Status:
COMPLETED
Eltrombopag for Treatment of Fanconi Anemia
Lead Sponsor:
Julian Sevilla
Conditions:
Fanconi Anemia
Eligibility:
All Genders
4-17 years
Phase:
PHASE2
Brief Summary
An open-label, phase II study to assess the efficacy and safety of eltrombopag for the treatment of children and adolescents with Fanconi anemia.
Detailed Description
Open-label, phase II study to assess the efficacy and safety of eltrombopag in the treatment of patients diagnosed with Fanconi anemia who have no immediate curative treatment for their bone marrow fa...
Eligibility Criteria
Inclusion
- Patients with a diagnosis of Fanconi anemia. Diagnosis will be defined by the presence of biallelic mutations in genes known to cause the disease and/or by positive chromosome breakage analysis of lymphocytes and/or skin fibroblasts (for cases of mosaicism).
- Age from 4 to ≤ 17 years.
- Lansky index \> 80%.
- Informed consent provided in accordance with current legislation.
- Presenting with one or more of one of the following clinically significant cytopenias:
- platelet count ≤ 50x109/L or transfusion dependence, having required at least 2 transfusions in the 8 weeks prior to inclusion in the study; granulocyte count \< 0.75x109/L; hemoglobin \< 9 gr/dL or transfusion-dependent anemia having received 2 transfusions in the 8 weeks prior to inclusion in the study.
Exclusion
- Patients with HLA-matched related donor or unrelated donor with a 12/12 match who is immediately available.
- Evidence of myelodysplastic syndrome or leukemia or cytogenetic abnormalities predictive of these disorders in bone marrow aspirates. In the event, the evaluations performed two months before patient inclusion in the clinical study will be considered valid.
- Baseline creatinine greater than 2.5 times the upper limit of normality.
- GOT/AST or GPT/ALT more than three times the upper limit of normality. Direct bilirubin greater than 1.5 times the upper limit of normality.
- Patients who are already receiving treatment with some drug for bone marrow failure may be included as long as the dose administered remains stable for at least two months. In the event that such treatment requires an increase in dose during the study, the patient must withdraw from the trial. Patients who have already started Revolade® treatment in the previous two months may also be included, and the blood counts and baseline bone marrow studies performed at the start of treatment will be used.
- Women of postpubertal age and therefore at risk of pregnancy should have a negative serum or urine pregnancy test at the screening visit and agree to use a contraceptive method throughout the treatment period and for at least one month after.
Key Trial Info
Start Date :
December 2 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
August 29 2023
Estimated Enrollment :
8 Patients enrolled
Trial Details
Trial ID
NCT06045052
Start Date
December 2 2020
End Date
August 29 2023
Last Update
September 21 2023
Active Locations (1)
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1
Hospital Infantil Universitario Niño Jesús (HIUNJ)
Madrid, Spain, 28009