Eltrombopag for People With Fanconi Anemia

Updated on 22 December 2020
anemia studies
fanconi's anemia
fanconi's syndrome


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.

For more information about this study you can access the NIH patient recruitment study page at https://www.cc.nih.gov/recruit/protocols/17_fanconi-anemia.html.



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.

Condition Anemia, Anemia, Anemia; Non-Hodgkin’s Lymphoma, Fanconi Anemia Rare Diseases Genetic Disease Reduced Blood Cell Count
Clinical Study IdentifierTX217949
Last Modified on22 December 2020


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Inclusion Criteria

People at least 4 years old with Fanconi Anemia with reduced blood cell counts
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How to participate?

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What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer  to help accelerate the development of new treatments and to get notified about similar trials.

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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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