4
Participants
Start Date
November 16, 2021
Primary Completion Date
May 2, 2025
Study Completion Date
May 21, 2025
Empagliflozin
This is an open-label pilot study to evaluate the efficacy, safety, and tolerability of empagliflozin as a treatment for severe congenital neutropenia (SCN) in patients with glucose-6-phosphatase 3 (G6PC3) deficiency. Participants will be on a 12-month daily regimen of empagliflozin at a starting dose of 10 mg (phase A), which may be increased after 2 months to 25 mg (phase B). Evaluate the safety and efficacy of the biomarker response (the change in absolute neutrophil count \[ANC\] after one year of empagliflozin treatment relative to baseline ANC prior to drug treatment) in patients with G6PC3 deficiency.
National Institutes of Health Clinical Center, Bethesda
National Institute of Allergy and Infectious Diseases (NIAID)
NIH