Diabetes Mellitus Associated With Genetic SyndromeNCT01302327
GLP Analogs for Diabetes in Wolfram Syndrome Patients
A study testing Exenatide in people with Diabetes Mellitus Associated With Genetic Syndrome and Wolfram Syndrome, run by Hadassah Medical Organization.
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Diabetes Mellitus Associated With Genetic Syndrome, Wolfram Syndrome
- What is being tested?
- Exenatide
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 1 Mar 2011to 1 Mar 2013
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- pregnant women
- patients who are unable to give inform consent.
About this study
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.
- Being tested
- Exenatide
- Keywords
- Wolfram syndrome, Diabetes mellitus, Exenatide, GLP-1 analog, beta cell function
- Medical subject headings
- Wolfram Syndrome, Diabetes Mellitus
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ExenatideDrug
Exenatide
| Study group | Role | What they receive |
|---|---|---|
| Exenatide | Experimental | Not described Receives: Drug: Exenatide |
What is being measured
The primary outcome is the main question the study is designed to answer.
beta cell function
IVGTT test and meal test will be performed before starting treatment with Exenetide and after 3 months of treatment.
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Feb 2011 (estimated)
- Study start1 Mar 2011
- Primary completion1 Mar 2013 (estimated)
- Study completion1 Mar 2013
Why it stopped: Very rare disease, we were unable to recruit patients
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Diabetes Mellitus Associated With Genetic SyndromeStudies of the same condition, open and under way.
- More trials run by Hadassah Medical OrganizationThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hadassah Medical Organization
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Adar Zinger, Hadassah Medical Organization
- Investigators
- Gil Leibowitz, MD, Principal investigator, Hadassah Medical Organization
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01302327
- Sponsor's protocol ID
- wolfram-HMO-CTIL
- Status last verified
- Mar 2022
- First submitted
- 22 Feb 2011
- Last updated
- 29 Mar 2022
- Results posted
- No
- Data monitoring committee
- No
Publications
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.
PubMed 27459537
Study at a glance
From the registry record, last updated by the sponsor on 29 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- GLP Analogs for Diabetes in Wolfram Syndrome Patients
- ClinicalTrials.gov ID
- NCT01302327
- Acronym / sponsor ID
- wolfram-HMO-CTIL
- Keywords
- Wolfram syndrome, Diabetes mellitus, Exenatide, GLP-1 analog, beta cell function
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Very rare disease, we were unable to recruit patients
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Hadassah Medical OrganizationUniversity, hospital or other
- Study start
- 1 Mar 2011
- Primary completion
- 1 Mar 2013 (estimated)
- Study completion
- 1 Mar 2013 (actual)
- First posted
- 24 Feb 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Exenatide | Experimental | Drug: Exenatide |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (2 listed)
- pregnant women
- patients who are unable to give inform consent.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ExenatideExenatide | Drug | Exenatide |
Primary outcome
beta cell function
Time frame: 3 months
IVGTT test and meal test will be performed before starting treatment with Exenetide and after 3 months of treatment.
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Mar 2022.
- Derived from this study: PubMed 27459537Danielpur 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Gil Leibowitz, MD principal investigator
Hadassah Medical Organization
Questions about this trial
What is this clinical trial studying?
A study testing Exenatide in people with Diabetes Mellitus Associated With Genetic Syndrome and Wolfram Syndrome, run by Hadassah Medical Organization.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 29 Mar 2022. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
beta cell function (time frame: 3 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01302327, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Mar 2022.