Sirolimus (Rapamune ) for Relapse Prevention in People With Severe Aplastic Anemia Responsive to Immunosuppressive Therapy
Public ClinicalTrials.gov record NCT02979873. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
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Official title
A Randomized Trial of Sirolimus (Rapamune(R)) for Relapse Prevention in Patients With Severe Aplastic Anemia Responsive to Immunosuppressive Therapy
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
Background: People with severe aplastic anemia (SAA) do not make enough red and white blood cells, and/or platelets. Their body's immune system stops the bone marrow from making these cells. Immunosuppression (IST) with anti-thymocyte globulin (ATG) and cyclosporine (CSA) leads to better blood counts. But when cyclosporine is stopped, the disease may return in 1 in 3 people. The drug sirolimus (SRL) may help by suppressing the immune system to prevent the return of aplastic anemia. Objective: To evaluate and compare the usefulness of SRL in preventing aplastic anemia from returning after cyclosporine is stopped, compared with stopping cyclosporine alone. Eligibility: 1. People ages 2 and older with SAA who: 2. Have responded to IST and continue to take cyclosporine 3. Are not taking drugs with hematologic effects Study Design: Participants will be screened with: • Medical history, physical exam, blood and urine tests, bone marrow biopsy and aspirate. Participants will be randomly assigned to a group. All will stop cyclosporine. Group 1 will take sirolimus by mouth at the same time each day for 12 weeks with close monitoring. Group 2 will not receive the study drug but will be monitored closely (Observation arm). Participants will have clinical tests for the first 3 months: * Weekly blood test Monthly fasting blood test * For group 1, measurements of sirolimus in the blood every 1-2 weeks Participants will have clinic visits at 3 months, 12 months, and annually for 5 years after the study starts. They may have another visit if their SAA returns.
Study identification
- NCT ID
- NCT02979873
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 84 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 2 Years to 99 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Dec 19, 2016 (Actual)
- Primary completion
- Sep 4, 2025 (Actual)
- Completion
- Jun 30, 2030 (Estimated)
- Last update posted
- Oct 6, 2026
2016 – 2030
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| National Institutes of Health Clinical Center | Bethesda | Maryland | 20892 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT02979873, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Oct 6, 2026 · Synced Nov 9, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT02979873 live on ClinicalTrials.gov.