New stem cell transplant trial offers hope for rare blood diseases
NCT ID NCT03520647
First seen Jun 27, 2026 · Last updated Jul 02, 2026 · Updated 1 time
Summary
This study tests a stem cell transplant using blood stem cells from a half-matched family donor (haploidentical) for people with severe aplastic anemia, related MDS, or PNH. Participants receive chemotherapy and a single radiation dose before the transplant, plus a drug to prevent graft-versus-host disease. The goal is to see if this approach improves survival without chronic GVHD at one year.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Peripheral blood stem cells and cyclophosphamide
- What this could lead to
- If successful, this approach could offer a safer, more available transplant option for people with severe blood disorders who lack a matched donor.
- What could go wrong
- This is an early-phase trial with only 56 participants. The transplant carries risks like graft-versus-host disease, infection, and organ damage. It may not work for everyone.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
National Institutes of Health Clinical Center
RECRUITINGBethesda, Maryland, 20892, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Could a new pill tame a rare blood disease? early safety trial begins
- Experimental drug targets bone marrow failure
- New drug pegcetacoplan tested in teens with rare blood disorder
- Simpler stem cell collection could ease donor burden in Life-Threatening anemia
- New drug cocktail aims to free aplastic anemia patients from transfusions
- New PNH drug VSA012 enters early human testing