Allo HSCT for High Risk Hemoglobinopathies

Launched by MASONIC CANCER CENTER, UNIVERSITY OF MINNESOTA · Mar 6, 2025

Keywords

ClinConnect Summary

This clinical trial, called "Allo HSCT for High Risk Hemoglobinopathies," is studying a treatment called allogeneic hematopoietic stem cell transplantation (HSCT) for patients with serious blood disorders like Sickle Cell Disease and certain types of thalassemia. The goal is to see how effective this transplant is for people who have a high risk of complications from their condition. The trial is currently recruiting participants of all ages, including those who have a fully matched sibling donor, and it aims to help patients who rely on frequent blood transfusions.

To be eligible for this trial, participants must have specific conditions such as Sickle Cell Disease, transfusion-dependent thalassemia, or Diamond Blackfan Anemia, among others. They should be in reasonably good health, with a performance score indicating they can handle the treatment. Participants will need to agree to certain precautions, like using effective contraception during and after the study. If you or someone you know is considering joining this trial, it’s important to understand the potential risks and benefits, and to discuss them thoroughly with a healthcare provider.

Gender

ALL

Eligibility 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