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

NCT04138251 | Unknown Phase 2

• 1 other identifier: 2018/26DEC/492
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Glycogen Storage Disease Type I; Glucose 6 Phosphatase Deficiency

Keywords

Empaglifozin

Outcome Measures

Primary Outcomes (2)

• Empaglifozin safety (blood test-glycemia): measured by absence of hypoglycaemia due to gliflozin treatment

  Empaglifozin safety is measured by absence of hypoglycaemia due to gliflozin treatment (continuous monitoring during the first 2 days of treatment and glycemia punctual monitoring every 7 days for 2 months) (mg/dl)

  from start of treatment to 2 months post treatment

• Empaglifozin Efficacy (blood test-hemogram)

  Efficacy of drug is measured by an Increased neutrophil count as compared to pre-treatment (10exp3/µl)

  from start of treatment to 2 months post treatment

Secondary Outcomes (5)

• Empaglifozin Clinical efficacy (questionnaire)

  from start of treatment to 2 months post treatment

• Empaglifozin Biological efficacy on blood 1,5-anhydroglucitol level (blood test-LCMS)

  from start of treatment to 2 months post treatment

• Empaglifozin Biological efficacy on 1,5-anhydroglucitol-6-phosphate levels in neutrophils (blood test-LCMS)

  from start of treatment to 2 months post treatment

• Empaglifozin Clinical efficacy on urinary 1,5-anhydroglucitol excretion increase (urine test-LCMS)

  from start of treatment to 2 months post treatment

• Empaglifozin Clinical efficacy on neutrophil function (blood test)

  from start of treatment to 2 months post treatment

Study Arms (1)

Treatment

EXPERIMENTAL

Oral empaglifozin 5 mg 1x/day, increase up to 10 mg 1x/day if no 25% decrease of blood1,5-anhydroglucitol level

Drug: Empagliflozin

Interventions

Empagliflozin DRUG

oral administration of Empagliflozin

Also known as: Jardiance

Treatment

Eligibility Criteria

• Age: 1 Year - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.)

You may not qualify if:

• 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.

Sponsors & Collaborators

• Cliniques universitaires Saint-Luc- Université Catholique de Louvain: lead

Study Sites (1)

Cliniques universitaires Saint-Luc

Brussels, 1200, Belgium

RECRUITING

Related Links

• Proc Natl Acad Sci U S A. 2019 Jan 22;116(4):1241-1250. doi: 10.1073/pnas.1816143116. Epub 2019 Jan 9.

MeSH Terms

Conditions

Glycogen Storage Disease Type I

Interventions

empagliflozin

Condition Hierarchy (Ancestors)

Glycogen Storage Disease; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Xavier Stephenne, MD, PhD

  Cliniques universitaires St Luc

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Xavier Stephenne, MD, PhD

CONTACT

32 2 7641377; xavier.stephenne@uclouvain.be

Julia Versavau

CONTACT

32 2 7641933

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 20, 2019
• First Posted: October 24, 2019
• Study Start: June 20, 2019
• Primary Completion: June 30, 2020
• Study Completion: June 30, 2020
• Last Updated: October 28, 2019

Record last verified: 2019-10

Data Sharing

• IPD Sharing: Will not share

Locations

BE (1)