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GLP Analogs for Diabetes in Wolfram Syndrome Patients

Primary Purpose

Diabetes Mellitus Associated With Genetic Syndrome, Wolfram Syndrome

Status
Withdrawn
Phase
Not Applicable
Locations
Study Type
Interventional
Intervention
Exenatide
Sponsored by
Hadassah Medical Organization
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Diabetes Mellitus Associated With Genetic Syndrome focused on measuring Wolfram syndrome, Diabetes mellitus, Exenatide, GLP-1 analog, beta cell function

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept 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.

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Experimental

    Arm Label

    Exenatide

    Arm Description

    Outcomes

    Primary Outcome Measures

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

    Secondary Outcome Measures

    Full Information

    First Posted
    February 22, 2011
    Last Updated
    March 14, 2022
    Sponsor
    Hadassah Medical Organization
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    1. Study Identification

    Unique Protocol Identification Number
    NCT01302327
    Brief Title
    GLP Analogs for Diabetes in Wolfram Syndrome Patients
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    March 2022
    Overall Recruitment Status
    Withdrawn
    Why Stopped
    Very rare disease, we were unable to recruit patients
    Study Start Date
    March 1, 2011 (undefined)
    Primary Completion Date
    March 1, 2013 (Anticipated)
    Study Completion Date
    March 1, 2013 (Actual)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Principal Investigator
    Name of the Sponsor
    Hadassah Medical Organization

    4. Oversight

    Data Monitoring Committee
    No

    5. 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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Diabetes Mellitus Associated With Genetic Syndrome, Wolfram Syndrome
    Keywords
    Wolfram syndrome, Diabetes mellitus, Exenatide, GLP-1 analog, beta cell function

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Not Applicable
    Interventional Study Model
    Single Group Assignment
    Masking
    None (Open Label)
    Allocation
    N/A
    Enrollment
    0 (Actual)

    8. Arms, Groups, and Interventions

    Arm Title
    Exenatide
    Arm Type
    Experimental
    Intervention Type
    Drug
    Intervention Name(s)
    Exenatide
    Intervention Description
    Exenatide
    Primary Outcome Measure Information:
    Title
    beta cell function
    Description
    IVGTT test and meal test will be performed before starting treatment with Exenetide and after 3 months of treatment.
    Time Frame
    3 months

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    18 Years
    Accepts Healthy Volunteers
    No
    Eligibility 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.
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Gil Leibowitz, MD
    Organizational Affiliation
    Hadassah Medical Organization
    Official's Role
    Principal Investigator

    12. IPD Sharing Statement

    Citations:
    PubMed Identifier
    27459537
    Citation
    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 beta-Cell Function in Type 2 Wolfram Syndrome. J Clin Endocrinol Metab. 2016 Oct;101(10):3592-3599. doi: 10.1210/jc.2016-2240. Epub 2016 Jul 26.
    Results Reference
    derived

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    GLP Analogs for Diabetes in Wolfram Syndrome Patients

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