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340 Study Matches
A Study to Learn About the Study Medicine Called Zavegepant (PF-07930207) in Children With a History of Migraine
Michelle Shin - michelle.shin@cchmc.org
ALL
6 years to 11 years old
PHASE1
NCT06995729
Inclusion Criteria:
• Participants aged 6 years to less than 12 years.
• Documented medical history of migraine with or without aura for at least 6 months before the Screening Visit.
• Weight \>15 kg at the Screening Visit
Exclusion Criteria:
• Evidence or history of clinically significant disease.
• Continuous migraine (defined as an unrelenting headache) within 1 month prior to Screening Visit.
• Atypical migraine types, complications of a migraine, or a confounding and clinically significant pain syndrome.
• Conditions that may affect the administration or absorption of the nasal product
• Any psychiatric condition that is uncontrolled and/or untreated, including: * Clinically significant depression (Promis Parent Proxy SF 2.0 Depressive Scale T score ≥70) * Suicidal ideation and behaviour (C-SSRS: any "yes" to items 2 to 5 or any "yes" to suicide behaviours)
• Serum Total bilirubin \>1.5 × ULN (Upper Limit of Normal), AST (Aspartate Transferase) or ALT (Alanine Transaminase) \>2 × ULN
• Abnormal ECG (Electrocardiogram) at screening visit
DRUG: Zavegepant
Acute Treatment of Migraine
migraine, zavegepant, pediatric
A Study to Assess the Incidence of Skin Cancers in Patients With Epidermolysis Bullosa Receiving Filsuvez (FOSteR)
Bret Augsburger - bret.augsburger@cchmc.org
ALL
NCT06423573
Inclusion Criteria:
* Patients with a confirmed diagnosis of dystrophic EB or junctional EB
Exclusion Criteria:
* None DRUG: Filsuvez
Epidermolysis Bullosa, Dystrophic, Epidermolysis Bullosa, Junctional
Filsuvez, Birch bark extract
Proton MRI of MSK Disorders
Ashley Borden, MPH - ashley.borden@cchmc.org
ALL
5 years and over
NCT07845708
Inclusion Criteria:
* Age ≥ 5 years
Exclusion Criteria:
* History of any comorbidity reviewed at the Screening phone call that, in the opinion of the investigator, might confound the results of the study or pose an additional risk to the subject.
* Standard MRI exclusion criteria as set forth by the CCHMC Radiology Department and IRC.
* Pregnancy DIAGNOSTIC_TEST: MRI
Muskuloskeletal Diseases, Healthy, Respiratory
Allogeneic Hematopoietic Cell Transplantation for Refractory Systemic Juvenile Idiopathic Arthritis.
Brian Hils - brian.hils@cchmc.org
ALL
Up to 35 years old
NCT07845942
Inclusion Criteria:
* Patients \<35 years of age are eligible, and participants must meet all the following criteria to be eligible for this study:
* Diagnosis: Systemic Juvenile Idiopathic Arthritis (sJIA) diagnosed prior to age 16, fulfilling ILAR classification criteria for sJIA or the provisional PRINTO criteria for systemic arthritis. The diagnosis and disease course must be reviewed and confirmed by a pediatric rheumatologist.
* Refractory Disease: sJIA that is refractory to standard therapy, defined as meeting at least one of these conditions despite appropriate treatment:
* Persistent JIA activity: Ongoing active disease (e.g. uncontrolled arthritis or systemic features such as fever, rash, organ inflammation) or progressive joint damage or liver dysfunction that has not responded to biologic agents (IL-1/IL-6 inhibitors) and conventional therapies, or inability to wean from high-dose corticosteroids (steroid dependence).
* Recurrent MAS: Two or more episodes of macrophage activation syndrome within a 2-year period, indicating a failure to control the hyperinflammation with medical management. (MAS is defined by clinical and laboratory criteria such as fever, cytopenias, hyperferritinemia, hemophagocytosis, etc., per standard definitions.)
* sJIA-LD: Presence of lung disease related to sJIA, either symptomatic LD (e.g. chronic cough, tachypnea, hypoxemia) or progressive LD on imaging (worsening interstitial changes), meeting clinical/imaging features such as patchy ground-glass opacities, septal and pleural thickening, or clubbing of digits. sJIA-LD should be confirmed by a pulmonologist/radiologist (e.g. HRCT findings).
