Efficacy and Safety of Empagliflozin in GSD-Ib Patients

Sponsor
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Study ID
NCT05960617
Phase
PHASE2
Status
Withdrawn

Conditions

  • Glycogen Storage Disease Type IB

Eligibility Criteria

Sex
ALL
Age
1 Year - 50 Years
Healthy Volunteers
Not accepted

Interventions

  • Empagliflozin — DRUG
    Oral administration of Empagliflozin: The starting dose was 0.3 mg/kg/day in 2 divided doses for 3 months. If the subject had an absolute neutrophil count \> 1.0 × 10\^9/L and clinical improvement (decreased number of infections and/or decreased IBD activity within 3 months), the maintenance dose was maintained. If the subject had an absolute neutrophil count \< 1.0 × 10\^9/L but clinical improvement (decreased number of infections and/or decreased IBD activity within 3 months), the maintenance dose was maintained and reassessed 1 month later. If the subject had an absolute neutrophil count \< 1.0 × 10\^9/L and no clinical improvement (no change in number of infections and/or no change in IBD activity within 3 months), the dose was increased by 0.1 mg/kg/day and reassessed 3 months later. Assessments were then performed every 3 months with the same dose modification criteria as above.

Study Details

Empagliflozin Treatment of GSD-1b patients

Key Dates

Start date
Jul 15, 2023
Status verified
Jun 2023
Primary completion
Jun 30, 2024
Completion
Dec 31, 2024

Study Design

Enrollment
0 participants (actual)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Oral administration of Empagliflozin
    All subjects will have a baseline assessment and be prospectively followed up for 12 months to examine their outcome after receiving empagliflozin.

Primary Outcome Measure

Change from Baseline in Absolute neutrophil count at 1 year [ Time Frame: 1 year ]

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