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

Not Applicable

Recruiting

• Conditions: Glycogen Storage Disease type 1b; Carbohydrate Metabolism, Inborn Errors; D005953

• Registration Number: JPRN-jRCTs071210064
• Lead Sponsor: akanishi Koichi

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-09-24
• Last Update Posted: 17 October 2023

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 5

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

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

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
umber of neutrophils in the blood

Secondary Outcome Measures

Name	Time	Method
blood glucose level<br>Properties of skin, mucous membranes and stool<br>Liver and spleen size<br>Test drug safety