New drug may keep aplastic anemia from returning
NCT ID NCT02979873
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests whether the drug sirolimus can prevent severe aplastic anemia from coming back after patients stop taking cyclosporine. About 84 people aged 2 and older who have responded to initial treatment will be randomly assigned to either take sirolimus for 3 months or just stop cyclosporine without any new drug. Researchers will monitor blood counts and check for relapse over 5 years.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
sirolimus (Rapamune)
What this could lead to
If it works, this could offer a way to prevent relapse in severe aplastic anemia patients after stopping standard therapy, reducing the need for retreatment.
What could go wrong
This is a small Phase 2 trial with only 84 participants, so results may not apply to everyone. Sirolimus can cause side effects like infections or kidney issues, and it may not prevent relapse in all patients.
Disclaimer
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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.
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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Locations
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National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States