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Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia
NCT01623167 · View on ClinicalTrials.gov ↗
Study Summary
Background: * Severe aplastic anemia is a rare and serious blood disorder. It happens when the immune system starts to attack the bone marrow cells. This causes the bone marrow to stop making red blood cells, platelets, and white blood cells. Standard treatment for this disease is horse-ATG and cyclosporine, which suppress the immune system and stop it from attacking the bone marrow. However, this treatment does not work in all people. Some people still have poor blood cell counts even after treatment. * Eltrombopag is a drug designed to mimic a protein in the body called thrombopoietin. It helps the body to make more platelets. It may also cause the body to make more red and white blood cells. Studies have shown that eltrombopag may be useful when added to standard treatment for severe aplastic anemia. It may help improve poor blood cell counts. Objectives: \- To test the safety and effectiveness of adding eltrombopag to standard immunosuppressive therapy for severe aplastic anemia. Eligibility: \- Individuals at least 2 years of age who have severe aplastic anemia that has not yet been treated. Design: * Participants will be screened with a physical exam, medical history, and blood tests. Blood and urine samples will be collected. * Participants will start treatment with horse-ATG and cyclosporine. Treatment will be given according to the standard of care for the disease. * Cohort 1: After 14 days, participants will start taking eltrombopag. They will take eltrombopag for up to 6 months. * Cohort 2: After 14 days, participants will start taking eltrombopag. They will take eltrombopag for up to 3 months. * Cohort 3 and Extension Cohort: Participants will start taking eltrombopag on Day 1. They will take eltrombopag for up to 6 months. * Participants may receive other medications to prevent infections during treatment. * Treatment will be monitored with frequent blood tests. Participants will also fill out questionnaires about their symptoms and their quality
Conditions Studied
Interventions
- DRUG Cohort 1: hATG, CsA, EPAG Day 14 to Month 6
- DRUG Cohort 2: hATG, CsA, EPAG Day 14 to Month 3
- DRUG Cohort 3: hATG, CsA (dose reduced), EPAG day 1 to month 6
- DRUG Extension Cohort
Study Locations (1)
Maryland
- National Institutes of Health Clinical Center, 9000 Rockville Pike — Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 207 participants |
| Start Date | 2012-07-02 |
| Est. Completion | 2029-11-30 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT01623167
The ClinicalTrials.gov registry entry for NCT01623167 describes a study currently listed as active not recruiting. It is categorized as Phase 1, 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 207 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 4 interventions — of which Cohort 1: hATG, CsA, EPAG Day 14 to Month 6 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: NCT01623167 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 NCT01623167 about?
NCT01623167 is a clinical study titled "Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia". Background: * Severe aplastic anemia is a rare and serious blood disorder. It happens when the immune system starts to attack the bone marrow cells. This causes the bone marrow to stop making red blood cells, platelets, and white blood cells. Standard treatment for this disease is horse-ATG and cyc...
What is the current status of trial NCT01623167?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 207 participants. The study started on 2012-07-02. Estimated completion is 2029-11-30.
What conditions does trial NCT01623167 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 NCT01623167?
The interventions under investigation include: Cohort 1: hATG, CsA, EPAG Day 14 to Month 6 (DRUG), Cohort 2: hATG, CsA, EPAG Day 14 to Month 3 (DRUG), Cohort 3: hATG, CsA (dose reduced), EPAG day 1 to month 6 (DRUG), Extension Cohort (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT01623167?
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 NCT01623167 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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