubin ≤ 1.5 x ULN for age; and,
• SGPT (ALT) ≤ 135 U/L (≤ 3x ULN). Note that for ALT, the upper limit of normal for all sites is defined as 45 U/L. * Cardiac Function
• Normal ejection fraction (≥ 55%) documented by either echocardiogram OR
• Normal fractional shortening (≥ 27%) documented by echocardiogram * Pulmonary Function No evidence of dyspnea at rest * Reproductive Function All females ≥ Tanner stage 2 and post-menarchal of childbearing potential must have a negative beta-HCG within 7 days prior to study registration. Males and females of reproductive age and childbearing potential must commit to using effective contraception for the duration of their participation. * Central Nervous System (CNS) Patients with a history of intraparenchymal or leptomeningeal based CNS disease must have no clinical or radiological evidence of active CNS disease at the time of study enrollment. Patients with skull-based tumors with direct intracranial extension are eligible as long as there are no neurologic signs or symptoms related to the lesion.
Exclusion Criteria:
* Patients who are pregnant, breast feeding, or unwilling to use effective contraception during the study * Patients who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study. * Patients with disease of any major organ system that would compromise their ability to withstand therapy. * Patients with \> Grade 2 diarrhea. * Patients who have undergone a prior allogeneic stem cell or solid organ transplant. * Patients who are on hemodialysis. * Patients with an active or uncontrolled infection. Patients on prolonged antifungal therapy are still eligible if they are culture negative, afebrile, and meet other organ function criteria. * Patients with known history of human immunodeficiency virus (HIV) infection, hepatitis B, or hepatitis C. Testing is not required in the absence of clinical findings or suspicion. * Patients must not have been diagnosed with any other malignancy. * Patients with history of Grade 4 Allergic reactions to anti-GD2 antibody therapy or reactions that caused permanent discontinuation of therapy. * Patients with history of progressive disease while receiving therapy per ANBL1221. * Patient declines participation in the NANT biology study and the site has not been granted a waiver from participation. * Systemic Steroids and Immunosuppressive Medications * Patients who have received pharmacologic doses of systemic steroids 7 days prior to study registration or likely to require them after study registration. Note: Exceptions are the following:
• Patients known to require 2 mg/kg or less of hydrocortisone (or an equivalent dose of an alternative corticosteroid) as premedication for blood product administration.
• The use of conventional doses of inhaled steroids for the treatment of asthma
• The use of physiologic doses of steroids for patients with known adrenal insufficiency. * Patients on any other immunosuppressive medications (e.g., cyclosporine, tacrolimus) at the time of study registration.
DRUG: Universal Donor (UD) TGFβi NK Cells, DRUG: Temozolomide, DRUG: Irinotecan, DRUG: Dinutuximab, DRUG: GM-CSF
Neuroblastoma
Relapsed Neuroblastoma, Refractory Neuroblastoma, Cellular Therapy, Immunotherapy, Chemoimmunotherapy
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Targeted Therapy Directed by Genetic Testing in Treating Patients With Locally Advanced or Advanced Solid Tumors, The ComboMATCH Screening Trial

This ComboMATCH patient screening trial is the gateway to a coordinated set of clinical trials to study cancer treatment directed by genetic testing. Patients with solid tumors that have spread to nearby tissue or lymph nodes (locally advanced) or have spread to other places in the body (advanced) and have progressed on at least one line of standard systemic therapy or have no standard treatment that has been shown to prolong overall survival may be candidates for these trials. Genetic tests look at the unique genetic material (genes) of patients' tumor cells. Patients with some genetic changes or abnormalities (mutations) may benefit from treatment that targets that particular genetic mutation. ComboMATCH is designed to match patients to a treatment that may work to control their tumor and may help doctors plan better treatment for patients with locally advanced or advanced solid tumors.

