StudyFinder
Search Results Within Category "Blood Disorders"
39 Study Matches
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
513-636-2799 - cancer@cchmc.org
ALL
NCT01351545
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
Trial of Scheduled Versus Treatment Administration of Donor-Derived Viral Specific T-cells for Viral Infections After Stem Cell Transplant
Celeste Dourson - celeste.dourson@cchmc.org
ALL
PHASE2
NCT04230356
SCHEDULED ARM:
Inclusion Criteria:
* Recipient must be at least 21 days after stem cell infusion
* Clinical status must allow tapering of any steroids to \< 0.5mg/kg prednisone or other steroid equivalent
* No critical illness making VST infusion hazardous
Exclusion Criteria:
* Active acute GVHD grades II-IV.
* Uncontrolled relapse of malignancy.
* Infusion of ATG or alemtuzumab within 2 weeks prior to VST infusion. Alemtuzumab levels will be collected in the second week following stem cell infusion in patients who received alemtuzumab as part of their conditioning regimen. The level must be less than or equal to 0.15 prior to infusion of VSTs. In patients with level greater than 0.15, alemtuzumab levels can be checked serially until a level ≤ 0.15 is obtained. They would become eligible for scheduled VST infusion at that point.
TREATMENT ARM
Inclusion Criteria:
* Blood adenovirus PCR ≥1,000
* Blood CMV PCR ≥ 500
* Blood EBV PCR ≥ 9,000
* Plasma BKV PCR \>1,000
* Evidence of invasive adenovirus infection. Adenovirus infection will be defined as the presence of adenoviral positivity as detected by PCR or culture from one site such as stool or blood or urine or nasopharynx. Adenovirus disease will be defined as the presence of adenoviral positivity as detected by culture or PCR from more than 2 sites such as stool or blood or urine or nasopharynx.
* Evidence of invasive CMV infection, defined as pneumonitis, retinitis, colitis, hepatitis
* Evidence of EBV-associated lymphoproliferation (EBV-LPD) defined as proven EBV-LPD by biopsy or probable EBV-LPD defined as an elevated EBV DNA level in the blood associated with clinical symptoms (adenopathy or fever or masses on imaging) but without biopsy confirmation.
* Evidence of symptomatic BK virus infection, defined as hemorrhagic cystitis or BK nephropathy.
* No active acute GVHD grades II-IV
* No uncontrolled relapse of malignancy
* No infusion of ATG or alemtuzumab within 2 weeks of VST infusion.
* Clinical status must allow tapering of any steroids to \< 0.5mg/kg prednisone or other steroid equivalent BIOLOGICAL: Viral Specific T-cells (VSTs) Scheduled, BIOLOGICAL: Viral Specific T-cells (VSTs) Treatment
Allogeneic Stell Cell Transplant, Viral Infection
A Socio-ecological Approach for Improving Self-management in Adolescents With SCD (SC-Thrive)
Ama Wilson - ama.wilson@cchmc.org
ALL
13 years to 21 years old
NA
NCT06290401
Inclusion Criteria:
* Patient of a participating SCD Clinic
* Confirmed diagnosis of SCD
* 13-21 years of age
Exclusion Criteria:
* Another chronic disease (which would complicate measurement of patient activation)
* Non-English-speaking
* Cognitive or psychiatric disorder that the physician or study therapists believe would impair study participation. BEHAVIORAL: SCThrive, OTHER: SCHealthED
Anemia, Sickle Cell
adolescent and young adult (AYA), self-management, behavioral intervention, sickle cell disease
Study of Oral Administration of LP-118 in Patients With Relapsed or Refractory CLL, SLL, MDS, MDS/MPN, AML, CMML-2, MPN-BP, ALL, MF, NHL, RT, MM or T-PLL.
- cancer@cchmc.org
ALL
13 years and over
PHASE1
NCT04771572
Inclusion Criteria:
• Male or female subjects, ≥ 18 years of age at the time of Screening with the following exception as outlined below: -For T cell and B cell ALL subjects with age between 13 - 18 years, their body weight shall be ≥ 40 kg.
