GLP Analogs for Diabetes in Wolfram Syndrome Patients

The recruitment status of this study is unknown because the information has not been verified recently.
Verified February 2011 by Hadassah Medical Organization.
Recruitment status was  Not yet recruiting
Information provided by:
Hadassah Medical Organization Identifier:
First received: February 22, 2011
Last updated: February 23, 2011
Last verified: February 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 Intervention
Diabetes Mellitus Associated With Genetic Syndrome
Wolfram Syndrome
Drug: Exenatide

Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment

Resource links provided by NLM:

Further study details as provided by Hadassah Medical Organization:

Primary Outcome Measures:
  • beta cell function [ Time Frame: 3 months ] [ Designated as safety issue: No ]
    IVGTT test and meal test will be performed before starting treatment with Exenetide and after 3 months of treatment.

Estimated Enrollment: 10
Study Start Date: March 2011
Estimated Primary Completion Date: March 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Exenatide Drug: Exenatide


Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01302327

Contact: Arik Tzukert, DMD 00 972 2 6776095
Contact: Hadas Lemberg, PhD 00 972 2 6777572

Sponsors and Collaborators
Hadassah Medical Organization
Principal Investigator: Gil Leibowitz, MD Hadassah Medical Organization
  More Information

Responsible Party: prof. Gil Lewbowitz, Hadassah medical center Identifier: NCT01302327     History of Changes
Other Study ID Numbers: wolfram-HMO-CTIL 
Study First Received: February 22, 2011
Last Updated: February 23, 2011
Health Authority: Israel: Ministry of Health

Keywords provided by Hadassah Medical Organization:
Wolfram syndrome
Diabetes mellitus
GLP-1 analog
beta cell function

Additional relevant MeSH terms:
Wolfram Syndrome
Diabetes Mellitus
Abnormalities, Multiple
Congenital Abnormalities
Cranial Nerve Diseases
Deaf-Blind Disorders
Diabetes Insipidus
Diabetes Mellitus, Type 1
Ear Diseases
Endocrine System Diseases
Eye Diseases
Eye Diseases, Hereditary
Genetic Diseases, Inborn
Glucose Metabolism Disorders
Hearing Disorders
Hearing Loss
Heredodegenerative Disorders, Nervous System
Kidney Diseases
Metabolic Diseases
Nervous System Diseases
Neurodegenerative Diseases
Neurologic Manifestations
Optic Atrophies, Hereditary
Optic Atrophy
Optic Nerve Diseases
Otorhinolaryngologic Diseases processed this record on May 26, 2016