Status:
UNKNOWN
Safety and Efficacy of Metformin for Treatment of Cytopenia in Children and Adolescents With Fanconi Anemia
Lead Sponsor:
Ain Shams University
Conditions:
Fanconi Anemia
Eligibility:
All Genders
5-18 years
Phase:
PHASE3
Brief 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.
Detailed Description
Fanconi anemia (FA) is a genetic disease characterized by bone marrow failure, cancer susceptibility, and developmental abnormalities. Allogeneic hematopoietic stem cell transplantation offers curativ...
Eligibility Criteria
Inclusion
- 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.
Exclusion
- Patients who underwent bone marrow transplantation.
- Patients with evidence of myelodysplasia, leukemia, or other concurrent malignancy.
- Patients who have a history of allergic reactions to metformin or similar compounds.
- Patients with a history of symptomatic hypoglycemia over the past year or hypoglycemia \< 50 mg/dL on screening and baseline laboratory assessments.
- Patients with type 1 diabetes mellitus.
- Patients with vitamin B12 deficiency.
- Patients with Glucose-6-Phosphate Dehydrogenase deficiency.
- Patients with abnormal Kidney function tests including serum creatinine, elevated liver function tests including live enzymes (ALT or AST \> 135 U/L, total bilirubin \> 1.5 x upper limit of normal for age, and/or patients with metabolic acidosis (bicarbonate \< 17 meq/L on venous blood gases).
Key Trial Info
Start Date :
October 5 2022
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
March 1 2025
Estimated Enrollment :
30 Patients enrolled
Trial Details
Trial ID
NCT06519786
Start Date
October 5 2022
End Date
March 1 2025
Last Update
July 25 2024
Active Locations (2)
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1
University of Alexandria
Alexandria, Egypt
2
Ain Shams University
Cairo, Egypt, 11566