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NCT00922883 · ClinicalTrials.gov registry record · Phase 2
A Pilot Study of the Thrombopoietin-Receptor Agonist Eltrombopag in Refractory Aplastic Anemia Patients
A Phase 2 study, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- Completed
- Registry status
- Phase 2
- Development phase
- 43
- Enrollment target
NCT00922883 is a Phase 2 study that has completed, run by National Heart, Lung, and Blood Institute (NHLBI). The registered enrollment target is 43 participants.
The verdict
NCT00922883, a Phase 2 study, has completed, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 43 participants
- Enrollment target
Study Summary
Severe aplastic anemia (SAA) is a life-threatening blood disease which can be effectively treated with immunosuppressive drug regimens or allogeneic stem cell transplantation. However, 20-40% of patients without transplant options do not respond to immunosuppressive therapies, and have persistent severe cytopenias, requiring regular platelet transfusions, which are expensive and inconvenient, and are a risk for further serious bleeding complications. Thrombopoietin (TPO) is the principal endogenous regulator of platelet production and also stimulates hematopoietic stem and progenitor cells. A small molecule oral TPO-agonist, eltrombopag has been shown to increase platelets in healthy subjects and in patients with immune thrombocytopenic purpura (ITP), and received FDA approval in 2008 for the treatment of thrombocytopenia in ITP. This Phase 2, non-randomized pilot study of eltrombopag in aplastic anemia patients with immunosuppressive therapy refractory thrombocytopenia will test the safety and potential efficacy of eltrombopag treatment patients with refractory thrombocytopenia following immunosuppression for aplastic anemia. Subjects will initiate study medication at an oral dose of 50 mg/day, which will be increased up to 150 mg/day as clinically indicated to the lowest dose that maintains a stable platelet count 20,000/(micro)L above baseline while maximizing tolerability. Response will be assessed at 3-4 months. Platelet response is defined as platelet count increases to 20,000/L above baseline at three months. or stable platelet counts with transfusion independence for a minimum of 8 weeks. Erythroid response for subjects with a pretreatment hemoglobin of less than 9 g/dL will be defined as an increase in hemoglobin by greater than or equal to 1.5g/dL without packed red blood cell (PRBC) transfusion support, or a reduction in the units of transfusions by an absolute number of at least 4 PRBC transfusions for eight consecutive weeks compared with the pretreat
Interventions
- DRUG Eltrombopag
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 43 participants |
| Start Date | 2009-05-29 |
| Est. Completion | 2018-04-24 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00922883
The ClinicalTrials.gov registry entry for NCT00922883 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 43 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 501 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which Eltrombopag is the first listed.
NCT00922883 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT00922883 about?
NCT00922883 is a clinical study titled "A Pilot Study of the Thrombopoietin-Receptor Agonist Eltrombopag in Refractory Aplastic Anemia Patients". Severe aplastic anemia (SAA) is a life-threatening blood disease which can be effectively treated with immunosuppressive drug regimens or allogeneic stem cell transplantation. However, 20-40% of patients without transplant options do not respond to immunosuppressive therapies, and have persistent se...
What is the current status of trial NCT00922883?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 43 participants. The study started on 2009-05-29. Estimated completion is 2018-04-24.
What interventions are being tested in trial NCT00922883?
The interventions under investigation include: Eltrombopag (DRUG).
Who is sponsoring clinical trial NCT00922883?
This trial is sponsored by National Heart, Lung, and Blood Institute (NHLBI), which has 501 total clinical trials registered on ClinicalTrials.gov.
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