New stem cell method aims to cut transplant risks

NCT ID NCT01174108

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

First seen Jun 25, 2026 · Last updated Jul 02, 2026 · Updated 4 times

Summary

This study tests a new way to do stem cell transplants for people with severe aplastic anemia or other bone marrow failure diseases. Doctors give a smaller number of donor white blood cells along with the stem cells to try to reduce serious side effects. Up to 120 patients aged 4 to 80 will take part, and they will be followed for up to 3 years.

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 and white blood cells
What this could lead to: If successful, this approach could make stem cell transplants safer and more effective for people with severe blood diseases.
What could go wrong: This is an early Phase 2 trial with only 120 participants, so results may not apply to everyone. There are still risks of serious side effects like graft-versus-host disease or infection.

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Conditions

The condition(s) this trial relates to.

acquired aplastic anemia aplastic anemia Hemoglobinuria, Paroxysmal myelodysplastic syndrome myelodysplastic syndrome with excess blasts myelodysplastic syndrome with ring sideroblasts myelodysplastic syndrome with single lineage dysplasia Myelodysplastic Syndromes paroxysmal nocturnal hemoglobinuria pure red-cell aplasia

As listed by the trial registrant

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

MDS (MYELODYSPLASTIC SYNDROME) SEVERE APLASTIC ANEMIA

Contacts and locations

Locations

National Institutes of Health Clinical Center
RECRUITING

Bethesda, Maryland, 20892, United States
University of Maryland, Baltimore (UMB)
COMPLETED

Baltimore, Maryland, 21201, United States

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