GLP Analogs for Diabetes in Wolfram Syndrome Patients
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|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||Phase|
|Diabetes Mellitus Associated With Genetic Syndrome Wolfram Syndrome||Drug: Exenatide||Not Applicable|
|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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01302327
|Contact: Arik Tzukert, DMD||00 972 2 email@example.com|
|Contact: Hadas Lemberg, PhD||00 972 2 firstname.lastname@example.org|
|Principal Investigator:||Gil Leibowitz, MD||Hadassah Medical Organization|