NCT04930627

Safety and Efficacy of Empagliflozin in GSD1b Patients With Neutropenia

Evaluation of Efficacy and Safety of Empagliflozin in Treatment of Neutropenia in Patients With Glycogenosis Ib

• Status: Unknown
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: Children's Memorial Health Institute, Poland · Academic / Other
• Sex: All
• Age: 4 Weeks
• Healthy volunteers: Not accepted

Summary

Treatment of neutropenia of Glycogenosis type 1b patients with empagliflozin

Detailed description

Symptoms of glycogen storage disease type Ib (GSD Ib) include - among others - hypoglycemia, hepatomegaly and neutropenia with concomitant neutrophil dysfunction, which results in recurrent bacterial and fungal infections, and inflammatory bowel disease. At present filgrastim is the only available drug to treat neutropenia in GSD Ib patients; it stimulates neutrophil production, but doesn't restore their function. Part of GSD Ib patients doesn't respond to filgrastim treatment. The latest research results showed, that neutropenia and neutrophil dysfunction in GSD Ib patients are results of extensive accumulation of 1,5-anhydroglucitol-phosphate. Empagliflozin, a SGLT2 inhibitor, inhibits renal glucose and 1,5-anhydroglucitol reabsorption and is an effective and safe method of treatment of neutropenia in this group of patients. Empagliflozin (Jardiance®) is a drug, which is registered in Poland to treat type II diabetes in adults. The aim of our study is to evaluate the efficacy and safety of neutropenia in patients with GSD Ib with empagliflozin (Jardiance®).

Conditions

• Glucose 6 Phosphatase Deficiency

Interventions

Type	Name	Description
DRUG	Empagliflozin	dosis depending on body weight: \<20 kg 5 mg 1x/day; 20-40 kg 2 x 5 mg; \>40 kg 2 x 10 mg

Timeline

• Start date: 2021-07-01
• Primary completion: 2024-06-01
• Completion: 2025-03-01
• First posted: 2021-06-18
• Last updated: 2021-06-23

Locations

1 site across 1 country: Poland