Safety and Efficacy of Metformin for Treatment of Cytopenia in Children and Adolescents With Fanconi Anemia
Status: Recruiting
Location: See all (2) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
SUMMARY
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 5
Maximum Age: 18
Healthy Volunteers: f
View:
• Age: 5 to 18 years
• Patients who are diagnosed with Fanconi anemia based on clinical features and confirmed by increased chromosomal breakage on diepoxybutane (DEB) stress testing.
• Presence of cytopenia (at least one of the following: hemoglobin (Hb) \< 10 g/dL, platelet count \< 100 x 109/L, or an absolute neutrophil count (ANC) \< 1.0 x 109/L
• Patients receiving other therapies e.g., androgens are eligible for enrollment after a one-month washout period before the start of metformin.
Locations
Other Locations
Egypt
University of Alexandria
RECRUITING
Alexandria
Ain Shams University
RECRUITING
Cairo
Contact Information
Primary
Sara M Makkeyah, MD
smakkeyah@med.asu.edu.eg
+201140105222
Time Frame
Start Date: 2022-10-05
Estimated Completion Date: 2025-03
Participants
Target number of participants: 30
Treatments
Experimental: Metformin group
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).
No_intervention: Other treatment group
Patients will receive supportive treatment as indicated
Related Therapeutic Areas
Sponsors
Leads: Ain Shams University