Status:
UNKNOWN
Safety, Efficacy Evaluation of Empagliflozin Administration for Neutropenia in Glycogenosis Type 1b and G6PC3 Deficiency
Lead Sponsor:
Cliniques universitaires Saint-Luc- Université Catholique de Louvain
Conditions:
Glycogen Storage Disease Type I
Glucose 6 Phosphatase Deficiency
Eligibility:
All Genders
1-18 years
Phase:
PHASE2
Brief Summary
Treatment of neutropenia of G6PC3 and Glycogenosis type 1b patients with empagliflozin
Detailed Description
Ubiquitous glucose-6-phosphatase deficiency (G6PC3) and glucose-6-phosphate transporter deficiency (G6PT/SLC37A4) both cause neutropenia. Studies on a G6PC3 deficient mouse model by Dr Veiga-da-Cunha ...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
June 20 2019
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
June 30 2020
Estimated Enrollment :
5 Patients enrolled
Trial Details
Trial ID
NCT04138251
Start Date
June 20 2019
End Date
June 30 2020
Last Update
October 28 2019
Active Locations (1)
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1
Cliniques universitaires Saint-Luc
Brussels, Belgium, 1200