• Eligible subject must have an advanced hematologic malignancy including: Group 1: Group 1a * Relapsed or refractory low risk tumor lysis CLL/SLL subjects (ALC \< 25 x 109 cells/L and all lymph nodes \< 5 cm) who have received at least two prior therapies. Subjects may also have slowly progressed on irreversible BTK inhibitors while on treatment with these agents. * For CLL/SLL subjects who come off BCR antagonist treatment (BTK inhibitors, P13K inhibitors, etc.) allows washout for 2 days as these subjects, progress quickly after treatment discontinuation and then remain eligible (steroids may be given during these two days to allow disease control). Group 1b * Morphologically confirmed diagnosis of MF in accordance with the WHO 2016 revised criteria, that is relapsed, intolerant, and/or refractory and that, in the opinion of the Investigator, subjects who have no available therapies known to provide clinical benefits; * Morphologically confirmed diagnosis of MDS/MPN, excluding juvenile myelomonocytic leukemia (JMML), in accordance with WHO 2016 revised criteria, that is relapsed and/or refractory and that, in the opinion of the Investigator, subjects who have no available therapies known to provide clinical benefits; * Chronic myelomonocytic leukemia (CMML) with \<9% blasts; * Or atypical chronic myeloid leukemia (aCML) with Hgb \> 10g/dL, WBC count \< 50 x 109 cells/L, \<10% immature circulating cells; * Or MDS/MPN with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T) with Hgb \> 10g/dL; * Or myelodysplastic/myeloproliferative neoplasm, unclassifiable (MDS/MPN-UC) * CMML-2 with 10-19% blasts as defined by WHO 2016 revised criteria that is relapsed and/or refractory to prior HMA therapy; * Relapsed and/or refractory MPN-BP as defined by WHO 2016 revised criteria that is transformed MPN with \>20% myeloid blasts in the peripheral blood or bone marrow, in the opinion of the Investigator, subjects who have no available therapies known to provide clinical benefits; * MDS subjects with refractory anemia with excess blasts (MDS-EB; subtype MDS-EB-1 or MDS-EB-2) as defined by WHO 2016 revised criteria and/or MDS with high- or very high-risk (risk score \> 4.5) per the Revised International Prognostic Scoring System (IPSS-R, refer to Appendix 11; Section 15.13) who have no available therapies known to provide clinical benefit; * Relapsed or refractory AML subjects (including de novo AML, secondary AML evolving from MDS or MPN or other antecedent hematologic disorder, and therapy-related AML) as defined by WHO 2016 revised criteria, subjects who have no available therapies known to provide clinical benefits; subjects with prior BCL-2 inhibitor therapy are permitted. WBC needs to be ≤ 25 × 109 cells/L at the time of initiating investigational therapy (hydroxyurea is allowed to control WBC prior to and during therapy). Group 1c * Relapsed or refractory low risk tumor lysis NHL (NHL histologies \[MZL, FL, WM, DLBCL, ATLL, PTCL, AITL, ALCL, MCL\] are to be included per the 2016 World Health Organization \[WHO\] criteria) subjects, must have histologically documented diagnosis of a non-Hodgkin lymphoma as defined in the WHO classification scheme. Subjects have received at least 2 prior therapies and have no available therapies known to provide clinical benefit; For subjects with indolent NHL (Grade 1\~3a FL, MZL) who have received two prior systemic therapies and have relapsed or progressed according to 2014 Lugano; * Low risk tumor lysis transformed follicular, MZL, WM (to large cell or aggressive lymphoma) subjects who must have received at least one prior systemic therapy for the transformed lymphoma (unless combination chemotherapy is not appropriate); * Low risk tumor lysis Richter transformation (RT): previously treated CLL and biopsy-proven Richter transformation with DLBCL histology after receiving at least one regimen for RT; * Relapsed or refractory multiple myeloma (MM) subjects who have received a proteasome inhibitor (PI), an immunomodulatory drug (IMiD), and an anti-CD38 and have no treatment options available known to provide clinical benefit; * Low risk tumor lysis T-cell prolymphocytic leukemia (T-PLL) subjects who have received one therapy for this and are relapsed or refractory; Group 1d * Relapsed or refractory ALL with dexamethasone