GLP Analogs for Diabetes in Wolfram Syndrome Patients
|ClinicalTrials.gov Identifier: NCT01302327|
Recruitment Status : Unknown
Verified February 2011 by Hadassah Medical Organization.
Recruitment status was: Not yet recruiting
First Posted : February 24, 2011
Last Update Posted : February 24, 2011
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.
|Condition or disease||Intervention/treatment|
|Diabetes Mellitus Associated With Genetic Syndrome Wolfram Syndrome||Drug: Exenatide|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Study Start Date :||March 2011|
|Estimated Primary Completion Date :||March 2013|
- beta cell function [ Time Frame: 3 months ]IVGTT test and meal test will be performed before starting treatment with Exenetide and after 3 months of treatment.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01302327
|Contact: Arik Tzukert, DMD||00 972 2 firstname.lastname@example.org|
|Contact: Hadas Lemberg, PhD||00 972 2 email@example.com|
|Principal Investigator:||Gil Leibowitz, MD||Hadassah Medical Organization|