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NCT01328587 · ClinicalTrials.gov registry record · Phase 2
Eltrombopag for Moderate Aplastic Anemia
A Phase 2 study of Moderate Aplastic Anemia and Unilineage Bone Marrow Failure Disorders, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- Active
- Registry status
- Phase 2
- Development phase
- 34
- Enrollment target
- 1
- Study location
NCT01328587: Active Phase 2 study of Moderate Aplastic Anemia and Unilineage Bone Marrow Failure Disorders, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
NCT01328587 is a Phase 2 study of Moderate Aplastic Anemia and Unilineage Bone Marrow Failure Disorders that is active but no longer recruiting, run by National Heart, Lung, and Blood Institute (NHLBI). The registered enrollment target is 34 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (74% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01328587, a Phase 2 study of Moderate Aplastic Anemia and Unilineage Bone Marrow Failure Disorders, is active but no longer recruiting, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- ACTIVE NOT RECRUITING
- Registry status
- Phase 2
- Development phase
- 34 participants
- Enrollment target
- 1
- Study location
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
Primary Outcome
Defined by changes in platelet count and/or platelet transfusion requirements or hemoglobin (Hb) and/or PRBC transfusion requirements and the toxicity profile as measured by CTCAE. Response for platelet lineage is defined as absolute increase of ≥20x10\^9/L above baseline at 16 or 20 weeks, with at least 2 serial measurements performed 1 week apart and sustained for 1 month or more without platelet transfusions, or for transfusion dependent patients stable platelet counts with transfusion indepe
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 |
What the registry record for NCT01328587 still lists
NCT01328587 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 34 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (74% lower).
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.
NCT01328587 reports a single indexed study location in Maryland.
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.
What interventions are being tested in trial NCT01328587?
The interventions under investigation include: Eltrombopag (DRUG).
Who is sponsoring clinical trial NCT01328587?
This trial is sponsored by National Heart, Lung, and Blood Institute (NHLBI), which has 501 total clinical trials registered on ClinicalTrials.gov.
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.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01328587's enrollment target sits among peer trials
34 1539th of 2000 higher than 448 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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