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

JRCT ID: jRCTs071210064

Registered date:24/09/2021

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Glycogen Storage Disease type 1b
• Date of first enrollment: 24/09/2021
• Target sample size: 5
• Study type: Interventional
• 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.

Outcome(s)

• Primary Outcome: Number of neutrophils in the blood

Secondary Outcome

blood 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
• Gender: Both

Include 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.

Exclude criteria

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

Related Information

• Primary Sponsor: Nakanishi Koichi