Second chance: stem cell transplant trial aims to rescue failing sickle cell treatments

NCT ID NCT04008368

Recruiting now 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 offers a second stem cell transplant to people with sickle cell disease or beta-thalassemia whose first transplant is losing its effect. Participants receive donor stem cells along with drugs to help their body accept them. The goal is to restore donor cell levels and control the disease long-term.

What this could mean

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

Active substance: Donor stem cells (CD34-selected cells)
What this could lead to: If successful, this could offer a way to regain disease control for people whose first stem cell transplant is wearing off, potentially reducing symptoms and complications.
What could go wrong: This is an early-phase trial with only 32 participants, so results may not apply to everyone. There are risks of graft rejection, graft-versus-host disease, and side effects from the conditioning drugs.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

beta thalassemia sickle cell disease

As listed by the trial registrant

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

MYELOID CHIMERISM

Contacts and locations

Locations

National Institutes of Health Clinical Center
RECRUITING

Bethesda, Maryland, 20892, United States

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