Exclusion Criteria:
* Uncontrolled Infection: Presence of a severe, uncontrolled infection at time of enrollment (e.g. sepsis, pneumonia not responding to therapy).
* Significant Organ Failure:
* Cardiac: EF \< 40%
* Renal: GFR \< 30 ml/hr
* Active Malignancy: Concurrent cancer diagnosis or history of malignant disease requiring active treatment.
* Pregnancy or Lactation: Female patients who are pregnant or breastfeeding. Female participants of childbearing potential must have a negative pregnancy test clinical result at screening (pregnancy testing is done as standard of care for all transplant patients). Refractory Systemic Juvenile Idiopathic Arthritis, Systemic Juvenile Idiopathic Arthritis, Systemic Juvenile Idiopathic Arthritis Associated Lung Disease
A Study of CD19 Targeted CAR T Cell Therapy in Pediatric Patients With Relapsed or Refractory B Cell Acute Lymphoblastic Leukemia (B ALL) and Aggressive Mature B-cell Non-Hodgkin Lymphoma (B NHL)
- cancer@cchmc.org
ALL
0 years to 18 years old
PHASE1
NCT06173518
INCLUSION CRITERIA:
* \< 18 years old at screening
* ≥ 6 kg body weight at screening
Pediatric patients with r/r B ALL
r/r CD19-positive aggressive mature B including the B NHL subtypes: i) diffuse large B cell lymphoma, ii) Burkitt's lymphoma, iii) primary mediastinal large B cell lymphoma, iv) high-grade B cell lymphoma (not otherwise specified).
* Karnofsky (age ≥ 10 years) or Lansky (age \< 10 year) performance status score ≥ 50%.
* In participants with B ALL, local documentation of CD19 expression on leukemic blasts in the BM, peripheral blood, or cerebrospinal fluid or biopsy done no more than 30 days prior to consent.
* Adequate renal, hepatic, pulmonary, and cardiac function.
EXCLUSION CRITERIA:
* Diagnosis of chronic myelogenous leukemia in lymphoid blast crisis.
* History or presence of clinically relevant central nervous system (CNS) pathology unrelated to CNS leukemia.
* Presence of active or uncontrolled fungal, bacterial, viral, or other infection requiring systemic antimicrobials for management.
* Received prior (\< 3 months before obe cel infusion) stem cell transplantation.
* Prior CD19 targeted therapy other than blinatumomab.
* Experienced Grade ≥ 3 neurotoxicity following blinatumomab.
BIOLOGICAL: AUTO1
Relapsed or Refractory B Cell Acute Lymphoblastic Leukemia, Relapsed or Refractory B Cell Non-Hodgkin Lymphoma
B cell acute lymphoblastic leukemia, B cell Non-Hodgkin lymphoma, Relapsed B cell acute lymphoblastic leukemia, Relapsed B cell Non-Hodgkin lymphoma, Refractory B cell acute lymphoblastic leukemia, Refractory B cell Non-Hodgkin lymphoma, Aggressive mature B cell Non-Hodgkin lymphoma, Pediatric ALL, Pediatric NHL, Obecabtagene autoleucel, CD19-positive CAR T cell, Obe-cel
EPIK-P4: A Phase II Single-arm Study to Assess the Efficacy, Safety and Pharmacokinetics of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum (PROS)
- sarah.price@cchmc.org
ALL
2 years to 100 years old
PHASE2
NCT06997588
Key
• Male or female participants aged ≥2 years at the time of informed consent/assent.
• Participants with diagnosis of PROS (according to Clinical Diagnostic Criteria for PROS proposed by Keppler Noreuil et al 2014) with symptomatic AND progressive overgrowth, who have syndromic disease or isolated features (with the exception of isolated macrodactyly, macrocephaly or epidermal nevus) at the time of informed consent/assent.
• Documented evidence of a somatic mutation(s) in the PIK3CA gene performed in local laboratories using a DNA-based test AND available archival tissue (if archival tissue sample is not available, a fresh biopsy should be performed, if it is not clinically contraindicated) at the time of informed consent/assent.
