Safety and Efficacy of Empagliflozin in GSD1b Patients With Neutropenia (NCT04930627) | Clinical Trial Compass
UnknownPhase 2
Safety and Efficacy of Empagliflozin in GSD1b Patients With Neutropenia
Poland20 participantsStarted 2021-07
Plain-language summary
Treatment of neutropenia of Glycogenosis type 1b patients with empagliflozin
Who can participate
Age range
4 Weeks
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Empaglifozin safety and tolerability measured by occurrence of adverse reactions
Timeframe: 2 years
Trial details
NCT IDNCT04930627
SponsorChildren's Memorial Health Institute, Poland