2024-512840-52-00
Active, not recruiting
Phase 2
Evaluation of the efficacy and safety of empagliflozin in the treatment of neutropenia in patients with glycogenosis Ib. EMPAtia.
Instytut Pomnik Centrum Zdrowia Dziecka2 sites in 1 country20 target enrollmentStarted: December 2, 2024Last updated:
Overview
- Phase
- Phase 2
- Status
- Active, not recruiting
- Sponsor
- Instytut Pomnik Centrum Zdrowia Dziecka
- Enrollment
- 20
- Locations
- 2
- Primary Endpoint
- The primary endpoint will be an assessment of the safety and tolerability of empagliflozin, expressed as the type and frequency of adverse reactions throughout the study period. Safety data will be collected at all visits and will be related to possible adverse reactions to empagliflozin as described in the summary of product characteristics.
Overview
Brief Summary
The primary objective of the study is to evaluate the safety and tolerability of neutropenia treatment with empagliflozin in patients with GSD Ib
Eligibility Criteria
- Ages
- 0 years to 65+ years (18-64 Years, 65+ Years, 0-17 Years)
- Accepts Healthy Volunteers
- Yes
Inclusion Criteria
- •Inclusion criteria for the study Patients who meet all of the following criteria will be included in the study: - males and females - age of 4 weeks or older - biochemically and/or molecularly diagnosed Ib glycogenosis with neutropenia and/or abnormal NADPH oxidase activity in neutrophils - the expression of voluntary informed consent by the legal representative for children < 13 years of age, the legal representative and the patient for children >=13 years of age, and the patient himself for adult patients.
Exclusion Criteria
- •Exclusion criteria for the study The following criteria exclude a patient from eligibility for the study: - risk of non-cooperation, including the risk of non-adherence to follow-up and non-adherence to dietary recommendations; - chronic kidney disease with eGFR < 60 ml/min/1.73 m2 - urinary tract infection (time criterion at the time of inclusion in the study, pending completion of treatment for UTI), - participation in another clinical trial (withdrawal period: 6 months from the end of participation until the date of signing the Informed Consent Form), - participation in a therapeutic experiment, other than experimental treatment with empagliflozin (grace period: 24 months from the end of participation until the date of signing the Informed Consent Form) - pregnancy, breastfeeding - allergy to the administered drugs - lack of consent to participate in the study.
Outcomes
Primary Outcomes
The primary endpoint will be an assessment of the safety and tolerability of empagliflozin, expressed as the type and frequency of adverse reactions throughout the study period. Safety data will be collected at all visits and will be related to possible adverse reactions to empagliflozin as described in the summary of product characteristics.
The primary endpoint will be an assessment of the safety and tolerability of empagliflozin, expressed as the type and frequency of adverse reactions throughout the study period. Safety data will be collected at all visits and will be related to possible adverse reactions to empagliflozin as described in the summary of product characteristics.
Secondary Outcomes
- The secondary objective is to assess efficacy expressed as restoration of neutrophil numbers and function
Investigators
Mariola Modzelewska
Scientific
Instytut Pomnik Centrum Zdrowia Dziecka
Study Sites (2)
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