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You can search the collection of research studies being conducted at the NIH Clinical Center at Search the Studies. In addition, clinicaltrials.gov provides patients, family members, and members of the public easy and free access to information on clinical studies for a wide range of diseases and conditions.

Patient Recruitment

Eltrombopag for Patients with Fanconi Anemia

Have You Been Diagnosed With Fanconi Anemia?

mother and child smiling

cells

Fanconi anemia is a rare genetic disorder that mostly affects children and often leads to bone marrow failure. Researchers at the National Institutes of Health (NIH) are investigating a new drug to improve blood counts in individuals diagnosed with Fanconi anemia. Eltrombopag is an oral drug that mimics a special protein that causes the body to make more platelets. Currently eltrombopag is used to treat low platelet counts in patients with hepatitis C and chronic immune thrombocytopenic purpura (ITP). Researchers are evaluating if this new drug will improve blood cell counts in individuals diagnosed with Fanconi anemia.

Eligible participants:

  • At least 4 years old.
  • Have been diagnosed with Fanconi anemia by genetic testing.
  • Have declined or not responded to treatment with danazol or oxymetholone.
  • Weight more than 12 kg (26 lbs)

What is involved?

  • A review of medical records, and physical exam to determine eligibility for the study.
  • Participating in the study may last up to 3.5 years.
  • Participants with improvement in their blood cell counts at 6 months will have the option to continue taking the medication for an additional 3 years.
  • You may receive blood and or platelet transfusions.
  • Tests, medication, and procedures conducted at the NIH Clinical Center are at no cost to you.
  • Compensation for travel may be provided.

Location: The NIH Clinical Center is America's research hospital is located in Bethesda, Maryland, on the Metro red line (Medical Center stop).

For more information, call:
Office of Patient Recruitment
1-800-411-1222
TTY: 1-866-411-1010
Se habla español

Or go online:
https://go.usa.gov/xE5Z8
Refer to study #17-H-0121

Department of Health and Human Services
National Institutes of Health Clinical Center (CC)
National Heart, Lung, and Blood Institute (NHLBI)

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This page last updated on 03/07/2019

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