Allo HSCT for High Risk Hemoglobinopathies
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
AgeUp to 55
SponsorMasonic Cancer Center, University of Minnesota
About this trial
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.
Eligibility criteria
Qualifiers
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
Disqualifiers
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
Trial design
Treatments tested in this trial
- Alemtuzumab
- Total Body Irradiation
- Cell Infusion
- Thymoglobulin
- Fludarabine
- Busulfan
- Cyclophosphamide
- Sirolimus
- Tacrolimus
- Mycophenolate Mofetil
- Plerixafor (mozobil)
Treatment groups
62 Participants
are divided into 5 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov