Prospective Cohort Study of Children With GSD1b Receiving Empagliflozin

Sponsor
Hong Kong Children's Hospital
Study ID
NCT04986735
Status
Unknown

Conditions

  • Glycogen Storage Disease Type IB

Eligibility Criteria

Sex
ALL
Age
6 Months - 18 Years
Healthy Volunteers
Not accepted

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.

Study Details

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

Key Dates

Start date
Aug 8, 2021
Status verified
Aug 2021
Primary completion
Dec 31, 2022
Completion
Dec 31, 2023

Study Design

Enrollment
11 participants (estimated)

Primary Outcome Measure

Efficacy of empagliflozin - usage of granulocyte colony stimulating factor (GCSF) [ Time Frame: from the start to the 52nd week of empagliflozin treatment ]

Central Contacts

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