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Mismatched donor stem cell transplant could widen treatment access for sickle cell disease

NCT ID NCT03653338

Recruiting now Disease control Sponsor: Paul Szabolcs Source: ClinicalTrials.gov ↗

First seen Jul 07, 2026 · Last updated Jul 08, 2026 · Updated 1 time

Summary

This study tests a stem cell transplant using cells from mismatched unrelated or partially matched family donors for people with severe sickle cell disease and other transfusion-dependent anemias. The approach removes certain immune cells (T-cells) from the donated stem cells to lower the risk of graft-versus-host disease, a serious complication. The goal is to see if this method can safely increase the number of patients who can receive a potentially curative transplant.

What this could mean

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

Active substance
T-cell depleted stem cells from mismatched unrelated or haploidentical related donors
What this could lead to
If successful, this approach could expand donor options for stem cell transplants, making curative-intent treatment available to more patients with severe sickle cell disease.
What could go wrong
This is an early-phase trial with only 5 participants, so results may not generalize. Risks include graft rejection, graft-versus-host disease, and treatment-related mortality.

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Conditions

The condition(s) this trial relates to.

anemia beta thalassemia beta-thalassemia major Diamond-Blackfan anemia sickle cell disease

As listed by the trial registrant

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

BETA-THALASSEMIA MAJOR DIAMOND BLACKFAN ANEMIA SICKLE CELL ANEMIA

Contacts and locations

Study contacts

  • Contact

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

  • Contact

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

Locations

  • Children's Hospital of Pittsburgh of UPMC

    RECRUITING

    Pittsburgh, Pennsylvania, 15224, United States

    Contact

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