Actively Recruiting
Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders
Led by Masonic Cancer Center, University of Minnesota · Updated on 2026-06-04
62
Participants Needed
1
Research Sites
104 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating allogeneic hematopoietic stem cell transplantation (HSCT) regimens for patients with high-risk hemoglobinopathies and other red cell transfusion-dependent disorders in this phase II trial. The study aims to assess different transplant approaches and their effects on outcomes such as graft versus host disease (GvHD) and survival. It is an open-label trial conducted at a single center, focusing on conditions like sickle cell disease, thalassemia, and Diamond Blackfan anemia. Participants will receive one of several transplantation regimens depending on donor type and age. These include matched sibling donor regimens using Alemtuzumab (Campath) and total body irradiation (TBI), or regimens with Thymoglobulin (ATG), Fludarabine, Busulfan, Cyclophosphamide, and other drugs tailored to donor match type. Cells will be infused on day 0 following conditioning treatments. Immunosuppressive drugs such as sirolimus or tacrolimus and mycophenolate mofetil are given to prevent rejection and GvHD, with dosing schedules varying by treatment arm. During the study, participants will be closely monitored for graft success and complications, including the incidence of GvHD over one year and survival outcomes at one and two years. Assessments include clinical evaluations, laboratory tests, and drug monitoring. The trial will also track failure-free survival and chronic GvHD status up to two years. Participants may be followed for up to eight years, with safety and efficacy evaluations guiding ongoing care.
CONDITIONS
Brief Title
Allo HSCT for High Risk Hemoglobinopathies
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of sickle cell disease (SCD) or transfusion-dependent alpha- or beta-thalassemia
- Diagnosis of Diamond Blackfan anemia or other non-malignant hematologic disorders
- SCD patients with a fully matched sibling donor can be considered regardless of symptom severity
- Karnofsky or Lansky performance score of 60% or higher; lower scores may be considered by study team
- Sexually active persons of childbearing potential or partners must agree to use effective contraception during treatment and for at least 4 months post-transplant
You will not qualify if you...
- Pregnant, breastfeeding, or planning pregnancy during the study
- Positive HIV infection with detectable viral load without established management plan
- Active uncontrolled infection; stable or improving infections after appropriate therapy may be allowed
- Known allergy to any study components
- Psychiatric or social conditions that may limit compliance with study requirements
- Other illnesses or medical issues judged by investigators to exclude participation
AI-Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Approximately 8 days (Day -8 to Day -1)
Participants receive preparatory drug treatments including Alemtuzumab, Thymoglobulin, Fludarabine, Busulfan, and Cyclophosphamide prior to stem cell infusion.
Daily visits for approximately 8 days
Duration - Day 0 to Day +30 or longer depending on arm
Participants undergo cell infusion (stem cell transplant) on Day 0 along with radiation therapy and start post-transplant medications according to assigned treatment arms.
1 infusion visit plus daily visits as needed during the first month
Duration - Up to 1 year post transplant depending on treatment arm
Participants receive ongoing immunosuppressive drugs such as Sirolimus, Tacrolimus, and Mycophenolate Mofetil to prevent graft versus host disease and support recovery.
Regular follow-up visits over the first year
Trial Site Locations
Total: 1 location
1
Masonic Cancer Center
Minneapolis, Minnesota, United States, 55455
Actively Recruiting
Research Team
A
Ashish Gupta, MBBS, MPH
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
OTHER
Number of Arms
5
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