Safety, Efficacy Evaluation of Empagliflozin Administration for Neutropenia in Glycogenosis Type … (NCT04138251) | Clinical Trial Compass
UnknownPhase 2
Safety, Efficacy Evaluation of Empagliflozin Administration for Neutropenia in Glycogenosis Type 1b and G6PC3 Deficiency
Belgium5 participantsStarted 2019-06-20
Plain-language summary
Treatment of neutropenia of G6PC3 and Glycogenosis type 1b patients with empagliflozin
Who can participate
Age range
1 Year – 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:
* Glycogenosis type 1b confirmed by biochemical analyzes and / or genetic analysis. These patients with Glycogenosis must have had a liver transplant
* Alternatively, G6PC3 deficiency confirmed by genetic analysis
* Age 1 to 18 years old female or male
* Informed consent signed by the recipient and / or parents / assigns.
* Information and agreement of the referring medical team.
* A Negative Blood Pregnancy Test at the time of screening and a negative urinary pregnancy test at Day 1 of the protocol are required for female with child bearing potential.
* Sexually active patients should use an effective method of contraception throughout the duration of the study and up to 7 days after the last dose of Empaglifozine. (The combination of a hormonal method and a barrier method; Two barrier methods, the male condom being one of these two methods;Use intrauterine device or tubal ligation;-A total sex abstinence.)
Exclusion Criteria:
* Presence of advanced fibrosis (Metavir F4) or cirrhosis.
* Impossibility of long-term and / or non-compliance monitoring.
* Other medical problems which, in the opinion of the physicians in charge of the patient, would constitute a contraindication to the procedure.
* Sexually active patients who do not consent to use effective contraception during the study.
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.
1Since this is a Phase 2 trial for a rare condition like Glycogen Storage Disease Type 1b or G6PC3 deficiency, what does that phase mean for how much is already known about empagliflozin's safety and effectiveness in people with my specific diagnosis?
2One of the main safety concerns being measured in this trial is hypoglycemia caused by the gliflozin treatment itself — given that my condition already affects blood sugar regulation, how would you monitor me for dangerous drops in blood sugar if I were to participate?
3The trial's recruitment status is listed as 'unknown,' so do you know whether this study is still actively enrolling patients, and if not, are there other similar trials or compassionate use options worth exploring?
4The trial measures efficacy through a blood count test (hemogram), which suggests it's looking at effects on neutropenia — how significant is my neutropenia right now, and would participating in a study focused on that problem make sense compared to what's currently being done to manage it?
5Are there any standard or established treatments for the neutropenia associated with my type of glycogen storage disease that I should consider first before looking into an experimental option like empagliflozin?
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 (blood test-glycemia): measured by absence of hypoglycaemia due to gliflozin treatment
Timeframe: from start of treatment to 2 months post treatment
2
Empaglifozin Efficacy (blood test-hemogram)
Timeframe: from start of treatment to 2 months post treatment
Trial details
NCT IDNCT04138251
SponsorCliniques universitaires Saint-Luc- Université Catholique de Louvain