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Recruitment Status : Withdrawn (Very rare disease, we were unable to recruit patients)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

Sponsor:

Information provided by (Responsible Party):

Adar Zinger, Hadassah Medical Organization

Study Description

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

Study Design

Layout table for study information

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

MedlinePlus Genetics related topics: Wolfram syndrome

Drug Information available for: Exenatide

Genetic and Rare Diseases Information Center resources: Wolfram Syndrome

U.S. FDA Resources

Arms and Interventions

Arm	Intervention/treatment
Experimental: Exenatide	Drug: Exenatide Exenatide

Outcome Measures

Primary Outcome Measures :

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.

Eligibility Criteria

Information from the National Library of Medicine

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.

Layout table for eligibility information

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

Criteria

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.

Contacts and Locations

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 ClinicalTrials.gov identifier (NCT number): NCT01302327

Sponsors and Collaborators

Hadassah Medical Organization

Investigators

Layout table for investigator information

Principal Investigator:	Gil Leibowitz, MD	Hadassah Medical Organization

More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Danielpur L, Sohn YS, Karmi O, Fogel C, Zinger A, Abu-Libdeh A, Israeli T, Riahi Y, Pappo O, Birk R, Zangen DH, Mittler R, Cabantchik ZI, Cerasi E, Nechushtai R, Leibowitz G. GLP-1-RA Corrects Mitochondrial Labile Iron Accumulation and Improves β-Cell Function in Type 2 Wolfram Syndrome. J Clin Endocrinol Metab. 2016 Oct;101(10):3592-3599. Epub 2016 Jul 26.

Layout table for additonal information

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

Keywords provided by Adar Zinger, Hadassah Medical Organization:


Wolfram syndrome Diabetes mellitus Exenatide GLP-1 analog beta cell function

Additional relevant MeSH terms:

Layout table for MeSH terms

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

To Top