- cancer@cchmc.org

ALL
PHASE2
This study is NOT accepting healthy volunteers
NCT05564377
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Inclusion Criteria:
* Patient must have measurable disease * Patient must have an Eastern Cooperative Oncology Group (ECOG) performance status between 0-2 OR patient must have Lansky performance status of \>= 50% or Karnofsky performance status of \>= 50% * Patient must be deemed potentially eligible for a ComboMATCH Treatment Trial as assessed by the enrolling provider * All patients must have sequencing results available from a National Cancer Institute (NCI) credentialed Designated Laboratory (DL) * Patients must have locally advanced or advanced histologically documented solid tumors requiring therapy and meet one of the following criteria: * Patients must have progressed on at least one line of standard systemic therapy OR * Patients whose disease has no standard treatment that has been shown to prolong overall survival * Patient must meet one of the following requirements: * Patients 18 years and older who have tumor amenable to minimal risk image-guided or direct vision biopsy and must be willing and able to undergo a tumor biopsy to obtain samples for research if the patient is to enroll in a ComboMATCH treatment trial OR * Patients 18 years and older who do not have disease that is biopsiable at minimal risk to the patient must confirm availability of an archival tumor tissue specimen for submission for research if the patient enrolls to a ComboMATCH Treatment Trial. This tumor tissue must meet the following criteria: * Tissue must have been collected within 12 months prior to registration to the EAY191 Registration Trial * Patient must not have had a Response Evaluation Criteria in Solid Tumors (RECIST) response (complete response \[CR\] or partial response \[PR\]) to any intervening therapy after collection of the tissue * Formalin-fixed paraffin-embedded tumor tissue block(s) or slides must be available OR * Patients under 18 years old must confirm availability of an archival tumor tissue specimen for submission for research if patient enrolls to a ComboMATCH Treatment Trial. This tumor tissue must meet the following criteria: * Formalin-fixed paraffin-embedded tumor tissue block(s) or slides must be available * NOTE: See specific ComboMATCH Treatment Trial protocol for tissue collection and management instructions. Performance of the mandatory research biopsy or submission of pre-trial formalin-fixed paraffin-embedded (FFPE) and collection and submission of the blood specimens for the integrated studies will be performed under the consent authority of the specific treatment trial protocol to which the patient is registered. No procedures to collect specimens for research only are to be performed for patients registered to the EAY191 Registration Trial only * NOTE: Each ComboMATCH Treatment Trial contains specific eligibility criteria. If patient is found to not be eligible for the assigned ComboMATCH Treatment Trial, indication of ineligibility will trigger re-evaluation and potential assignment to another Treatment Trial
DRUG: Alpelisib, DRUG: Binimetinib, PROCEDURE: Biopsy Procedure, PROCEDURE: Biospecimen Collection, PROCEDURE: Bone Marrow Aspiration, PROCEDURE: Bone Marrow Biopsy, PROCEDURE: Bone Scan, PROCEDURE: Computed Tomography, PROCEDURE: Echocardiography Test, DRUG: Fluorouracil, DRUG: Fulvestrant, DRUG: Ipatasertib, DRUG: Leucovorin, PROCEDURE: Magnetic Resonance Imaging, PROCEDURE: Multigated Acquisition Scan, PROCEDURE: Mutation Carrier Screening, DRUG: Neratinib Maleate, DRUG: Nilotinib Hydrochloride Monohydrate, DRUG: Olaparib, DRUG: Oxaliplatin, DRUG: Paclitaxel, DRUG: Palbociclib, BIOLOGICAL: Panitumumab, PROCEDURE: Positron Emission Tomography, DRUG: Selumetinib Sulfate, DRUG: Sotorasib
Advanced Malignant Solid Neoplasm, Anatomic Stage III Breast Cancer AJCC v8, Anatomic Stage IV Breast Cancer AJCC v8, Locally Advanced Malignant Solid Neoplasm, Malignant Female Reproductive System Neoplasm, Metastatic HER2-Negative Breast Carcinoma, Metastatic Malignant Solid Neoplasm, Recurrent Endometrial Carcinoma, Recurrent Fallopian Tube Carcinoma, Recurrent Malignant Female Reproductive System Neoplasm, Recurrent Malignant Solid Neoplasm, Recurrent Ovarian Carcinoma, Recurrent Primary Peritoneal Carcinoma, Unresectable HER2-Negative Breast Carcinoma, Unresectable Malignant Solid Neoplasm
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