Using Metformin to Treat Blood Disorders in Children with Fanconi Anemia
Safety and Efficacy of Metformin for Treatment of Cytopenia in Children and Adolescents With Fanconi Anemia
This study is testing if metformin can safely help improve blood counts in children and teens with Fanconi anemia who have low blood cell levels.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Enrollment | 30 (estimated) |
| Ages | 5 Years to 18 Years |
| Sex | All |
| Sponsor | Ain Shams University Academic / other |
| Locations | 2 sites (Alexandria and 1 other locations) |
| Trial ID | NCT06519786 on ClinicalTrials.gov |
What this trial studies
This interventional trial aims to evaluate the safety and efficacy of metformin in treating cytopenia in children and adolescents diagnosed with Fanconi anemia. The study will involve participants aged 5 to 18 years who exhibit specific blood count deficiencies. Metformin is being investigated as a potential alternative to oxymetholone, which, while effective, has a high toxicity profile. The trial will assess how metformin can improve blood counts and reduce cytopenia in this patient population.
Who should consider this trial
Good fit: Ideal candidates are children and adolescents aged 5 to 18 years diagnosed with Fanconi anemia and experiencing cytopenia.
Not a fit: Patients who have undergone bone marrow transplantation or have concurrent malignancies will not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a safer alternative for managing cytopenia in children with Fanconi anemia.
How similar studies have performed: While the use of metformin in this context is novel, previous studies have shown its potential in reducing chromosomal damage in similar conditions.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * 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 Criteria: * 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).
Where this trial is running
Alexandria and 1 other locations
- University of Alexandria — Alexandria, Egypt (Recruiting)
- Ain Shams University — Cairo, Egypt (Recruiting)
Study contacts
- Study coordinator: Sara M Makkeyah, MD
- Email: smakkeyah@med.asu.edu.eg
- Phone: +201140105222
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.