GLP Analogs for Diabetes in Wolfram Syndrome Patients
Recruitment status was: Not yet recruiting
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.
Diabetes Mellitus Associated With Genetic Syndrome
|Study Design:||Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
- beta cell function [ Time Frame: 3 months ] [ Designated as safety issue: No ]IVGTT test and meal test will be performed before starting treatment with Exenetide and after 3 months of treatment.
|Study Start Date:||March 2011|
|Estimated Primary Completion Date:||March 2013 (Final data collection date for primary outcome measure)|
Please refer to this study by its ClinicalTrials.gov identifier: NCT01302327
|Principal Investigator:||Gil Leibowitz, MD||Hadassah Medical Organization|