Safety and Efficacy of Metformin for Treatment of Cytopenia in Children and Adolescents With Fanconi Anemia

Launched by AIN SHAMS UNIVERSITY · Jul 20, 2024

Keywords

ClinConnect Summary

This clinical trial is studying the safety and effectiveness of a medication called metformin in treating a condition known as cytopenia in children and adolescents with Fanconi Anemia. Cytopenia means that a person has low levels of certain blood cells, which can lead to problems like anemia (low red blood cells), low platelets, or low white blood cells. The researchers want to see if metformin, which is commonly used to treat diabetes, can help improve blood cell counts in young patients with this condition.

To participate in the trial, children and teens between the ages of 5 and 18 who have been diagnosed with Fanconi Anemia and have low blood cell counts may be eligible. However, those who have had a bone marrow transplant, certain blood cancers, or specific health issues related to blood sugar or kidney function are not eligible. Participants in the study can expect to receive metformin and will be monitored closely for any effects the medication may have on their health. This trial is currently recruiting participants, and it provides an opportunity to explore a potential new treatment option for managing cytopenia in these young patients.

Gender

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Eligibility 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).