Prospective Cohort Study of Children With GSD1b Receiving Empagliflozin

NCT04986735 | Unknown DMC

• 1 other identifier: KWOK-HKCH-GSD1-EMPA
• Study Type: observational
• Target: 11
• Locations: 1 country
• Sites: 1

Brief Summary

This is a prospective cohort study of children with GSD1b to evaluate their outcome after using empagliflozin for neutrophil defects.

Conditions

Glycogen Storage Disease Type IB

Outcome Measures

Primary Outcomes (1)

• Efficacy of empagliflozin - usage of granulocyte colony stimulating factor (GCSF)

  Dosage and frequency of administration of GCSF

  from the start to the 52nd week of empagliflozin treatment

Secondary Outcomes (8)

• Efficacy of empagliflozin - neutrophil number and function

  from the start to the 52nd week of empagliflozin treatment

• Efficacy of empagliflozin - bowel manifestations

  from the start to the 52nd week of empagliflozin treatment

• Efficacy of empagliflozin - frequency of infections

  from the start to the 52nd week of empagliflozin treatment

• Efficacy of empagliflozin - biochemical improvement

  from the start to the 52nd week of empagliflozin treatment

• General metabolic control - GSD1b metabolic & imaging profile, concomitant interventions

  from the start to the 52nd week of empagliflozin treatment

Interventions

Empagliflozin DRUG

All subjects will have a baseline assessment and be prospectively followed up for 52 weeks to examine their outcome after receiving empagliflozin for neutropenia and neutrophil dysfunction.

Also known as: Jardiance

Eligibility Criteria

• Age: 6 Months - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Probability Sample

Study Population

GSD 1b patients (aged 6 months to 18 years) with diagnosis confirmed by enzymatic and/or genetic analysis, and has been on regular GCSF treatment for \>= 1 month

You may qualify if:

• \- Subject (aged 6 months to 18 years) is enzymatically/genetically confirmed to have GSD 1b and has been on regular GCSF treatment for \>= 1 month

You may not qualify if:

• Subject fails to provide relevant background medical information, or comply with all requirements of the clinical trial, or sign the informed consent
• Subject has any co-morbidity or condition that could increase the risk of empagliflozin treatment (e.g. renal failure with eGFR \<30 mL/min/1.73m2 or requiring dialysis, diabetes requiring insulin \&/or oral hypoglycemic agents, dyslipidemia requiring pharmacological intervention)
• Subject is pregnant, or a sexually active female who does not consent to use effective contraception during the study

Sponsors & Collaborators

• Hong Kong Children's Hospital: lead

Study Sites (1)

Hong Kong Children's Hospital

Hong Kong, Hong Kong

RECRUITING

MeSH Terms

Conditions

Glycogen Storage Disease IB

Interventions

empagliflozin

Study Officials

• Mei Kwun Kwok

  Hong Kong Children's Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

MEI KWUN KWOK, MB,BS

CONTACT

852-57413216; kwokmk@ha.org.hk

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Consultant

Study Record Dates

• First Submitted: July 27, 2021
• First Posted: August 3, 2021
• Study Start: August 8, 2021
• Primary Completion: December 31, 2022
• Study Completion: December 31, 2023
• Last Updated: August 16, 2021

Record last verified: 2021-08

Data Sharing

• IPD Sharing: Will not share

Locations

HK (1)