Safety and Efficacy of Empagliflozin in GSD1b Patients With Neutropenia

• Register: ClinicalTrials.gov
• Last refreshed on: 28 June 2021
• Main ID: NCT04930627
• Date of registration: 11/06/2021
• Prospective Registration: Yes
• Primary sponsor: Children's Memorial Health Institute, Poland
• Public title: Safety and Efficacy of Empagliflozin in GSD1b Patients With Neutropenia EMPAtia
• Scientific title: Evaluation of Efficacy and Safety of Empagliflozin in Treatment of Neutropenia in Patients With Glycogenosis Ib
• Date of first enrolment: July 2021
• Target sample size: 20
• Recruitment status: Not yet recruiting
• URL: https://clinicaltrials.gov/show/NCT04930627
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Poland

Contacts

• Name: Magdalena Kaczor, MD

Key inclusion & exclusion 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



Age minimum: N/A
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Glucose 6 Phosphatase Deficiency

Intervention(s)

Drug: Empagliflozin

Primary Outcome(s)

Empaglifozin safety and tolerability measured by occurrence of adverse reactions [Time Frame: 2 years]

Secondary Outcome(s)

Degree of metabolic compensation [Time Frame: 2 years]

Efficacy of neutropenia treatment measured as percentage of the patients [Time Frame: 2 years]

Dosis change/withdrawal of filgrastrim [Time Frame: 2 years]

Secondary ID(s)

2021-000580-78

EMPAtia

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Department of Internal Medicine, Hypertension and Vascular Diseases, The Medical University of Warsaw