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NCT01623167 · ClinicalTrials.gov registry record · Phase 1

Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia

A Phase 1 study of Severe Aplastic Anemia, sponsored by National Heart, Lung, and Blood Institute (NHLBI).

Active
Registry status
Phase 1
Development phase
207
Enrollment target
1
Study location

NCT01623167 is a Phase 1 study of Severe Aplastic Anemia that is active but no longer recruiting, run by National Heart, Lung, and Blood Institute (NHLBI). The registered enrollment target is 207 participants, above the 114-participant average among 16 other Severe Aplastic Anemia trials with a reported enrollment target (82% higher). The trial reports 1 study location across 1 state.

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The verdict

NCT01623167, a Phase 1 study of Severe Aplastic Anemia, is active but no longer recruiting, sponsored by National Heart, Lung, and Blood Institute (NHLBI).

ACTIVE NOT RECRUITING
Registry status
Phase 1
Development phase
207 participants
Enrollment target
1
Study location

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

FieldValue
Enrollment Target 207 participants
Start Date 2012-07-02
Est. Completion 2029-11-30
Phase Phase 1

What the Registry Record Tells You About NCT01623167

The ClinicalTrials.gov registry entry for NCT01623167 describes a study currently listed as active not recruiting, categorized as Phase 1. The registered enrollment target is 207 participants, a figure that helps gauge the scale of data the investigators plan to collect, above the 114-participant average among 16 other Severe Aplastic Anemia trials with a reported enrollment target (82% higher). 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 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.

NCT01623167 reports 1 study location spanning 1 distinct geographic area - top geographies include Maryland.

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.

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).

Who is sponsoring clinical trial NCT01623167?

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 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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