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 range1 Year – 18 Years
SexALL
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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.
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