run-in \[5 days, dexamethasone 10mg/m2 (divided BID)\]; * Or r/r ALL in remission but with detectable MRD (MRD +) by any detection method per institution standard of practice; * IT chemo (per institutional SOC) is permitted prior to LP-118 C1D1 dosing, and then concomitantly on treatment if in best interest of the subject; * Relapsed or refractory ALL subjects with B cell phenotype who have received at least two prior therapeutic regimens (such as multi-agent chemotherapy and/or tyrosine kinase inhibitors including bosutinib, dasatinib, imatinib, nilotinib or ponatinib) and failed, or are currently ineligible/intolerant for CD19-based target therapy (e.g. Blinatumomab); Relapsed or refractory ALL subjects with T cell phenotype who have received at least one prior therapy and failed. * Relapsed or refractory ALL subjects with age between 13 - 18 years and have body weight ≥ 40kg, ALL subjects with B cell phenotype who have received at least two prior therapeutic regimens (such as multi-agent chemotherapy and/or tyrosine kinase inhibitors including bosutinib, dasatinib, imatinib, nilotinib or ponatinib) and progressed, or are currently ineligible/intolerant for CD19-based target therapy (e.g. Blinatumomab); Relapsed or refractory ALL subjects with T cell phenotype who have received at least one prior therapy and progressed. Group 2 * Relapsed or refractory intermediate and high risk tumor lysis CLL/SLL subjects who have received at least two prior therapies; * Relapsed or refractory intermediate and high risk tumor lysis NHL (NHL histologies \[MZL, FL, WM, DLBCL, ATLL, PTCL, AITL, ALCL, MCL\] are to be included per the 2016 World Health Organization \[WHO\] criteria) subjects, must have histologically documented diagnosis of a non-Hodgkin lymphoma as defined in the WHO classification scheme. Subjects have received at least 2 prior therapies and have no available therapies known to provide clinical benefit; For subjects with indolent NHL (Grade 1\~3a FL, MZL) who have received two prior systemic therapies and have relapsed or progressed according to 2014 Lugano; * Intermediate and high risk tumor lysis transformed follicular, MZL, WM (to large cell or aggressive lymphoma) subjects who must have received at least one prior systemic therapy for the transformed lymphoma (unless combination chemotherapy is not appropriate); * Intermediate and high risk tumor lysis Richter transformation (RT): previously treated CLL and biopsy-proven Richter transformation with DLBCL histology after receiving at least one regimen for RT; * Intermediate and high risk tumor lysis T-cell prolymphocytic leukemia (T-PLL) subjects who have received one therapy for this and are relapsed or refractory;
• For Group 1d ALL subjects only, white blood cell (WBC) count ≤ 25 × 109 cells/L at the time of enrollment (glucocorticoids or hydroxyurea is permitted to control WBC count prior to and during therapy).
• Eastern Cooperative Oncology Group (ECOG) performance score ≤ 2.
• Adequate cardiac function defined as shortening fraction of ≥ 40% by 2D echocardiogram without Doppler.
• Subject must have adequate bone marrow (independent of growth factor support), coagulation, renal, and hepatic function, per laboratory reference ranges at Screening as follows: Bone marrow criteria: * Group 1 (r/r low risk tumor lysis CLL/SLL (ALC \< 25 x 109 cells/L and all lymph nodes \< 5 cm), NHL, RT, MM, T-PLL): * Absolute Neutrophil Count (ANC) ≥ 1 x 109/L (An exception is for subjects with an ANC\<1 x 109/L and bone marrow heavily infiltrated with underlying disease) * Platelets ≥ 50 x 109/L on day of screening (entry platelet count must be independent of transfusion with 14 days of screening); * Hemostasis criteria: Activated partial thromboplastin time (APPT) and prothrombin time (PT) ≤ 1.5 × the upper limit of normal (ULN); * Renal function criteria: Serum creatinine ≤ ULN (per local institution reference range) or Calculated creatinine clearance (Cr Cl) ≥ 60 mL/min using 24-hour CrCl OR by Cockcroft-Gault formula using actual body weight. * Hepatic function criteria: Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3.0 × ULN; bilirubin ≤ 1.5 × ULN (except subjects with Gilbert's Syndrome, who may have a bilirubin \> 1.5 × ULN, per discussion between the Investigator and the Medical Monitor).
