neutropenia and / or abnormal neutrophil function in glycogenosis I b
Conditions
Interventions
Trade Name: Jardiance, empagliflozin, 10 mg film- coated tablets
Product Name: Jardiance
Pharmaceutical Form: Film-coated tablet
Sponsors
The Children’s Memorial Health Institute
Eligibility
Sex/Gender
All
Inclusion criteria
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
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary goal of the research is to assess safeness and tolerance of treating neutropenia with empagliflozin in the case of patients suffering from glycogen storage disease type Ib. ;Secondary Objective: The secondary goal is the assessment of the effectiveness expressed as restoring the count and functioning of neutrophils. The tertiary goal is reducing/stopping dosage of filgrastym and thus limiting the undesirable effects related to the treatment utilizing filgrastym. The quaternary goal will be the assessment of the metabolic control.;Primary end point(s): The primary End Point will be the assessment of safeness and tolerance of empagliflozin expressed as a type and a frequency of occurrence of undesirable reactions throughout the entire period of the research. ;Timepoint(s) of evaluation of this end point: The data pertaining to safeness will be collected during all examination appointments and will be cross-referenced with the possible undesirable effects of empagliflozin described in the characteristics of the medicinal product. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The secondary End Point will be the assessment of effectiveness of treatment of the neutropenia of patients suffering from glycogen storage disease type 1b expressed as: - the percentage of patients taking empagliflozin who achived the neutrophils count in bloodat the level of >= 500 cells/ul for the period of at least 6 months. - the percentage of patients taking empagliflozin who were discovered to display the proper level of NADPH activity of the neutrophils isolated from the peripheral blood during the final examination, - the percentage of patients taking empagliflozin who were discovered to display the decreased frequency of the bacterial and fungal infections requiring antibiotics treatments throughout the entire period of the empagliflozin treatment in comparison to the data from the period preceeding introduction of the treatment. This frequency shall be expressed as the number of antibiotic treatments/year, - the percentage of patients taking empagliflozin who were discovered to display the decreased frequency of infection-related hospitalizations throughout the entire period of the empagliflozin treatment in comparison to the data from the period preceeding introduction of the treatment. This frequency shall be expressed as the number of infection-related hospitalizations/year. - the percentage of patients taking empagliflozin who were discovered to display reduced average number of bowel discharge instances per day (calculated from the period of 7 days preceeding the most recent examination), the reduced frequency of helcosis/ulceration in the buccal cavity and the reduced concentration of the calprotectin median in feces (from the entire research period) in comparison to the data from the period preceding introduction of the treatment. The tertiary End Point will be the assessment of the possibility for reducing the filgrastym dosage expressed as: - the percentage of patients who after taking empagliflozin for a per | — |
Countries
Poland
Contacts
Public ContactDariusz Rokicki
The Children’s Memorial Health Institute
Outcome results
None listed