GLP Analogs for Diabetes in Wolfram Syndrome Patients

Wolfram Syndrome Clinical Trial

Status Withdrawn

Clinical Trial 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.

Clinical Trial Description

n/a

Related Conditions & MeSH terms

• Diabetes Mellitus
• Diabetes Mellitus Associated With Genetic Syndrome
• Syndrome
• Wolfram Syndrome

• NCT number: NCT01302327
• Study type: Interventional
• Source: Hadassah Medical Organization
• Status: Withdrawn
• Phase: N/A
• Start date: March 1, 2011
• Completion date: March 1, 2013