Safety and Efficacy of Empagliflozin in GSD1b Patients With Neutropenia

NCT04930627 | Unknown Phase 2

• 2 other identifiers: EMPAtia2021-000580-78
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

Treatment of neutropenia of Glycogenosis type 1b patients with empagliflozin

Conditions

Glucose 6 Phosphatase Deficiency

Outcome Measures

Primary Outcomes (1)

• Empaglifozin safety and tolerability measured by occurrence of adverse reactions

  Empaglifozin saftey and tolerability measured by occurrence of adverse reactions

  2 years

Secondary Outcomes (3)

• Efficacy of neutropenia treatment measured as percentage of the patients

  2 years

• Dosis change/withdrawal of filgrastrim

  2 years

• Degree of metabolic compensation

  2 years

Study Arms (1)

oral administration of Empagliflozin

EXPERIMENTAL

Drug: Empagliflozin

Interventions

Empagliflozin DRUG

dosis depending on body weight: \<20 kg 5 mg 1x/day; 20-40 kg 2 x 5 mg; \>40 kg 2 x 10 mg

Also known as: Jardiance

oral administration of Empagliflozin

Eligibility Criteria

• Age: 4 Weeks+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Minimum age 4 weeks old female Or Male
• GSD1b confirmed by genetic analysis with neutropenia and/or reduced respiratory burst
• Informed consent signed by the parents/assigns, and the recipient (\>13 years old)

You may not qualify if:

• Risk of non-compliance
• Chronic renal diseases (eGFR \< 60 ml/min/1,73 m2)
• Active urinary tract infection (temporal criterion, up to recovery)
• Participation in another clinical trial (minimum 6 months from the end of participation until the date of signing the Informed Consent Form)
• Participation in therapeutic experiment, in addition to the experimental treatment with empagliflozin (minimum 12 months from the end of participation until the date of signing the Informed Consent Form)
• Pregnancy, breastfeeding
• Allergy to Empagliflozin
• Lack of informed consent

Sponsors & Collaborators

• Children's Memorial Health Institute, Poland: lead
• Department of Internal Medicine, Hypertension and Vascular Diseases, The Medical University of Warsaw: collaborator

Study Sites (1)

The Children's Memorial Health Institute

Warsaw, 04-730, Poland

Location

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 Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Pricipal Investigator

Study Record Dates

• First Submitted: June 11, 2021
• First Posted: June 18, 2021
• Study Start: July 1, 2021
• Primary Completion: June 1, 2024
• Study Completion: March 1, 2025
• Last Updated: June 23, 2021

Record last verified: 2021-06

Locations

PL (1)