Safety and Efficacy of Empagliflozin in GSD1b Patients With Neutropenia

• Status: Not yet recruiting
• Trial end date: 2025-03-01
• Target enrollment: 0
• Participant gender: All

Summary

Treatment of neutropenia of Glycogenosis type 1b patients with empagliflozin

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Children's Memorial Health Institute, Poland
• Collaborator: Department of Internal Medicine, Hypertension and Vascular Diseases, The Medical University of Warsaw
• Treatments: Empagliflozin

Criteria

Inclusion Criteria:

• Minimum age 4 weeks old female Or Male

• GSD1b confirmed by genetic analysis with neutropenia and/or reduced respiratory burst

• Informed consent signed by the parents/assigns, and the recipient (>13 years old)

Exclusion Criteria:

• Risk of non-compliance

• Chronic renal diseases (eGFR < 60 ml/min/1,73 m2)

• Active urinary tract infection (temporal criterion, up to recovery)

• Participation in another clinical trial (minimum 6 months from the end of participation until the date of signing the Informed Consent Form)

• Participation in therapeutic experiment, in addition to the experimental treatment with empagliflozin (minimum 12 months from the end of participation until the date of signing the Informed Consent Form)

• Pregnancy, breastfeeding

• Allergy to Empagliflozin

• Lack of informed consent