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New stem cell transplant aims to ease severe blood disorders

NCT ID NCT07599176

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

First seen Jun 25, 2026 · Last updated Jul 14, 2026 · Updated 12 times

Summary

This clinical trial tests a partial stem cell transplant for people with severe sickle cell disease or beta-thalassemia who need regular blood transfusions. The transplant uses a lower-intensity conditioning regimen with alemtuzumab, low-dose radiation, and abatacept to help donor cells take hold. The goal is to achieve long-term donor cell levels that reduce disease complications. The study is currently recruiting 90 participants.

What this could mean

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

Active substance
stem cell transplant with alemtuzumab, total body irradiation, and abatacept
What this could lead to
If successful, this could offer a long-term treatment option for severe sickle cell disease and beta-thalassemia, reducing the need for blood transfusions.
What could go wrong
This is an early phase 1/2 trial with a small number of participants. Risks include graft-versus-host disease, infection, and the possibility that the transplant may not work as hoped.

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.

BETA-THALASSEMIA SICKLE CELL DISEASE

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • National Institutes of Health Clinical Center

    RECRUITING

    Bethesda, Maryland, 20892, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

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