• Females of childbearing potential (i.e., non-postmenopausal for at least 2 years or surgically sterile) and non-sterile males must practice at least 1 of the following methods of birth control with their partner(s) throughout the study and for 90 days after discontinuing study drug: * Total abstinence from sexual intercourse as the preferred lifestyle of the subject; periodic abstinence is not acceptable; * Surgically sterile partner(s) by vasectomy, bilateral orchiectomy, bilateral tubal ligation, bilateral oophorectomy or hysterectomy; * Intrauterine device; * Hormonal contraceptives (oral, parenteral, vaginal ring or transdermal) associated with inhibition of ovulation initiated for at least 1 month prior to study drug administration.
• Females of childbearing potential must have a negative pregnancy result as follows: * At Screening on a serum sample obtained within 7 days prior to the first study drug administration, and * Prior to dosing on a urine or serum sample obtained on the first day of study drug administration if ithas been \> 7 days since obtaining the serum pregnancy test results in Screening. * If a urine pregnancy test at any timepoint during the study is positive or indeterminate, a serum pregnancy test will be performed for confirmation
• Male subjects must refrain from sperm donation, from initial study drug administration until 90 days after the last dose of study drug.
• Subject must be able to understand and voluntarily sign and date an informed consent form (ICF), approved by an IRB, prior to any protocol-related procedures.
Exclusion Criteria:
A subject will not be eligible for study participation if he/she meets any of the following criteria.
• Subjects who have undergone autologous/allogeneic hematopoietic stem cell transplantation (HSCT) therapy within 60 days of the first dose of LP-118, or subjects on immunosuppressive therapy post-HSCT at the time of Screening, or currently with clinically significant graft-versus-host disease (GVHD) as per treating physician (Subjects in relapse after allogeneic transplantation must be off treatment with systemic immunosuppressive agents for at least 4 weeks. The use of topical steroids and/or up to 20 mg/day prednisone or equivalent systemic steroids for ongoing GVHD is permitted.
• Subject has a history of other malignancies within past 12 months that are active and could result in competing risks. These cases shall be discussed with the Medical Monitor with the exception below. * Subject with breast cancer or prostate cancer on endocrine therapy with stable disease; * Continuation of maintenance therapy in patients with adequately treated malignancy * Adequately treated in situ carcinoma of the cervix uteri; * Basal cell carcinoma of the skin or localized squamous cell carcinoma of the skin; * Previous malignancy confined and surgically resected (or treated with other modalities) with curative intent. * Cancer with expected survival of 2 years or more or that will not confound evaluation of LP-118 treatment
• Subject has received any of the following therapies within 14 days or 5 half-lives (whichever is shorter) prior to the first dose of LP-118, or has not recovered to ≤ Grade 2 clinically significant AEs of the previous therapy (excluding neuropathy): * Any anti-neoplastic therapy including chemotherapy, hormonal therapy, radiotherapy, biologic or immunotherapy, targeted small molecule agents, etc. (corticosteroid therapy \< 20 mg/day prednisone equivalent according to institutional guidelines to treat disease associated symptoms are permitted); * For MF subjects who come off JAK2 antagonists, allow washout for 2 days as these subject's progress quickly after treatment discontinuation and remain eligible (steroids may be given during these two days to allow disease control). * Subjects in need of immediate cytoreduction should be excluded. * Any investigational therapy. * Live vaccines
• Subject has received the following medications, therapies, or natural products within 7 days prior to the first dose of LP-118: * Cytochrome P450, family 3, subfamily A (CYP3A) strong inhibitors (itraconazole, etc.), or substrates (Appendix 19); * Subject has received strong Cytochrome P450, family 3, subfamily A (CYP3A) inducers within 14 days prior to the first dose of LP-118 (Appendix 19); * Grapefruit, grapefruit products, Seville oranges (including marmalade containing Seville oranges) or Star fruit; * There is a 28-day washout period required for subjects who have had prior CAR-T treatment if there is no evidence of cytokine release syndrome (CRS) or other adverse events related to the CAR-T treatment, per discussion with the Medical Monitor.
• Subject has a significant history of renal, neurologic, psychiatric, pulmonary, endocrinologic, metabolic, immunologic, cardiovascular, or hepatic disease that, in the opinion of the Investigator, would adversely affect his/her participation in this study. Any other medical or social condition deemed by the investigator to be likely to interfere with a subject's ability to participate in the study, place the subject at unacceptable risk or interfere with the interpretation of the results. For subjects who have required surgical intervention for any above diseases within the past 6 months, a discussion with the Investigator and the Medical Monitor is needed.
