Hematopoietic Cell Transplant and Gene Therapy for Non-Malignant Blood Disorders Biobank Resource (BMT CTN 2402)
Medical College of Wisconsin
Summary
A prospective, multicenter study that will establish a repository of biospecimens and clinical data from patients undergoing hematopoietic stem cell transplant (HCT) or gene therapy (GT) for treatment of non-malignant blood diseases.
Description
This is a prospective, multicenter, study that will establish a repository of biospecimens and clinical data from patients undergoing hematopoietic stem cell transplant (HCT) or gene therapy (GT) for treatment of non-malignant blood diseases. The enrollment goal is 375 participants and up to all consenting 100 related donors (approximately 100 are anticipated), accrued over 4 years. Participants will be followed until the end of study, but continued collection of specimens and clinical data may continue to allow up to 15 years of follow-up for all enrolled participants if funds are available.…
Eligibility
- Age range
- Not specified
- Sex
- All
- Healthy volunteers
- Yes
HCT/GT Inclusion: 1. Patients with a diagnosis of Aplastic Anemia (AA), hemoglobinopathies or bone marrow failure from other causes except for malignant diseases will be eligible for enrollment on this protocol: 1. AA will be defined as having peripheral blood cytopenias with a hypocellular bone marrow for age and a clinical diagnosis of aplastic anemia as determined by their treating physicians. 2. Hemoglobinopathies include sickle cell disease or thalassemia. Patients receiving potentially curative therapy with HCT or GT for hemoglobinopathies will be eligible for this study. 3. I…