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

Sponsor:

Information provided by:

Hadassah Medical Organization

ClinicalTrials.gov Identifier:

NCT01302327

First received: February 22, 2011

Last updated: February 23, 2011

Last verified: February 2011

History of Changes

Purpose

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:

Genetics Home Reference related topics: Lenz microphthalmia syndrome neurohypophyseal diabetes insipidus nonsyndromic deafness oculofaciocardiodental syndrome Pendred syndrome type 1 diabetes Wolfram syndrome

Drug Information available for: Exenatide

Genetic and Rare Diseases Information Center resources: Neurogenic Diabetes Insipidus Wolfram Syndrome

U.S. FDA Resources

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 Exenatide

Eligibility


Ages Eligible for Study:	18 Years and older
Genders Eligible for Study:	Both
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

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 ClinicalTrials.gov identifier: NCT01302327

Contacts


Contact: Arik Tzukert, DMD	00 972 2 6776095	arik@hadassah.org.il
Contact: Hadas Lemberg, PhD	00 972 2 6777572	lhadas@hadassah.org.il

Sponsors and Collaborators

Hadassah Medical Organization

Investigators


Principal Investigator:	Gil Leibowitz, MD	Hadassah Medical Organization

More Information

No publications provided


Responsible Party:	prof. Gil Lewbowitz, Hadassah medical center
ClinicalTrials.gov 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 Exenatide GLP-1 analog beta cell function

Additional relevant MeSH terms:


Diabetes Mellitus Syndrome Wolfram Syndrome Abnormalities, Multiple Blindness Congenital Abnormalities Cranial Nerve Diseases Deaf-Blind Disorders Deafness Diabetes Insipidus Diabetes Mellitus, Type 1 Disease 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

ClinicalTrials.gov processed this record on February 27, 2015

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