MT2014-10C: Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders

2014OC034

This is a study to collect the outcomes of stem cell transplantation for patients with hematologic diseases other than cancer.

2014-09-02

2025-01-01

2025-08-01

Recruiting

25

Inclusion Criteria: * Diagnosis of Sickle Cell Disease, Thalassemia, Diamond Blackfan Anemia or other non-malignant hematologic disorders for which a stem cell transplant is indicated * Acceptable stem cell source identified * Performance status of ≥ 70% (Karnofsky),or ≥ 70 (Lansky play score) * Creatinine \<2.0 mg/dl for adults or glomerular filtration rate \> 50 ml/min for children * Bilirubin, Aspartate Aminotransferase, Alkaline phosphatase \<5 times the upper limit of institutional normal * Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction \> 40% Exclusion Criteria: * active, uncontrolled infection * pregnant or breastfeeding * HIV positive

{'studyType': 'INTERVENTIONAL', 'phases': ['NA'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 25, 'type': 'ESTIMATED'}}

2024-02-06