GLP Analogs for Diabetes in Wolfram Syndrome Patients

Wolfram Syndrome Clinical Trial

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT01302327
• Other study ID #: wolfram-HMO-CTIL
• Status: Withdrawn
• Phase: N/A
• Start date: March 1, 2011
• Est. completion date: March 1, 2013

Study information

• Verified date: March 2022
• Source: Hadassah Medical Organization
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: March 1, 2013
• Est. primary completion date: March 1, 2013
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Diabetes Mellitus
• Diabetes Mellitus Associated With Genetic Syndrome
• Syndrome
• Wolfram Syndrome

Intervention

Drug:
• Exenatide
Exenatide

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Hadassah Medical Organization

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	beta cell function	IVGTT test and meal test will be performed before starting treatment with Exenetide and after 3 months of treatment.	3 months