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Allo HSCT for High Risk Hemoglobinopathies
Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders
Who can join
Up to age 55 · All sexes · Healthy volunteers welcome
Full eligibility criteria
Inclusion Criteria: * Sickle Cell Disease (SCD) * SCD Patients with a fully matched sibling donor (MSD) irrespective of the frequency or severity of symptoms MSD transplant can be considered. Parents/patient must be counseled as to the risks and benefits and provide their voluntary informed consent * Transfusion Dependent Alpha- or Beta- Thalassemia * Diamond Blackfan Anemia * Other Non-Malignant Hematologic Disorders * Karnofsky ≥ 60%, Lansky play score ≥ 60. Patients with lower performance score can be considered based on study team's evaluation. * Sexually active persons of childbearing potential or persons with partners of childbearing potential must agree to use a highly effective form of contraception during study treatment and for at least 4 months after the transplant. Exclusion Criteria: * Pregnant, breastfeeding or intending to become pregnant during the study. Persons of childbearing potential must have a negative pregnancy test (serum or urine) within 30 days of the start of treatment * HIV infection with a detectable viral load. All HIV+ patients must be evaluated by infectious disease (ID) and an HIV management plan established prior to transplantation. * Active, uncontrolled infection - infection that is stable or improving after 1 week of appropriate therapy (4 weeks for presumed or documented fungal infections) will be permitted * Known allergy to any of the study components * Psychiatric illness/social situations that, in the judgement of the enrolling Investigator, would limit compliance with study requirements * Other illness or a medical issue that, in the judgement of the enrolling Investigator, would exclude the patient from participating in this study
About the study
A single center, open label, interventional, phase II trial for donor transplant for high risk hemoglobinopathies and other red cell transfusion dependent disorders utilizing allogeneic hematopoietic stem cell transplantation (HSCT) regimens.
What is being tested
- Alemtuzumab (drug)
- Total Body Irradiation (radiation)
- Cell Infusion (biological)
- Thymoglobulin (drug)
- Fludarabine (drug)
- Busulfan (drug)
Sponsor: Masonic Cancer Center, University of Minnesota · Participants: 62 · Started: Nov 19, 2024
Contact the study team
- Ashish Gupta, MBBS, MPH · Phone: 612-626-2961
Official record on ClinicalTrials.gov — NCT06872333
Locations in the U.S.
| Minnesota | Masonic Cancer Center, Minneapolis |
Conditions
From ClinicalTrials.gov, data retrieved Sep 29, 2026. Each study sets its own eligibility; the study team decides who can join.