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
  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:


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:

  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 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 Insipidus
Diabetes Mellitus
Wolfram Syndrome
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
Kidney Diseases
Urologic Diseases
Abnormalities, Multiple
Congenital Abnormalities
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on July 24, 2014