About the Study

Who runs this study?

Principle Investigator
Andre Larochelle, M.D., Ph.D.
National Heart, Lung and Blood Institute
National Institutes of Health

Research Nurse
Bretagne Cowling, RN
National Heart, Lung and Blood Institute
National Institutes of Health

What is this study about?

The goal of this clinical trial is to assess the safety and efficacy of eltrombopag at improving blood counts in patients with Fanconi anemia.

What kind of commitment is involved?

• First visit at the NIH: Evaluation to determine eligibility, including:
  • Medical history and physical examination
  • Collection of blood samples
  • A pregnancy test in women of childbearing potential
  • A bone marrow biopsy test
  • A questionnaire about your quality of life
  • A 24-hour urine collection may be performed
• Treatment period: eligible patients will take eltrombopag pills by mouth once a day for 6 months. While on eltrombopag during the first 6 months:
  • Bloods tests will be performed every 2 weeks. These tests can be done at the NIH or at the patient's local clinic.
  • Patients will also need to come to the NIH 3 months and 6 months after the first dose of eltrombopag to have tests done.
• At the end of 6 months of treatment:
  • Patients who demonstrate response by protocol criteria at 6 months will be offered participation in the extended phase of the study for an additional 3 years. While on eltrombopag in the extended phase of the study:
    • Patients must be evaluated at the NIH every 6 months.
    • Patients must continue periodic laboratory monitoring at the NIH or at the patient's local clinic every month.
  • Patients who do not demonstrate response by protocol criteria at 6 months will stop eltrombopag. These patients will be offered a follow-up evaluation at the NIH 6 months following being taken off eltrombopag, but this visit will not be required.

How can I ask questions or ask for help?

Email us at bretagne.cowling@nih.gov (or click on Contact). We will respond within 24 hours.