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Eltrombopag for Moderate Aplastic Anemia
NCT01328587 · View on ClinicalTrials.gov ↗
Study Summary
Background: * Moderate aplastic anemia is a blood disease which may require frequent blood and platelet transfusions. Sometimes patients with this disease can be treated with immunosuppressive drugs. Not all patients respond and not all patients are suitable for this treatment. * Thrombopoietin (TPO) is a protein made by the body. The bone marrow needs TPO to produce platelets. TPO may also be able to stimulate bone marrow stem cells to produce red cells and white cells. However, TPO cannot be given by mouth. This has led researchers to develop the drug eltrombopag, which acts in the same way and can be given by mouth. Eltrombopag has been shown to safely increase platelet numbers in healthy volunteers and in patients with other chronic blood diseases, including severe aplastic anemia. Researchers are interested in looking at whether eltrombopag can be given to people with moderate aplastic anemia and significantly low blood cell counts. Objectives: \- To evaluate the safety and effectiveness of eltrombopag in people with moderate aplastic anemia or patients with bone marrow failure and unilineage cytopenia who need treatment for significantly low blood cell counts. Eligibility: \- People at least 2 years of age who have moderate aplastic anemia or bone marrow failure and unilineage cytopenia,and significantly low blood cell counts. Design: * Patients will be screened with a physical examination, medical history, blood tests, a bone marrow biopsy, and an eye exam. * Patients will receive eltrombopag by mouth once a day. * Patients will have weekly blood tests to monitor the effectiveness of the treatment and adjust the dose in response to possible side effects. * Patients may continue to take eltrombopag if their platelet count or hemoglobin increases, their requirement for platelet or blood transfusion decreases after 16 to 20 weeks of treatment, and there have been no serious side effects. Access to the drug will continue until the study is closed. Patients
Conditions Studied
Interventions
- DRUG Eltrombopag
Study Locations (1)
Maryland
- National Institutes of Health Clinical Center, 9000 Rockville Pike — Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 34 participants |
| Start Date | 2011-04-01 |
| Est. Completion | 2025-06-30 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT01328587
The ClinicalTrials.gov registry entry for NCT01328587 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 34 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 2 conditions, with Moderate Aplastic Anemia appearing as the primary indexed condition, and to 1 intervention — of which Eltrombopag 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: NCT01328587 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 NCT01328587 about?
NCT01328587 is a clinical study titled "Eltrombopag for Moderate Aplastic Anemia". Background: * Moderate aplastic anemia is a blood disease which may require frequent blood and platelet transfusions. Sometimes patients with this disease can be treated with immunosuppressive drugs. Not all patients respond and not all patients are suitable for this treatment. * Thrombopoietin (TP...
What is the current status of trial NCT01328587?
This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 34 participants. The study started on 2011-04-01. Estimated completion is 2025-06-30.
What conditions does trial NCT01328587 study?
This clinical trial studies the following conditions: Moderate Aplastic Anemia, Unilineage Bone Marrow Failure Disorders. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT01328587?
The interventions under investigation include: Eltrombopag (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT01328587?
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 NCT01328587 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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