GLP Analogs for Diabetes in Wolfram Syndrome Patients

Brief Title

GLP Analogs for Diabetes in Wolfram Syndrome Patients


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.
    



Study Type

Interventional


Primary Outcome

beta cell function


Condition

Diabetes Mellitus Associated With Genetic Syndrome

Intervention

Exenatide

Study Arms / Comparison Groups

 Exenatide
Description:  

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

10

Start Date

March 2011


Primary Completion Date

March 2013

Eligibility Criteria

        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.
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Gil Leibowitz, MD, 00 972 2 6776095, [email protected]



Administrative Informations


NCT ID

NCT01302327

Organization ID

wolfram-HMO-CTIL



Study Sponsor

Hadassah Medical Organization


Study Sponsor

Gil Leibowitz, MD, Principal Investigator, Hadassah Medical Organization


Verification Date

February 2011