Could a new antibody make stem cell transplants a real cure for blood disorders?

NCT ID NCT05357482

First seen Nov 01, 2025 · Last updated May 13, 2026 · Updated 33 times

Summary

This study tests whether adding a new antibody drug (briquilimab, JSP191) to a standard stem cell transplant can help more people with sickle cell disease or beta-thalassemia achieve a full donor blood system, potentially curing their condition. About 40 participants aged 13 and older will receive the transplant and be followed for at least a year. The goal is to see if this approach increases the chance of long-term success without needing lifelong medications.

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Contacts and locations

Locations

  • National Institutes of Health Clinical Center

    Bethesda, Maryland, 20892, United States

Conditions

Explore the condition pages connected to this study.