Stem Cell Transplants for Sickle Cell Disease and Thalassemia

Overseen By Ashish Gupta, MBBS, MPH

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: Masonic Cancer Center, University of Minnesota

Disqualifiers: Pregnancy, HIV, Uncontrolled infection, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 6-8 weeks

62 Participants Needed

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

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.

I have Diamond Blackfan Anemia.

I have a blood disorder that is not cancer.

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 7 days of the start of treatment

HIV Positive

Known allergy to any of the study components

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants undergo allogeneic hematopoietic stem cell transplantation (HSCT) with various regimens based on donor matching

6-8 weeks

Follow-up

Participants are monitored for safety and effectiveness, including survival and GvHD outcomes

2 years

Open-label extension (optional)

Participants may continue to be monitored for long-term outcomes beyond the initial follow-up period

Long-term

Treatment Details

Interventions

• Allogeneic Hematopoietic Stem Cell Transplantation

Trial Overview 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.

Participant Groups

Group I: Arm D Experimental Treatment 8 Interventions

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

Group II: Arm C Experimental Treatment 6 Interventions

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

Group III: Arm B Experimental Treatment 6 Interventions

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

Group IV: Arm A Experimental Treatment 5 Interventions

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

Who Is Running the Clinical Trial?

Masonic Cancer Center, University of Minnesota

Lead Sponsor

Trials

285

Recruited

15,700+