• Karnofsky (in participants \>16 years of age at study entry) or Lansky (≤16 years of age at study entry) performance status index ≥50.
• PGI-S score of mild, moderate, severe, or very severe at screening.
• Adequate bone marrow and organ function.
• Presence of at least 1 PROS-related measurable lesion (longest diameter ≥2 cm) confirmed by BIRC assessment and associated with complaints, clinical symptoms or functional limitations affecting the participant's everyday life. Key
• Participant with only isolated macrodactyly, epidermal nevus/nevi and macroencephaly (the only clinical feature or a combination of any of three of them), in absence of other PROS-related lesions at the time of informed consent/assent.
• Previous treatment with alpelisib and/or any other phosphatidylinositol 3-kinase (PI3K) inhibitor(s) (except treatment attempt, defined as the attempt to treat PROS with any of PI3K inhibitors, with treatment duration less than 2 weeks and stopped at least 2 weeks prior to the first dose of study medication with alpelisib).
• Debulking or other major surgery performed within 3 months at the time of informed consent/assent.
• Radiation exposure for PROS treatment purpose within 12 months prior to informed consent/assent.
• Clinically meaningful PROS-related thrombotic event (Grade 2 and more as per CTCAE v4.03) within 15 days before study treatment initiation, and/or sclerotherapy/embolization for vascular complications performed within 6 weeks before informed consent/assent.
• Clinically meaningful bleeding from PROS-related lesion (Grade 2 and more as per CTCAE v4.03) within 15 days before study treatment initiation.
• Participants with clinically significant worsening of PROS-related laboratory abnormalities, physical signs and symptoms (such as, but not limited to increase of D-dimers, worsening of underlying pain, newly occurring swelling or redness) indicating an uncontrolled condition during the screening phase. Other inclusion/exclusion criteria may apply
Inclusion Criteria:
• Male or female participants aged ≥2 years at the time of informed consent/assent.
• Participants with diagnosis of PROS (according to Clinical Diagnostic Criteria for PROS proposed by Keppler Noreuil et al 2014) with symptomatic AND progressive overgrowth, who have syndromic disease or isolated features (with the exception of isolated macrodactyly, macrocephaly or epidermal nevus) at the time of informed consent/assent.
• Documented evidence of a somatic mutation(s) in the PIK3CA gene performed in local laboratories using a DNA-based test AND available archival tissue (if archival tissue sample is not available, a fresh biopsy should be performed, if it is not clinically contraindicated) at the time of informed consent/assent.
• Karnofsky (in participants \>16 years of age at study entry) or Lansky (≤16 years of age at study entry) performance status index ≥50.
• PGI-S score of mild, moderate, severe, or very severe at screening.
• Adequate bone marrow and organ function.
• Presence of at least 1 PROS-related measurable lesion (longest diameter ≥2 cm) confirmed by BIRC assessment and associated with complaints, clinical symptoms or functional limitations affecting the participant's everyday life. Key
Exclusion Criteria:
• Participant with only isolated macrodactyly, epidermal nevus/nevi and macroencephaly (the only clinical feature or a combination of any of three of them), in absence of other PROS-related lesions at the time of informed consent/assent.
• Previous treatment with alpelisib and/or any other phosphatidylinositol 3-kinase (PI3K) inhibitor(s) (except treatment attempt, defined as the attempt to treat PROS with any of PI3K inhibitors, with treatment duration less than 2 weeks and stopped at least 2 weeks prior to the first dose of study medication with alpelisib).
• Debulking or other major surgery performed within 3 months at the time of informed consent/assent.
• Radiation exposure for PROS treatment purpose within 12 months prior to informed consent/assent.
• Clinically meaningful PROS-related thrombotic event (Grade 2 and more as per CTCAE v4.03) within 15 days before study treatment initiation, and/or sclerotherapy/embolization for vascular complications performed within 6 weeks before informed consent/assent.
• Clinically meaningful bleeding from PROS-related lesion (Grade 2 and more as per CTCAE v4.03) within 15 days before study treatment initiation.
