Eltrombopag for People With Fanconi Anemia (NCT03206086) | Clinical Trial Compass
Active — Not RecruitingPhase 2
Eltrombopag for People With Fanconi Anemia
United States25 participantsStarted 2018-11-02
Plain-language 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....
Who can participate
Age range
6 Years – 99 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
* INCLUSION CRITERIA:
* Confirmed diagnosis of Fanconi anemia. Diagnosis is confirmed by a biallelic mutation in a known FANC gene and/or by positive chromosome breakage analysis in lymphocytes and/or skin fibroblasts (for mosaicism).
* One or more of the following three clinically-significant cytopenias: platelet count \<= 50,000/microliter or platelet-transfusion-dependence (requiring at least 4 platelet transfusions in the 8 weeks prior to study entry, see definition of platelet transfusion units at 8.2.1); neutrophil count less than 1000/microliter; hemoglobin less than 10 g/dL or red cell transfusion- dependence (requiring at least 4 transfusions of PRBCs (adult patient 4 units PRBC, pediatric patients at least 10ml/kg/transfusion) in the eight weeks prior to study entry.
* Failed or declined treatment with androgens (danazol or oxymetholone).
* Age \>= 6 years old.
* Weight \>=10kg.
EXCLUSION CRITERIA:
* Known active or uncontrolled infections not adequately responding to appropriate therapy.
* Evidence for MDS or AML as defined by WHO criteria.
* Any cytogenetic abnormality associated with poor prognosis in FA, including gains of chromosome 3q, gains of chromosome 1q, deletions of chromosome 7, and complex cytogenetics (88-90) identified from bone marrow aspirate. Patients with known biallelic mutations in BRCA2 (FANCD1).
* Active malignancy or likelihood of recurrence of malignancies within 12 months
* Moribund status such that death within 7 to 10 days is likely. C…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Proportion of drug responders
Timeframe: 6 months
2
Toxicity profile
Timeframe: 6 months
Trial details
NCT IDNCT03206086
SponsorNational Heart, Lung, and Blood Institute (NHLBI)