• Subject has baseline prolongation of the heart rate-corrected QT (QTcF) interval ≥ 480 ms (calculated per Fridericia's formula \[QTcF = QT/RR (1/3)\])), a cardiovascular disability status of New York Heart Association Class ≥ 2 or associated other significant screening ECG or ultrasonic cardiogram abnormalities, per Investigator's judgement. For any subject with underlying RBBB or LBBB, cardiology review is needed to correct QTcF calculation using Sponsor recommended formula.
• Subject has significant a history of congenital long QT syndrome or Torsades de Pointes (TdP), uncontrolled or symptomatic arrhythmias, congestive heart failure, myocardial infarction, stroke, or intracranial hemorrhage within 6 months prior to the first dose of LP-118.
• Subject exhibits evidence of other clinically significant uncontrolled condition(s) including, but not limited to: * Uncontrolled active systemic infection (bacterial, fungal, viral); * Known poorly controlled human immunodeficiency virus (HIV) or active hepatitis B or C infection (active hepatitis B defined as HbsAg positive, or HbcAb positive with detectable HBV DNA load; active hepatitis C defined as HCV antibody positive with HCV RNA positive); * Unexplained fever \> 38.5°C within 7 days prior to the first dose of study drug administration (at the discretion of the Investigator if the fever is considered attributed to the subject's malignancy or an explained infection may be enrolled).
• A female subject is pregnant or breast-feeding.
• Subject incapacity to swallow oral medications, with any malabsorption condition, known dysphagia, short-gut syndrome, gastroparesis, or other conditions that, in the opinion of the Investigator, may limit the ingestion or gastrointestinal absorption, distribution, metabolism and excretion of drugs administered orally.
• Subjects with known and active central nervous system (CNS) involvement at Screening.
• Subjects with known hypersensitivity to any of the components of LP-118 (see Investigators Brochure for a list of components).
• Subjects who are taking QT-prolonging drugs that are known to cause Torsades de Pointes (TdP) (See Appendix 17 for the list of medications that are associated with TdP). In the event a prohibited medication might cause TdP, the PI must first determine if the risk to benefit is in favor of the subject and then discuss with the Medical Monitor about that particular medication on a case-by-case basis.
• Major surgery within 14 days prior to the first dose of study drug.
DRUG: LP-118
Non Hodgkin Lymphoma, Richter Transformation, Multiple Myeloma, T-cell-prolymphocytic Leukemia, Acute Myeloid Leukemia, Acute Lymphocytic Leukemia, Myeodysplastic Syndrome, Myelodysplastic/Myeloproliferative Neoplasm, Myelofibrosis, Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma, Chronic Myelomonocytic Leukemia-2, Myelodysplastic Neoplasm in Blast Phase
Hematological Malignancies, Relapsed, Refractory
Verifying Antibodies After Live Immunization Delivery (VALID): A Study of Measles Vaccine Immunogenicity in Children With Sickle Cell Disease (VALID)
MacKenzie Tasset - macKenzie.tasset@cchmc.org
ALL
6 months to 6 years old
NCT07356050
Inclusion Criteria:
• Participants with confirmed Sickle Cell Disease.
• Participants 6 months and 6 years of age and due for measles vaccination within 3 months per national guidelines.
• Willing and able to provide informed consent
• Ability to comply with study related evaluations and follow-up visits.
Exclusion Criteria:
1\. Known primary immunodeficiency syndrome, cancer, or acquired immunodeficiency syndrome (AIDS) that would preclude vaccination with live virus vaccines. Sickle Cell Disease, Measles Vaccination, Sickle Cell Anemia
Measles Vaccine Immunogenicity
A Phase 1b, Open-Label Study of DISC-3405 in Participants With Sickle Cell Disease (SCD)
Kara McClure - kara.mcClure@cchmc.org
ALL
18 years and over
PHASE1
NCT07187973
Inclusion Criteria:
• Aged 18 years or older at the time of signing the informed consent form (ICF).
• Male or female study participants with SCD HbSC or HbSS.
• Participants who have been diagnosed with any of the following SCD-related complications: between 1-10 episodes of VOC in the past 12 months, any history of sickle cell related retinopathy, silent cerebral infarct, avascular necrosis, sensorineural hearing loss; or at least 1 episode of priapism, hepatic sequestration, splenic sequestration, or splenic infarct within the last 12 months as assessed locally.