• Participants with clinically significant worsening of PROS-related laboratory abnormalities, physical signs and symptoms (such as, but not limited to increase of D-dimers, worsening of underlying pain, newly occurring swelling or redness) indicating an uncontrolled condition during the screening phase. Other inclusion/exclusion criteria may apply
DRUG: Alpelisib
PIK3CA-related Overgrowth Spectrum (PROS)
PIK3CA-related overgrowth spectrum (PROS), Alpelisib (BYL719), Phase II, Adult, Pediatric, Adolescent
Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation. (EPIK-L1)
Price Sarah - sarah.price@cchmc.org
ALL
0 years to 100 years old
PHASE2
NCT05948943
Key inclusion criteria:
• Signed informed consent and assent (when applicable) from the participant, parent, legal authorized representative or guardian.
• Participant must be willing to remain at the clinical site as required by the protocol and be willing to adhere to study restrictions and examination schedules.
• Participant has a physician confirmed and documented diagnosis of a symptomatic LyM at the time of informed consent (Note: the physician must confirm that the LyM cannot be included under the PROS diagnostic criteria).
• Participant is not considered as a candidate for or is not willing to receive non-drug therapies including but not limited to sclerotherapy, embolization, and surgery until the completion of Week 24 in Stage 1 and 2.
• Participant has evidence of a somatic mutation(s) in the PIK3CA gene prior to randomization.
• Participant has at least one measurable LyM lesion confirmed by BIRC assessment prior to randomization.
• Participants must be able to ingest study drug (either in tablet form or as a drinkable suspension \[Groups 1 to 4\] or granules or as an oral suspension \[Group 5\]) as assessed within 7 days before study treatment start. Drug administration via feeding tubes is allowed. Key exclusion criteria:
• Participant has a physician-confirmed and documented diagnosis of PROS at the time of informed consent.
• Participant has a physician-confirmed and documented diagnosis of a Central Conducting Lymphatic Anomaly, General Lymphatic Anomaly, Gorham-Stout disease, Kaposiform lymphangiomatosis at the time of informed consent.
• Participant has a known history of Stevens-Johnson syndrome, erythema multiforme, or toxic epidermal necrolysis at the time of informed consent.
• Participant has an established diagnosis of type I diabetes mellitus or uncontrolled type II diabetes mellitus at the time of informed consent.
• Participant had previous treatment with alpelisib and/or any other PI3K inhibitors with treatment duration longer than 2 weeks at the time of informed consent. Other inclusion/exclusion criteria may apply
• Signed informed consent and assent (when applicable) from the participant, parent, legal authorized representative or guardian.
• Participant must be willing to remain at the clinical site as required by the protocol and be willing to adhere to study restrictions and examination schedules.
• Participant has a physician confirmed and documented diagnosis of a symptomatic LyM at the time of informed consent (Note: the physician must confirm that the LyM cannot be included under the PROS diagnostic criteria).
• Participant is not considered as a candidate for or is not willing to receive non-drug therapies including but not limited to sclerotherapy, embolization, and surgery until the completion of Week 24 in Stage 1 and 2.
• Participant has evidence of a somatic mutation(s) in the PIK3CA gene prior to randomization.
• Participant has at least one measurable LyM lesion confirmed by BIRC assessment prior to randomization.
• Participants must be able to ingest study drug (either in tablet form or as a drinkable suspension \[Groups 1 to 4\] or granules or as an oral suspension \[Group 5\]) as assessed within 7 days before study treatment start. Drug administration via feeding tubes is allowed. Key exclusion criteria:
• Participant has a physician-confirmed and documented diagnosis of PROS at the time of informed consent.
• Participant has a physician-confirmed and documented diagnosis of a Central Conducting Lymphatic Anomaly, General Lymphatic Anomaly, Gorham-Stout disease, Kaposiform lymphangiomatosis at the time of informed consent.
• Participant has a known history of Stevens-Johnson syndrome, erythema multiforme, or toxic epidermal necrolysis at the time of informed consent.
• Participant has an established diagnosis of type I diabetes mellitus or uncontrolled type II diabetes mellitus at the time of informed consent.
