GLP Analogs for Diabetes in Wolfram Syndrome Patients

Status

Withdrawn

Phases

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01302327

Enrollment

Registered

2011-02-24

Start date

2011-03-01

Completion date

2013-03-01

Last updated

2022-03-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Diabetes Mellitus Associated With Genetic Syndrome, Wolfram Syndrome

Keywords

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

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.

Interventions

DRUGExenatide

Exenatide

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

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.

Design outcomes

Primary

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

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026