• Hgb ≥7.0 g/dL during Screening. The first 2 participants must have an Hgb ≥9 g/dL.
• Normal alpha globin gene screen.
• Absolute reticulocyte count or % reticulocyte count \>1.5 × upper limit of normal (ULN) during Screening.
• TSAT ≥15% at Screening.
• Ferritin ≥50 ng/mL for HbSC or ≥100 ng/mL for HbSS (ferritin must be \<1000 ng/mL at Screening).
• For participants taking hydroxyurea, L-glutamine, or crizanlizumab, stable dose for at least 2 months prior to Screening and with no anticipated need for dose adjustments during the study.
• If male, not vasectomized for at least 6 months, with female sexual partner(s) of childbearing potential, agrees he and partner will use double methods of the following highly effective methods of birth control (described below) from the first dose of randomized study drug until 120 days after the last administration of study drug and must not donate sperm during their study participation:
• Stable hormonal contraceptive (≥3 months; female partner) in conjunction with a barrier method (eg, condom \[male or female\] or diaphragm).
• Intrauterine device, in place for at least 3 months (female partner) in conjunction with a barrier method (eg, condom \[male or female\] or diaphragm).
• Surgically sterile by hysterectomy, bilateral oophorectomy, or bilateral tubal ligation (female partner) in conjunction with a barrier method (eg, condom \[male or female\] or diaphragm).
• If female, then EITHER postmenopausal, defined as at least 12 months natural, spontaneous amenorrhea, 6 months of spontaneous amenorrhea with serum follicle-stimulating hormone (FSH) \>40 mIU/mL at Screening, or at least 6 weeks following surgical menopause (bilateral oophorectomy with or without hysterectomy); surgically sterile, OR agree to use 1 of the following highly effective methods of birth control on Day 1 (or earlier) and for at least 120 days after the last administration of study drug:
• Stable hormonal contraceptive (≥3 months) in conjunction with a barrier method (eg, condom \[male or female\] or diaphragm).
• Intrauterine device, in place for at least 3 months in conjunction with a barrier method (eg, condom \[male or female\] or diaphragm).
• Tubal ligation or single male partner with vasectomy in conjunction with a barrier method (eg, condom \[male or female\] or diaphragm).
• Negative pregnancy test (females of childbearing potential) prior to dosing.
• Able to understand the study aims, procedures, and requirements, and provide written informed consent.
• Able to comply with all study procedures.
Exclusion Criteria:
• Participants who are receiving regularly scheduled blood (RBC) transfusion therapy or phlebotomy or have received RBC transfusion or phlebotomy within 60 days of Screening.
• Hospitalized for VOC or other sickle cell related complication within 14 days of Screening.
• Participants with clinically significant bacterial, fungal, parasitic, or viral infection.
• Active HIV, hepatitis B, or C. A positive hepatitis or HIV result should be discussed between the Investigator and Sponsor prior to enrollment.
• Significant renal dysfunction, evidenced by estimated glomerular filtration rate of \<60 mL/min/1.73 m2 at the Screening visit, as assessed locally.
• Hepatic dysfunction characterized by alanine aminotransferase (ALT) \>2.5 × ULN.
• Any episode of ACS in the last 6 months.
• Prior or planned hematopoietic stem cell transplant or gene therapy.
• History of unstable or deteriorating cardiac or pulmonary disease within 6 months prior to Screening.
• History of invasive malignancies within the last 5 years, except localized cured prostate cancer and cervical cancer, or other malignancies deemed acceptable by the Sponsor.
• Major surgery within 8 weeks before Screening or incomplete recovery from any previous surgery.
• A history or known allergic reaction to any IP excipients or history of anaphylaxis to any food or drug.
• History of alcohol dependence or excessive alcohol consumption, as assessed by the Investigator.
• Other medical or psychiatric condition or laboratory finding not specifically noted above that, in the judgment of the Investigator or Sponsor, would put the participant at an unacceptable risk or otherwise preclude the participant from participating in the study.
• Condition or concomitant medication that would confound the ability to interpret clinical, clinical laboratory, including a major psychiatric condition that has had an exacerbation or required hospitalization in the last 6 months.