• Participant had previous treatment with alpelisib and/or any other PI3K inhibitors with treatment duration longer than 2 weeks at the time of informed consent. Other inclusion/exclusion criteria may apply
DRUG: Alpelisib, DRUG: Placebo
Lymphatic Malformations
Lymphatic malformations (LyM), Cystic lymphangioma, Cystic hygroma, Lymphangioma circumscriptum, Cavernous lymphangioma, Lymphangioma, Macrocystic lymphatic malformation, Microcystic lymphatic malformationn, Alpelisib
Study to Determine the Efficacy and Safety of Asciminib in Pediatric Patients With Ph+ CML-CP
- cancer@cchmc.org
ALL
1 year to 18 years old
PHASE2
NCT07354074
Key
• Signed informed consent must be obtained prior to participation in the study.
• Male or female participants 1 and \< 18 years of age at study enrollment
• Diagnosis of CML-CP (Apperley et al 2025) with cytogenetic confirmation of Philadelphia positive (Ph+) chromosome
• For participants with CML-CP newly diagnosed within 3 months of screening OR 5 For participants with CML - CP with high risk of developing resistance or intolerance to previous TKI:
• Unfavourable response to TKI is defined following the Apperley et al 2025 guidelines as: * At three months after the initiation of therapy: BCR::ABL1 ratio \> 10% IS (if confirmed within 1-3 months) * At six months after the initiation of therapy: BCR::ABL1 ratio \> 10% IS * At twelve months after initiation of therapy: BCR::ABL1 ratio \> 1% IS * At any time loss of previous response * At any time emergent resistant BCR::ABL1 mutations or high-risk ACA from prior TKI treatment as per local test results
• Intolerance to TKI is defined as: * Non-hematologic intolerance: participants with grade 3 or 4 toxicity while on therapy (in which case the patient is eligible whether or not there was a dose reduction); or with persistent grade 2 toxicity unresponsive to optimal management including dose adjustments (unless dose reduction is not considered in the best interest of the patient if response is already suboptimal) * Hematologic intolerance: participants with grade 3 or 4 toxicity (absolute neutrophil count \[ANC\] or platelets) while on therapy that is recurrent after dose reduction to the lowest doses of the TKI 6\. Evidence of typical BCR::ABL1 transcript \[e14a2 and/or e13a2\] at the time of screening which are amenable to standardized RQ-PCR quantification. 7\. Performance status: Karnofsky ≥ 50% for participants ≥ 16 years of age, and Lansky ≥ 50 for participants \< 16 years of age at the time of screening. Key
• Known second chronic phase (CP) of CML after previous progression to Accelerated Phase (AP)/Blast Phase (BP).
• Previous treatment with a hematopoietic stem-cell transplantation.
• Patient planned to undergo allogeneic hematopoietic stem cell transplantation
• Known presence of a BCR::ABL1 mutation with known resistance to study treatment in accordance with the most recent public version of international CML clinical guidelines (e.g. NCCN CML treatment guidelines v 1.2026 and Apperley et al 2025) any time prior to study entry Other inclusion/exclusion criteria may apply.
Inclusion Criteria:
Participants eligible for inclusion in this study must meet all of the following criteria:
• Signed informed consent must be obtained prior to participation in the study.
• Male or female participants 1 and \< 18 years of age at study enrollment
• Diagnosis of CML-CP (Apperley et al 2025) with cytogenetic confirmation of Philadelphia positive (Ph+) chromosome
• For participants with CML-CP newly diagnosed within 3 months of screening OR 5 For participants with CML - CP with high risk of developing resistance or intolerance to previous TKI:
• Unfavourable response to TKI is defined following the Apperley et al 2025 guidelines as: * At three months after the initiation of therapy: BCR::ABL1 ratio \> 10% IS (if confirmed within 1-3 months) * At six months after the initiation of therapy: BCR::ABL1 ratio \> 10% IS * At twelve months after initiation of therapy: BCR::ABL1 ratio \> 1% IS * At any time loss of previous response * At any time emergent resistant BCR::ABL1 mutations or high-risk ACA from prior TKI treatment as per local test results
• Intolerance to TKI is defined as: * Non-hematologic intolerance: participants with grade 3 or 4 toxicity while on therapy (in which case the patient is eligible whether or not there was a dose reduction); or with persistent grade 2 toxicity unresponsive to optimal management including dose adjustments (unless dose reduction is not considered in the best interest of the patient if response is already suboptimal) * Hematologic intolerance: participants with grade 3 or 4 toxicity (absolute neutrophil count \[ANC\] or platelets) while on therapy that is recurrent after dose reduction to the lowest doses of the TKI 6\. Evidence of typical BCR::ABL1 transcript \[e14a2 and/or e13a2\] at the time of screening which are amenable to standardized RQ-PCR quantification. 7\. Performance status: Karnofsky ≥ 50% for participants ≥ 16 years of age, and Lansky ≥ 50 for participants \< 16 years of age at the time of screening. Key
Exclusion Criteria:
• Known second chronic phase (CP) of CML after previous progression to Accelerated Phase (AP)/Blast Phase (BP).
