A Phase 3 interventional study of Metformin in Fanconi Anemia, sponsored by Ain Shams University. Recruiting at 2 sites in Egypt. Open to participants aged 5 Years to 18 Years. Per ClinicalTrials.gov, last updated 2024-07-25.
Sponsored by Ain Shams University · Phase 3, Interventional, and Supportive care
Prospective interventional open-label non-randomized controlled trial to assess safety and efficacy of metformin in treating cytopenia in children and adolescents with Fanconi Anemia.
Fanconi anemia (FA) is a genetic disease characterized by bone marrow failure, cancer susceptibility, and developmental abnormalities. Allogeneic hematopoietic stem cell transplantation offers curative therapy for hematologic complications of FA.
Oxymetholone is commonly used in the management of FA as it improves blood counts, red cells, and platelets. However, its use is limited by its high toxicity profile.
Metformin is a potential agent that reduces levels of both chromosomal radials and breaks in FA cells and increases the size of the hematopoietic stem cell compartment thus reducing cytopenia in patients with FA.
Exclusion Criteria:
Patients will receive metformin immediate-release tablets orally for 24 weeks. Starting dose will be 500 mg once daily for all patients and the dose will be increased by 500 mg weekly until the goal dose is achieved (500 mg twice daily for patients \< 10 years of age, and 1000 mg twice daily for patients 10 years or older).
Drug: Metformin
Patients will receive supportive treatment as indicated
Patients will receive metformin immediate-release tablets orally for 24 weeks. Starting dose will be 500 mg once daily for all patients and the dose will be increased by 500 mg weekly until the goal dose is achieved (500 mg twice daily for patients \< 10 years of age, and 1000 mg twice daily for patients 10 years or older)
Also known as: Glucophage
Hematologic response (erythroid)
Erythroid response (pretreatment, \< 11 g/dL): Hgb increase by \> 1.5 g/dL or Relevant reduction of units of RBC transfusions by an absolute number of at least 4 RBC transfusions/8 wk compared with the pretreatment transfusion number in the previous 8 wk.
Time frame: 24 weeks
Hematologic response (platelets)
Platelet response (pretreatment, \< 100x10e9/L): Absolute increase of \> 30 x 10e9/L for patients starting with \> 20 x 10e9/L platelets or Increase from \< 20 x 10e9/L to \> 20 x10e9/L and by at least 100%
Time frame: 24 weeks
Hematologic response (Neutrophil count)
Neutrophil response (pretreatment, \< 1.0 x 10e9/L): At least 100% increase and an absolute increase \> 0.5 x 10e9/L
Time frame: 24 weeks
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Ain Shams University