A gentler transplant may cure sickle cell and thalassemia — can the body accept donor cells?

NCT ID NCT02105766

First seen Aug 18, 2026 · Last updated Aug 18, 2026

Summary

This trial tests a modified stem cell transplant for people with severe sickle cell disease or beta-thalassemia who are at higher risk of transplant failure. The approach uses low-dose radiation and a mix of immunosuppressive drugs to help the body accept donor stem cells without a full-intensity conditioning regimen. Participants receive donor stem cells and are monitored for a year or more to see if the donor cells produce healthy red blood cells. The goal is to achieve a durable cure with fewer side effects than traditional transplants.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
A combination of immunosuppressive drugs (alemtuzumab, sirolimus, cyclophosphamide, pentostatin), low-dose radiation, and donor stem cells (filgrastim-mobilized peripheral blood).
What this could lead to
If successful, this approach could offer a cure for sickle cell disease and beta-thalassemia, freeing patients from lifelong symptoms and treatments.
What could go wrong
This is a Phase 2 trial, so results are uncertain. Risks include graft failure, graft-versus-host disease, and side effects from immunosuppression and radiation.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • National Institutes of Health Clinical Center

    Bethesda, Maryland, 20892, United States

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