Prospective Cohort Study of Children With GSD1b Receiving Empagliflozin (NCT04986735) | Clinical Trial Compass
UnknownNot Applicable
Prospective Cohort Study of Children With GSD1b Receiving Empagliflozin
Hong Kong11 participantsStarted 2021-08-08
Plain-language summary
This is a prospective cohort study of children with GSD1b to evaluate their outcome after using empagliflozin for neutrophil defects.
Who can participate
Age range
6 Months – 18 Years
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:
\- Subject (aged 6 months to 18 years) is enzymatically/genetically confirmed to have GSD 1b and has been on regular GCSF treatment for \>= 1 month
Exclusion Criteria:
* Subject fails to provide relevant background medical information, or comply with all requirements of the clinical trial, or sign the informed consent
* Subject has any co-morbidity or condition that could increase the risk of empagliflozin treatment (e.g. renal failure with eGFR \<30 mL/min/1.73m2 or requiring dialysis, diabetes requiring insulin \&/or oral hypoglycemic agents, dyslipidemia requiring pharmacological intervention)
* Subject is pregnant, or a sexually active female who does not consent to use effective contraception during the study
* History of liver transplantation is NOT an exclusion criterium
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
Efficacy of empagliflozin - usage of granulocyte colony stimulating factor (GCSF)
Timeframe: from the start to the 52nd week of empagliflozin treatment