GLP Analogs for Diabetes in Wolfram Syndrome Patients

Not Applicable

Withdrawn

• Conditions: Wolfram Syndrome; Diabetes Mellitus Associated With Genetic Syndrome
• Interventions: Drug: Exenatide

• Registration Number: NCT01302327
• Lead Sponsor: Hadassah Medical Organization

Brief 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2011-02-22
• Study First Submitted QC: 2011-02-23
• Study First Posted: 2011-02-24
• Study Start: 2011-03-01
• Primary Completion: 2013-03-01
• Study Completion: 2013-03-01
• Status Verified: 2022-03
• Last Update Submitted: 2022-03-14
• Last Update Posted: 2022-03-29

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

1. Genetic or definitive clinical diagnosis of Wolfram's syndrome including: diabetes mellitus, optic atrophy and at least one additional neurological dysfunction (diabetes insipidus, sensorineural deafness, neurogenic bladder or other type of autonomic or peripheral neuropathy)
2. Age >18 years
3. Duration of diabetes of <10 years.

Exclusion Criteria

1. pregnant women
2. patients who are unable to give inform consent.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Exenatide	Exenatide	-

Primary Outcome Measures

Name	Time	Method
beta cell function	3 months	IVGTT test and meal test will be performed before starting treatment with Exenetide and after 3 months of treatment.