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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT04138251
• Date of registration: 20/06/2019
• Prospective Registration: Yes
• Primary sponsor: Cliniques universitaires Saint-Luc- Université Catholique de Louvain
• Public title: Safety, Efficacy Evaluation of Empagliflozin Administration for Neutropenia in Glycogenosis Type 1b and G6PC3 Deficiency GLYCO-1B
• Scientific title: Evaluation of the Safety and Efficacy of Empagliflozin Administration as a Treatment for Neutropenia in Patients With Glycogenosis Type 1b and G6PC3 Deficiency
• Date of first enrolment: June 20, 2019
• Target sample size: 5
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/show/NCT04138251
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Belgium

Contacts

• Name: Xavier Stephenne, MD, PhD

• Name: Xavier Stephenne, MD, PhD

• Name: Xavier Stephenne, MD, PhD
• Affiliation: Cliniques universitaires St Luc

Key inclusion & exclusion 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.



Age minimum: 1 Year
Age maximum: 18 Years
Gender: All

Health Condition(s) or Problem(s) studied

Glycogen Storage Disease Type I

Glucose 6 Phosphatase Deficiency

Intervention(s)

Drug: Empagliflozin

Primary Outcome(s)

Empaglifozin Efficacy (blood test-hemogram) [Time Frame: from start of treatment to 2 months post treatment]

Empaglifozin safety (blood test-glycemia): measured by absence of hypoglycaemia due to gliflozin treatment [Time Frame: from start of treatment to 2 months post treatment]

Secondary Outcome(s)

Empaglifozin Clinical efficacy (questionnaire) [Time Frame: from start of treatment to 2 months post treatment]

Empaglifozin Biological efficacy on blood 1,5-anhydroglucitol level (blood test-LCMS) [Time Frame: from start of treatment to 2 months post treatment]

Empaglifozin Biological efficacy on 1,5-anhydroglucitol-6-phosphate levels in neutrophils (blood test-LCMS) [Time Frame: from start of treatment to 2 months post treatment]

Empaglifozin Clinical efficacy on neutrophil function (blood test) [Time Frame: from start of treatment to 2 months post treatment]

Empaglifozin Clinical efficacy on urinary 1,5-anhydroglucitol excretion increase (urine test-LCMS) [Time Frame: from start of treatment to 2 months post treatment]

Secondary ID(s)

2018/26DEC/492

Source(s) of Monetary Support

Please refer to primary and secondary sponsors