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GLP Analogs for Diabetes in Wolfram Syndrome Patients

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01302327
Recruitment Status : Withdrawn (Very rare disease, we were unable to recruit patients)
First Posted : February 24, 2011
Last Update Posted : March 29, 2022
Information provided by (Responsible Party):
Adar Zinger, 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.

Condition or disease Intervention/treatment Phase
Diabetes Mellitus Associated With Genetic Syndrome Wolfram Syndrome Drug: Exenatide Not Applicable

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Study Start Date : March 1, 2011
Estimated Primary Completion Date : March 1, 2013
Actual Study Completion Date : March 1, 2013

Resource links provided by the National Library of Medicine

Drug Information available for: Exenatide

Arm Intervention/treatment
Experimental: Exenatide Drug: Exenatide

Primary Outcome Measures :
  1. 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.

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

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 identifier (NCT number): NCT01302327

Sponsors and Collaborators
Hadassah Medical Organization
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Principal Investigator: Gil Leibowitz, MD Hadassah Medical Organization
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Adar Zinger, Physician, Hadassah Medical Organization Identifier: NCT01302327    
Other Study ID Numbers: wolfram-HMO-CTIL
First Posted: February 24, 2011    Key Record Dates
Last Update Posted: March 29, 2022
Last Verified: March 2022
Keywords provided by Adar Zinger, Hadassah Medical Organization:
Wolfram syndrome
Diabetes mellitus
GLP-1 analog
beta cell function
Additional relevant MeSH terms:
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Wolfram Syndrome
Diabetes Mellitus
Pathologic Processes
Glucose Metabolism Disorders
Metabolic Diseases
Endocrine System Diseases
Deaf-Blind Disorders
Hearing Loss
Hearing Disorders
Ear Diseases
Otorhinolaryngologic Diseases
Optic Atrophies, Hereditary
Optic Atrophy
Optic Nerve Diseases
Cranial Nerve Diseases
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Sensation Disorders
Neurologic Manifestations
Vision Disorders
Eye Diseases, Hereditary
Eye Diseases
Diabetes Insipidus
Kidney Diseases
Urologic Diseases