New stem cell transplant trial offers hope for rare blood diseases
NCT ID NCT03520647
First seen Jun 27, 2026 · Last updated Jun 27, 2026
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.
Disclaimer
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This is a summary of
the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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National Institutes of Health Clinical Center
RECRUITINGBethesda, Maryland, 20892, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••