• Previous treatment with a hematopoietic stem-cell transplantation.
• Patient planned to undergo allogeneic hematopoietic stem cell transplantation
• Known presence of a BCR::ABL1 mutation with known resistance to study treatment in accordance with the most recent public version of international CML clinical guidelines (e.g. NCCN CML treatment guidelines v 1.2026 and Apperley et al 2025) any time prior to study entry Other inclusion/exclusion criteria may apply.
DRUG: Asciminib single agent
Chronic Myelogenous Leukemia, Leukemia, Myelogenous, Chronic, Philadelphia Chromosome Positive
Asciminib, ABL001, Pediatric participants, Philadelphia chromosome positive chronic myeloid leukemia in chronic phase, Ph+ CML-CP, tyrosine kinase inhibitor, TKI, Molecular Response, MR, CML, Chronic phase, T3151, Ph+
Comparing Analgesic Regimen Effectiveness and Safety for Surgery for Kids Trial (CARES for Kids)
Knox Jennifer - jennifer.knox@cchmc.org
ALL
12 years to 20 years old
PHASE4
NCT06671002
Inclusion Criteria:
* No significant analgesic medication use before surgery as defined in the protocol. For this study, we define significant analgesic medication use before surgery as prescriptions reported by the child or caregiver for 6 days or more of opioid medications in the past 90 days (aligning with American Pediatric Guidelines that were published in September 2024 after the project was funded), excepting surgical team prescriptions within the week of surgery.
* Undergo one the following: tonsil removal (with or without adenoid removal), gallbladder removal (laparoscopic), and knee scope (arthroscopy).
Exclusion Criteria:
* Anticipated other surgery within 12 months
* Anticipated life expectancy of \<12 months
* Those that have legal guardians (due to special permission to enroll in trials)
* Participants with complex chronic conditions per the Pediatric Medical Complexity Algorithm (e.g., trisomy 21)
* Patients with contraindications to non-steroidal anti-inflammatory drug (NSAID) drugs in the NSAID arm, opioid drugs in the opioid arm, or acetaminophen will be excluded.
* Liver disease
* Acute psychiatric instability (defined as current uncontrolled severe depression, severe post-traumatic stress disorder (PTSD), or suicidal ideation), substance use disorder not in remission or treatment, and history of diversion of controlled substances (opioids) DRUG: NSAID, DRUG: Opioid, DRUG: Acetaminophen, DRUG: Ibuprofen
Surgery, Pain, Post Operative
Medications after surgery, NSAIDS, Acetaminophen, Opioids, Analgesics, non-narcotic, Tonsil removal, Gallbladder removal, Knee scope, Analgesics, opioid
Pompe Disease Registry Protocol
Trial Transparency email recommended (Toll free number for US & Canada) - contact-us@sanofi.com
ALL
NCT00231400
Inclusion Criteria:
All patients with a confirmed diagnosis of Pompe disease who have signed the informed consent and authorization form(s) are eligible for inclusion. Confirmed diagnosis is defined as documented GAA enzyme deficiency from blood, skin, or muscle tissue and/or documentation of 2 GAA gene mutations.
Exclusion Criteria:
There are no exclusion criteria in this Registry Glycogen Storage Disease Type II, Pompe Disease
Glycogen Storage Disease Type II (GSD-II), GSD-II, Pompe Disease, Pompe Disease (late-onset), Acid Maltase Deficiency Disease, Glycogenosis II