New drug may keep aplastic anemia from returning

NCT ID NCT02979873

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

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.

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.

acquired aplastic anemia aplastic anemia

As listed by the trial registrant

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

SEVERE APLASTIC ANEMIA

Contacts and locations

Locations

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

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Other studies related to the condition(s) this trial covers.