NIPH Clinical Trials Search

JRCT ID: jRCTs071210064

Registered date:24/09/2021

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

Basic Information

Recruitment status Recruiting
Health condition(s) or Problem(s) studiedGlycogen Storage Disease type 1b
Date of first enrollment24/09/2021
Target sample size5
Countries of recruitment
Study typeInterventional
Intervention(s)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 5mg) to 0.7mg/kg/day (max 20mg). We obtain an appropriate doase while paying attention to side effects such as neutrophil count / blood glucose trends and urinary tract infections. During the course, imaging tests, development tests, and general tests will be performed to evaluate efficacy and safety.


Primary OutcomeNumber of neutrophils in the blood
Secondary Outcomeblood glucose level Properties of skin, mucous membranes and stool Liver and spleen size Test drug safety

Key inclusion & exclusion criteria

Age minimum>= 1weeks old
Age maximum< 50age old
Include criteria1. 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.
Exclude criteria1. Moderate to severe renal dysfunction 2. Patients with severe liver dysfunction 3. Pregnant woman

Related Information


Public contact
Name Yasutsugu Chinen
Address 207, Uehara, Nishihara, Okinawa, Japan Okinawa Japan 903-0215
Telephone +81-98-895-1154
Affiliation Graduate School of Medicine, University of the Ryukyus
Scientific contact
Name Koichi Nakanishi
Address 207, Uehara, Nishihara, Okinawa, Japan Okinawa Japan 903-0215
Telephone +81-98-895-1154
Affiliation University of the Ryukyus Hospital