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Sirolimus (Rapamune ) for Relapse Prevention in People With Severe Aplastic Anemia Responsive to Immunosuppressive Therapy
NCT02979873 · View on ClinicalTrials.gov ↗
Study Summary
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. The treatment cyclosporine leads to better blood counts. But when this treatment is stopped, the disease may return in 1 in 3 people. The drug sirolimus may help by suppressing the immune system. Objective: To evaluate and compare the usefulness of sirolimus in preventing aplastic anemia from returning after cyclosporine is stopped, compared with stopping cyclosporine alone. Eligibility: People ages 2 and older with SAA who: Have responded to immunosuppressive therapy that includes cyclosporine, and continue to take cyclosporine Are not taking drugs with hematologic effects Design: Participants will be screened with: Medical history Physical exam Blood and urine tests Bone marrow biopsy: The area above the hipbone will be numbed. A thin needle will remove some bone marrow. 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 3 months with close monitoring. Group 2 will not receive the study drug but will be monitored closely. 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. These will include: Blood and urine tests Bone marrow biopsy
Conditions Studied
Interventions
- DRUG Sirolimus
Study Locations (1)
Maryland
- National Institutes of Health Clinical Center — Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 84 participants |
| Start Date | 2016-12-19 |
| Est. Completion | 2030-06-30 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT02979873
The ClinicalTrials.gov registry entry for NCT02979873 describes a study currently listed as active not recruiting. It is categorized as Phase 2, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 84 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is National Heart, Lung, and Blood Institute (NHLBI), which has 381 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Severe Aplastic Anemia appearing as the primary indexed condition, and to 1 intervention — of which Sirolimus is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT02979873 reports 1 study location spanning 1 distinct geographic area — top geographies include Maryland. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT02979873 about?
NCT02979873 is a clinical study titled "Sirolimus (Rapamune ) for Relapse Prevention in People With Severe Aplastic Anemia Responsive to Immunosuppressive Therapy". 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. The treatment cyclosporine leads to better blood counts. But when this treatment is stopped, the disease may...
What is the current status of trial NCT02979873?
This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 84 participants. The study started on 2016-12-19. Estimated completion is 2030-06-30.
What conditions does trial NCT02979873 study?
This clinical trial studies the following conditions: Severe Aplastic Anemia. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT02979873?
The interventions under investigation include: Sirolimus (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT02979873?
This trial is sponsored by National Heart, Lung, and Blood Institute (NHLBI), which has 381 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT02979873 being conducted?
This trial has 1 study location across Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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