Donor transplant for high risk blood disorders
Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders
This study is testing if a stem cell transplant from a matched sibling can help people with high-risk blood disorders like sickle cell disease and thalassemia feel better.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 62 (estimated) |
| Ages | N/A to 55 Years |
| Sex | All |
| Sponsor | Masonic Cancer Center, University of Minnesota Academic / other |
| Locations | 1 site (Minneapolis, Minnesota) |
| Trial ID | NCT06872333 on ClinicalTrials.gov |
What this trial studies
This phase II trial investigates the use of allogeneic hematopoietic stem cell transplantation (HSCT) for patients with high risk hemoglobinopathies, including sickle cell disease and thalassemia. The study involves a single center and employs an open-label design, utilizing various interventions such as Alemtuzumab, Total Body Irradiation, and Fludarabine. Eligible participants must have a fully matched sibling donor and meet specific health criteria. The trial aims to assess the effectiveness of this transplant approach in improving patient outcomes.
Who should consider this trial
Good fit: Ideal candidates include individuals with sickle cell disease or transfusion-dependent thalassemia who have a fully matched sibling donor.
Not a fit: Patients who are pregnant, breastfeeding, or those who do not have a matched sibling donor may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve the quality of life and health outcomes for patients with severe blood disorders.
How similar studies have performed: Other studies have shown promise with similar HSCT approaches for hemoglobinopathies, indicating potential for success in this trial.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * 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 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 * 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 this trial is running
Minneapolis, Minnesota
- Masonic Cancer Center — Minneapolis, Minnesota, United States (Recruiting)
Study contacts
- Study coordinator: Ashish Gupta, MBBS, MPH
- Email: gupta461@umn.edu
- Phone: 612-626-2961
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.