Empagliflozin as a Treatment for Severe Congenital Neutropenia Due to G6PC3 Deficiency

PHASE1CompletedINTERVENTIONAL
Enrollment

4

Participants

Timeline

Start Date

November 16, 2021

Primary Completion Date

May 2, 2025

Study Completion Date

May 21, 2025

Conditions
Crohn's DiseaseGlycogen MetabolismInflammatory Bowel Disease (IBD)
Interventions
DRUG

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.

Trial Locations (1)

20892

National Institutes of Health Clinical Center, Bethesda

All Listed Sponsors
lead

National Institute of Allergy and Infectious Diseases (NIAID)

NIH

NCT05078879 - Empagliflozin as a Treatment for Severe Congenital Neutropenia Due to G6PC3 Deficiency | Biotech Hunter | Biotech Hunter