NCT01302327

GLP Analogs for Diabetes in Wolfram Syndrome Patients

• Status: Withdrawn
• Phase: N/A
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: Hadassah Medical Organization · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

Wolfram syndrome, also referred to as DIDMOAD (diabetes insipidus, diabetes mellitus, optic atrophy and deafness) is a genetic syndrome characterized by beta-cell dysfunction and apoptosis leading to diabetes, neurodegeneration and psychiatric illness. Accumulating evidence indicates that beta-cell failure and neuronal cell dysfunction in Wolfram's syndrome results from a high level of ER stress in affected cells. The current treatment of Wolfram syndrome is insulin, which fails to prevent the progression of beta-cell failure. Several studies showed that GLP-1 analogs are very effective in protecting beta-cells from ER stress. Herein, the investigators suggest studying the impact of GLP-1 analogs in the treatment of patients with Wolfram syndrome. The investigators will Study the effects of GLP-1 analog (Exanatide) on beta-cell function and glycemic control of patients with Wolfram syndrome. Evaluation of beta cell function will be done by performing meal test and IVGTT test before starting GLP-1 therapy, and after 3 month of treatment.

Conditions

• Diabetes Mellitus Associated With Genetic Syndrome
• Wolfram Syndrome

Interventions

Type	Name	Description
DRUG	Exenatide	Exenatide

Timeline

• Start date: 2011-03-01
• Primary completion: 2013-03-01
• Completion: 2013-03-01
• First posted: 2011-02-24
• Last updated: 2022-03-29