• If female, pregnant or breastfeeding.
• Participation in any other clinical protocol or investigational study that involves administration of experimental therapy and/or therapeutic devices within 30 days of Screening.
• Participants with a history of transient ischemic attack or stroke may be considered in consultation with Sponsor.
DRUG: DISC-3405
Sickle Cell Disease
Effectiveness of Nontraditional Hydroxyurea Algorithms: Novel and Clinical Evaluations (ENHANCE) (ENHANCE)
Charles T. Quinn, M.D., M.S. - charles.quinn@cchmc.org
ALL
6 month(s) and over
PHASE4
NCT07177300
Inclusion Criteria:
* Diagnosis of sickle cell anemia (HbSS) or sickle-β0-thalassemia (HbSβ0)
* Age 6 months at the time of enrollment
* Clinical decision by patient, family, and healthcare provider to initiate hydroxyurea therapy
Exclusion Criteria:
* Current treatment with regularly scheduled blood transfusions
* Sickle-hemoglobin C disease (HbSC), sickle-β+-thalassemia (HbSβ+) DRUG: PK-optimized oral hydroxyurea at MTD until 15 years of age.
Sickle Cell Anemia (HbSS), Sickle-β0-thalassemia (HbSβ0)
sickle cell anemia, SCD, sickle cell, hydroxyurea
A Study About How Blood Cell Growth Patterns Relate to Heart Health After Treatment for Hodgkin Lymphoma
- cancer@cchmc.org
ALL
7 years and over
NCT05705531
Inclusion Criteria:
* Patient must be \>= 7 years of age at the time of enrollment (age to perform an MRI without sedation).
* History of pathologically confirmed classical Hodgkin Lymphoma (cHL) initially diagnosed when the patient was \>= 2 and \< 22 years of age.
* As part of frontline therapy for cHL, the patient must have received a cumulative doxorubicin equivalent anthracycline dose of ≥ 200 mg/m\^2 as estimated in doxorubicin isotoxic equivalents dose conversion calculation.
* Note: History of COG therapeutic trial participation is not required. Institutional records (e.g., clinic note, treatment summary, chemotherapy roadmap) can be used as reference documentation of receipt of anthracycline dose.
* All systemic cancer treatment must have been completed ≥ 2 years prior to study enrollment.
* Not known to have had a primary event (relapse/second malignancy/death).
* Note: Subjects treated at another institution are eligible if they are now being followed at the current COG institution, if the study procedures can be performed and the data accessible by a COG institution where the study is open.
* Patient must have access to cardiac MRI at the enrolling institution and must be able to complete cardiac MRI without sedation.
Exclusion Criteria:
* Medical contraindication to undergoing a non-contrast cardiac MRI.
* Patients with nodular lymphocyte-predominant HL.
* Received cancer therapy in addition to that for primary Hodgkin Disease (e.g., for disease progression or recurrence, or subsequent malignant neoplasm).
* History of CTCAE grade 3 or higher cardiovascular disease or condition known to exist prior to the patient's initial diagnosis of cHL.
* Note: exceptions are made for congenital conditions considered fully resolved by surgery and chronic conditions such as hypertension or hypercholesterolemia that are managed with medical intervention.
* History of an immunodeficiency that existed prior to cHL diagnosis, such as primary immunodeficiency syndromes, organ transplant recipients and conditions requiring systemic immunosuppressive agents. PROCEDURE: Archive Sample Retrieval, PROCEDURE: Biospecimen Collection, OTHER: Electronic Health Record Review, PROCEDURE: Magnetic Resonance Imaging, OTHER: Survey Administration
Cardiovascular Disorder, Classic Hodgkin Lymphoma, Clonal Hematopoiesis
TRANSPIRE: Lung Injury in a Longitudinal Cohort of Pediatric HSCT Patients
Sara Loveless - sara.loveless@cchmc.org
ALL
Up to 24 years old
NCT04098445
Inclusion Criteria:
* Subjects ≤ 24 years of age undergoing allogeneic or autologous HSCT.
Exclusion Criteria:
* Subjects over 24 years of age. Hematopoietic Stem Cell Transplant (HSCT), Diffuse Alveolar Hemorrhage, Thrombotic Microangiopathies, Interstitial Pneumonitis, Bronchiolitis Obliterans