Stem Cell Transplants for Sickle Cell Disease and Thalassemia

Recruiting in Palo Alto (17 mi)

Age: < 65

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Recruiting

Sponsor: Masonic Cancer Center, University of Minnesota

No Placebo Group

Prior Safety Data

Breakthrough Therapy

Trial Summary

What is the purpose of 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

This trial is for individuals with severe blood disorders like Sickle Cell Disease and Thalassemia, who haven't found relief through other treatments. Participants must be willing to use effective contraception and have a matched sibling donor for the transplant. They should be in relatively stable health (Karnofsky score ≥ 60%) but exceptions may be made after team evaluation.

Inclusion Criteria

I have Sickle Cell Disease.

I need regular blood transfusions for my Thalassemia.

I have Diamond Blackfan Anemia.

I have a blood disorder that is not cancer.

Participant Groups

The study tests a combination of drugs (like Tacrolimus, Thiotepa) and procedures (Total Body Irradiation, Stem Cell Transplant) to treat high-risk hemoglobinopathies using donor stem cells. It's an open-label phase II trial, meaning everyone gets the treatment and knows what it is.

4Treatment groups

Experimental Treatment

Group I: Arm DExperimental Treatment8 Interventions

Arm D: Haploindentical or mismatched unrelated donors (MMUD) - 0-55 years; ATG/Thiotepa/Cyclophosphamide/MESNA/Flu/TBI

Group II: Arm CExperimental Treatment6 Interventions

Arm C Fully Matched unrelated donor (MUD)- - 0-55 years; ATG/Flu/Bu

Group III: Arm BExperimental Treatment6 Interventions

Arm B Matched sib regimen - 0-55 (per physician preference for patients over 6) ATG/Flu/Bu

Group IV: Arm AExperimental Treatment5 Interventions

Arm A Matched sib regimen - Age 6 -55 (per physician preference for patients over 6) Campath/TBI