The assessment of effectiveness and safeness of utilizing empagliflozin in the treatment of neutropenia of patients suffering from glycogenolysis type Ib

Phase 1

• Conditions: neutropenia and / or abnormal neutrophil function in glycogenosis I b; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: EUCTR2021-000580-78-PL
• Lead Sponsor: The Children’s Memorial Health Institute

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-04-19
• Last Update Posted: 14 March 2022

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

The patients meeting all the criteria listed below will be included into the research:
- men and women
- older than 4 weeks
- biochemically and / or molecularly diagnosed glycogenosis Ib with neutropenia and / or abnormal NADPH oxidase activity in neutrophils
- expressing voluntary and informed consent by a statutory representative in the case of children below 13 years of age, a statutory representative and a patient in the case of children above 13 years of age and by a patient himself in the case of adult patients.

Are the trial subjects under 18? yes
Number of subjects for this age range: 20
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 4
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

Exclusion Criteria

5.The following patients will not be included into the research:
- patients who are suspected of not cooperating, including the patients not arriving for follow-up examinations or not following dietary recommendations;
- suffering from chronic kidney disease with Estimated Glomerular Filtration Rate < 60 ml/min/1.73 m2
- suffering from urinary tract infection at the time of qualifying for the research (time criterion at the time of inclusion into the research, until the urinary tract infection treatment is concluded),
- participating in other clinical trials (grace period: 6 months counting from the conclusion of participation in other trials to the date of signing the Informed Consent Form),
- participating in other forms of medicinal experiments apart from the experimental
empagliflozin treatment (grace period: 24 months counting from the date of concluding participation in the experiment to the date of signing the Informed Consent Form).
- allergic to the administered drugs
- pregnant and breast-feeding women
- patients who did not give consent for participation in the research

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified