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GLP Analogs for Diabetes in Wolfram Syndrome Patients

NCT01302327 · Status: WITHDRAWN · Phase: NA · Type: INTERVENTIONAL

Last updated 2022-03-29

No results posted yet for this study

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.

Conditions

  • Diabetes Mellitus Associated With Genetic Syndrome
  • Wolfram Syndrome

Interventions

DRUG

Exenatide

Exenatide

Sponsors & Collaborators

  • Hadassah Medical Organization

    lead OTHER

Principal Investigators

  • Gil Leibowitz, MD · Hadassah Medical Organization

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2011-03-01
Primary Completion
2013-03-01
Completion
2013-03-01

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01302327 on ClinicalTrials.gov