Pilot Study of Metformin for Patients With Fanconi Anemia

Inclusion Criteria

• •Age \> 6 years and ≤35 years
• •Lansky/Karnofsky performance status ≥ 50% for patients ≥16 years of age and Lansky ≥ 50% for patients \<16 years of age (see Appendix A)
• •Diagnosis requirement
• •Participants must have a clinical diagnosis of Fanconi Anemia.
• •* Participants must have confirmed diepoxybutane-mitomycin C (DEB/MMC) stress testing to document diagnosis of Fanconi Anemia.
• •* Patients must have at least one of the following cytopenias: Hemoglobin \<10g/dL; Platelets \<100k/uL; Absolute neutrophil count \<1000/uL
• •Participants must have normal organ function as defined below:
• •* Hepatic Function : Total bilirubin ≤ 1.5 x upper limit of normal for age; alanine aminotransferase (ALT)/aspartate aminotransferase (AST) ≤ 135 U/L
• •* Renal Function: A serum creatinine based on age/gender as follows:
• •Age Maximum Serum Creatinine (mg/dL) Male Female
• •1. to \< 2 years 0.6 0.6
• •2. to \< 6 years 0.8 0.8
• •6 to \< 10 years 1 1 10 to \< 13 years 1.2 1.2 13 to \< 16 years 1.5 1.4
• •≥ 16 years 1.7 1.4
• •AND
• •Creatinine clearance ≥ 60 mL/min/1.73 m2 for participants with creatinine levels above institutional normal
• •Normal cardiac status as documented clinically, otherwise they will need an echocardiogram prior to enrollment
• •Serum bicarbonate must be \>17.
• •Participants of child-bearing or child-fathering potential must agree to use adequate contraception (hormonal birth control; intrauterine device; double barrier method; or total abstinence) throughout their participation, including up until 30 days after last dose of Metformin.
• •Patients must be able to swallow pills.
• •Ability to understand and/or the willingness of the patient (or parent or legally authorized representative, if minor) to provide informed consent, documented using an institutionally approved informed consent procedure