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NCT03206086 · ClinicalTrials.gov registry record · Phase 2

Eltrombopag for People With Fanconi Anemia

A Phase 2 study of Fanconi Anemia, sponsored by National Heart, Lung, and Blood Institute (NHLBI).

Active
Registry status
Phase 2
Development phase
25
Enrollment target
1
Study location

NCT03206086 is a Phase 2 study of Fanconi Anemia that is active but no longer recruiting, run by National Heart, Lung, and Blood Institute (NHLBI). The registered enrollment target is 25 participants, below the 335-participant average among 15 other Fanconi Anemia trials with a reported enrollment target (93% lower). The trial reports 1 study location across 1 state.

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The verdict

NCT03206086, a Phase 2 study of Fanconi Anemia, is active but no longer recruiting, sponsored by National Heart, Lung, and Blood Institute (NHLBI).

ACTIVE NOT RECRUITING
Registry status
Phase 2
Development phase
25 participants
Enrollment target
1
Study location

Study Summary

Background: Fanconi anemia is a genetic disease. Some people with it have reduced blood cell counts. This means their bone marrow no longer works properly. These people may need blood transfusions for anemia (low red blood cells) or low platelet counts or bleeding. Researchers want to see if a new drug will help people with this disease. Objective: To find out if a new drug, eltrombopag, is effective in people with Fanconi anemia. To know how long the drug needs to be given to improve blood counts. Eligibility: People at least 6 years old with Fanconi anemia with reduced blood cell counts. Design: Participants will be screened with blood and urine tests. They will repeat this before starting to take the study drug. Participants will take eltrombopag pills by mouth once a day for 24 weeks. They will be monitored closely for side effects. Participants will have blood tests every 2 weeks while on eltrombopag. Participants will visit NIH 3 months and 6 months after starting eltrombopag. At these visits, participants will: Answer questions about their medical history, how they are feeling, and their quality of life Have a physical exam Have blood and urine tests Have a bone marrow sample taken by needle from the hip. The area will be numbed. If participants blood cell counts improve, they might join the extended access part of the study. They will continue taking eltrombopag for 3 years and sign a different consent. After 24 weeks of treatment, if there is no improvement in blood cell counts, participants will stop taking eltrombopag. They will return for an optional follow-up visit that repeats the study visits....

Conditions Studied

Interventions

  • DRUG Eltrombopag

Study Locations (1)

Maryland

  • National Institutes of Health Clinical Center - Bethesda

Trial Details

FieldValue
Enrollment Target 25 participants
Start Date 2018-11-02
Est. Completion 2028-08-01
Phase Phase 2

What the Registry Record Tells You About NCT03206086

The ClinicalTrials.gov registry entry for NCT03206086 describes a study currently listed as active not recruiting, categorized as Phase 2. The registered enrollment target is 25 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 335-participant average among 15 other Fanconi Anemia trials with a reported enrollment target (93% lower). 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 Fanconi Anemia appearing as the primary indexed condition, and to 1 intervention - of which Eltrombopag is the first listed.

NCT03206086 reports 1 study location spanning 1 distinct geographic area - top geographies include Maryland.

Frequently Asked Questions

What is clinical trial NCT03206086 about?

NCT03206086 is a clinical study titled "Eltrombopag for People With Fanconi Anemia". Background: Fanconi anemia is a genetic disease. Some people with it have reduced blood cell counts. This means their bone marrow no longer works properly. These people may need blood transfusions for anemia (low red blood cells) or low platelet counts or bleeding. Researchers want to see if a new ...

What is the current status of trial NCT03206086?

This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 25 participants. The study started on 2018-11-02. Estimated completion is 2028-08-01.

What conditions does trial NCT03206086 study?

This clinical trial studies the following conditions: Fanconi Anemia.

What interventions are being tested in trial NCT03206086?

The interventions under investigation include: Eltrombopag (DRUG).

Who is sponsoring clinical trial NCT03206086?

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 NCT03206086 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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