Allo HSCT for High Risk Hemoglobinopathies
About this 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.
- Condition
- Graft Failure, Sickle Cell Disease, Hemoglobinopathies
- Tested
- Alemtuzumab, Total Body Irradiation, Cell Infusion, Thymoglobulin, Fludarabine, Busulfan, Cyclophosphamide, Sirolimus, T...
- Sponsor
- Masonic Cancer Center, University of Minnesota
Who can join
- Age
- Up to 55 years
- Sex
- All sexes
- Healthy volunteers
- Accepted
Inclusion 9
- Maximum age: 55 years
- Study condition: Graft Failure, Sickle Cell Disease, Hemoglobinopathies
- 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 6
- 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
Where
1 site, 1 recruiting
Masonic Cancer Center
Minneapolis, Minnesota, United States
Contact
-
Ashish Gupta, MBBS, MPH
612-626-2961 gupta461@umn.edu
Potential match only. Final eligibility is determined by the study team.