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Dataset Details (NCT03398824)

Superfund Research Program

Title: Pilot Study of Metformin for Patients With Fanconi Anemia

Accession Number: NCT03398824

Link to Dataset:


Data Type(s): Clinical Trials

Summary: This is a single institution, open-label, single arm pilot study of Metformin in patients with Fanconi Anemia (FA) and cytopenias with the primary endpoint of hematologic response. This study will also assess safety, tolerability, and the biologic effects of Metformin in patients with FA.

Publication(s) associated with this dataset:
  • Pollard JA, Furutani E, Liu S, Esrick E, Cohen LE, Bledsoe J, Liu C, Lu K, Ramirez de Haro MJ, Surralles J, Malsch M, Kuniholm A, Galvin A, Armant M, Kim AS, Ballotti K, Moreau L, Zhou Y, Babushok D, Boulad F, Carroll C, Hartung H, Hont A, Nakano T, Olson T, Sze S, Thompson AA, Wlodarski MW, Gu X, Libermann TA, D'Andrea A, Grompe M, Weller E, Shimamura A. 2022. Metformin for treatment of cytopenias in children and young adults with Fanconi anemia. Blood Adv 6(12):3803-3811. doi:10.1182/bloodadvances.2021006490 PMID:35500223
Project(s) associated with this dataset:
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