Safety, Efficacy Evaluation of Empagliflozin Administration for Neutropenia in Glycogenosis Type 1b and G6PC3 Deficiency

• Status: Unknown status
• Trial end date: 2020-06-30
• Target enrollment: 0
• Participant gender: All

Summary

Treatment of neutropenia of G6PC3 and Glycogenosis type 1b patients with empagliflozin

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Cliniques universitaires Saint-Luc- Université Catholique de Louvain
• Treatments: Empagliflozin

Criteria

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.