Efficacy of the selective SGLT2 Inhibitor for Glycogen Storage Disease Type 1b

Exploratory clinical trial of selective SGLT2 inhibitor for Glycogen Storage Disease type 1b

Status

Recruiting

Phases

Unknown

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCTs071210064

Enrollment

Registered

2021-09-24

Start date

2021-09-24

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Glycogen Storage Disease type 1b Carbohydrate Metabolism, Inborn Errors

Interventions

After taking informed consent, study items such as medical history, physical findings, imaging tests, and blood tests are performed. Participants take Empagliflozin orally from 0.1-0.3 mg/kg/day (max

D005953

Glycogen Storage Disease 1 (GSD I)

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Patients with Glycogen storage disease type 1b at the University ot the Ryukyus* 2. Patients with blood neutropenia or who need treatment with G-CSF drug 3. Frequnet diet, nighttime continuous nutrition, or similar diet is required 4. 1 week to 50 years old * This research registrants Include one case of neonatal onset with severe hypoglycemia and neutropenia that required immediate living-donor liver transplantation.

Exclusion criteria

Exclusion criteria: 1. Moderate to severe renal dysfunction 2. Patients with severe liver dysfunction 3. Pregnant woman

Design outcomes

Primary

Measure	Time frame
Number of neutrophils in the blood	—

Secondary

Measure	Time frame
blood glucose level Properties of skin, mucous membranes and stool Liver and spleen size Test drug safety	—

Contacts

Public ContactYasutsugu Chinen

Graduate School of Medicine, University of the Ryukyus

ychinen@cs.u-ryukyu.ac.jp+81-98-894-1417

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026