Eltrombopag for Fanconi Anemia
Trial Summary
Do I need to stop my current medications to join the trial?
The trial protocol does not specify if you need to stop taking your current medications. However, you must not have taken androgens like danazol or oxymetholone within 4 weeks before starting eltrombopag.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications, but you must not have taken androgens like danazol or oxymetholone in the 4 weeks before starting the study drug.
What data supports the idea that Eltrombopag for Fanconi Anemia is an effective treatment?
The available research shows that Eltrombopag can be effective for Fanconi Anemia. In one case, it was used alongside another drug, oxymetholone, and helped improve blood cell production in three different types of blood cells. This improvement meant the patient no longer needed blood transfusions before undergoing a stem cell transplant, which is a major treatment for Fanconi Anemia. This suggests that Eltrombopag can help manage the condition and improve outcomes when combined with other treatments.12345
What evidence supports the effectiveness of the drug Eltrombopag for treating Fanconi Anemia?
Eltrombopag has been shown to help increase blood cell production in conditions like severe aplastic anemia and chronic immune thrombocytopenia, which are similar to the blood cell issues seen in Fanconi Anemia. In one case, Eltrombopag, combined with another drug, helped a Fanconi Anemia patient improve blood cell counts and reduce the need for transfusions before a stem cell transplant.12345
What safety data exists for Eltrombopag in treating Fanconi Anemia?
The provided research does not contain safety data for Eltrombopag (also known as Promacta or Revolade) in the treatment of Fanconi Anemia. The studies focus on the adverse effects of all-trans retinoic acid (ATRA) in acute promyelocytic leukemia, which is unrelated to Eltrombopag or Fanconi Anemia.678910
Is the drug Eltrombopag a promising treatment for Fanconi Anemia?
Eltrombopag is a promising drug because it helps increase platelet counts, which are important for blood clotting. It has been effective in treating other conditions with low platelet counts, like chronic immune thrombocytopenia and severe aplastic anemia, by boosting blood cell production. This suggests it could be beneficial for Fanconi Anemia, which also involves blood cell issues.123411
How is the drug eltrombopag unique in treating Fanconi Anemia?
Eltrombopag is unique because it is an oral drug that stimulates platelet production by activating thrombopoietin receptors, which is different from other treatments that may involve more invasive procedures like blood transfusions or bone marrow transplants. It is also used in other conditions to increase platelet counts, making it a novel option for managing blood cell deficiencies in Fanconi Anemia.123411
What is the purpose of this trial?
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 lifeHave a physical examHave blood and urine testsHave 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....
Research Team
Andre Larochelle, M.D.
Principal Investigator
National Heart, Lung, and Blood Institute (NHLBI)
Eligibility Criteria
This trial is for people aged 2 or older with Fanconi anemia who have low blood cell counts and weigh over 10kg. They should not have had recent cancer, thromboembolic events, HIV, certain genetic mutations, severe illnesses that could affect the drug's tolerance, or be on other investigational drugs.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants take eltrombopag pills by mouth once a day for 24 weeks and are monitored closely for side effects
Follow-up
Participants are monitored for safety and effectiveness after treatment
Extension
Participants with improved blood cell counts may continue taking eltrombopag for 3 years
Treatment Details
Interventions
- Eltrombopag
Eltrombopag is already approved in United States, European Union, Canada, Japan, China for the following indications:
- Severe aplastic anemia
- Chronic immune thrombocytopenia
- Thrombocytopenia in patients with chronic hepatitis C
- Severe aplastic anemia
- Chronic immune thrombocytopenia
- Severe aplastic anemia
- Chronic immune thrombocytopenia
- Severe aplastic anemia
- Chronic immune thrombocytopenia
- Severe aplastic anemia
- Chronic immune thrombocytopenia
Find a Clinic Near You
Who Is Running the Clinical Trial?
National Heart, Lung, and Blood Institute (NHLBI)
Lead Sponsor