Half-Matched stem cell transplant shows promise for sickle cell disease

NCT ID NCT03077542

Ongoing Disease control Sponsor: National Heart, Lung, and Blood Institute (NHLBI) Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a stem cell transplant from a half-matched relative (haploidentical donor) for adults with severe sickle cell disease. The goal is to see if a gentler conditioning regimen can reduce complications like graft rejection and graft-versus-host disease. Participants receive donor stem cells after low-dose radiation and immune-suppressing drugs, then are monitored for up to 5 years.

What this could mean

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

Active substance: haploidentical stem cell transplant
What this could lead to: If successful, this could offer a safer stem cell transplant option for people with severe sickle cell disease, potentially reducing or eliminating disease complications.
What could go wrong: This is an early-phase trial with only 57 participants, so results may not apply to everyone. Risks include graft rejection, severe graft-versus-host disease, and side effects from the conditioning drugs.

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Conditions

The condition(s) this trial relates to.

graft versus host disease sickle cell disease

As listed by the trial registrant

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

SICKLE CELL DISEASE

Contacts and locations

Locations

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.