NCT06519786

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

• Status: Unknown
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 30 (estimated)
• Sponsor: Ain Shams University · Academic / Other
• Sex: All
• Age: 5 Years – 18 Years
• Healthy volunteers: Not accepted

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 curative therapy for hematologic complications of FA. Oxymetholone is commonly used in the management of FA as it improves blood counts, red cells, and platelets. However, its use is limited by its high toxicity profile. Metformin is a potential agent that reduces levels of both chromosomal radials and breaks in FA cells and increases the size of the hematopoietic stem cell compartment thus reducing cytopenia in patients with FA.

Conditions

• Fanconi Anemia

Interventions

Type	Name	Description
DRUG	Metformin	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)

Timeline

• Start date: 2022-10-05
• Primary completion: 2024-10-01
• Completion: 2025-03-01
• First posted: 2024-07-25
• Last updated: 2024-07-25

Locations

2 sites across 1 country: Egypt