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. |
ClinicalTrials.gov 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Withdrawn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
|
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
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 |
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 |

Arm | Intervention/treatment |
---|---|
Experimental: Exenatide |
Drug: Exenatide
Exenatide |
- 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.

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, Learn About Clinical Studies.
Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- 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)
- Age >18 years
- Duration of diabetes of <10 years.
Exclusion Criteria:
- pregnant women
- patients who are unable to give inform consent.

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 ClinicalTrials.gov identifier (NCT number): NCT01302327
Principal Investigator: | Gil Leibowitz, MD | Hadassah Medical Organization |
Responsible Party: | Adar Zinger, Physician, Hadassah Medical Organization |
ClinicalTrials.gov 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 |
Wolfram syndrome Diabetes mellitus Exenatide GLP-1 analog beta cell function |
Wolfram Syndrome Diabetes Mellitus Syndrome Disease Pathologic Processes Glucose Metabolism Disorders Metabolic Diseases Endocrine System Diseases Deaf-Blind Disorders Deafness 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 Blindness Vision Disorders Eye Diseases, Hereditary Eye Diseases Diabetes Insipidus Kidney Diseases Urologic Diseases |