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Eltrombopag for People With Fanconi Anemia

NCT03206086 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 25

Last updated 2025-08-24

No results posted yet for this 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

  • Fanconi Anemia

Interventions

DRUG

Eltrombopag

Daily dose - Non-Asian (\>=12): 150 mg Non-Asian (6-11): 75 mg East Asian, South East Asian (\>=12): 75 mg East Asian, South East Asian (6-11): 37.5 mg

Sponsors & Collaborators

  • National Heart, Lung, and Blood Institute (NHLBI)

    lead NIH

Principal Investigators

  • Andre Larochelle, M.D. · National Heart, Lung, and Blood Institute (NHLBI)

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Years
Max Age
99 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-11-02
Primary Completion
2025-08-01
Completion
2028-08-01
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03206086 on ClinicalTrials.gov