UnknownPhase 2/3

Treatment of Wolfram Syndrome Type 2 With the Chelator Deferiprone and Incretin Based Therapy

Israel20 participantsStarted 2016-12

Plain-language summary

Patients who are genetically diagnosed with the recently reported and rare Wolfram syndrome type 2 ( WFS2) and have the degenerative and symptomatic disease including signs such as diabetes, platelet aggregation defect or visual problems will be asked to participate in this study. Knowing the pathomechanism of WFS2 with rapid cell death, after doing baseline investigations to asses the severity of their disease, the participants will be offered a chelator therapy with in addition to the antioxidant Acetylcystein, in diabetic patients an Incertin (GLP-1 ) therapy will be offered as well. The baseline investigations will be repeated after 2 months and after 5 months of therapy in order to asses the progression of the disease and to show if the chelator and anti oxidant therapy and in diabetic patients the GLP-1 therapy could stop the progression of the disease.

Who can participate

Age range

3 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Male or female patients, of any age genetically and clinically diagnosed with Wolfram syndrome type 2. Exclusion Criteria: * Patients who are non-cooperative. * Patients with bone marrow disease or neutropenia.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Insulin levels in blood in response to Glucose Challenge - Oral Glucose tolerance test (OGTT ) and Intra venous glucose tolerance test (IVGTT)

Timeframe: 5 months

Platelet aggregation to Adenosine diphosphate (ADP) and Collagen - blood test for Platelet function test

Timeframe: 5 months

Nerve conduction velocity in VEP

Timeframe: 5 months

HBA1C level

Timeframe: 5 months

daily glucose level measurement in the blood using Glucometer two to 5 times daily

Timeframe: 5 months

C-Peptide levels in blood in response to Glucose Challenge - Oral Glucose tolerance test ( OGTT ) and Intra venous glucose tolerance test IVGTT

Timeframe: 5 months

Trial details

NCT IDNCT02882477

SponsorHadassah Medical Organization

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2018-05

Contact for this trial

David Zangen, Professor

0097507874488 zangend@hadassah.org.il

View on ClinicalTrials.gov More Diabetes Mellitus trials

Plain-language summary

Who can participate

Age range

3 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Insulin levels in blood in response to Glucose Challenge - Oral Glucose tolerance test (OGTT ) and Intra venous glucose tolerance test (IVGTT)

Timeframe: 5 months

Platelet aggregation to Adenosine diphosphate (ADP) and Collagen - blood test for Platelet function test

Timeframe: 5 months

Nerve conduction velocity in VEP

Timeframe: 5 months

HBA1C level

Timeframe: 5 months

daily glucose level measurement in the blood using Glucometer two to 5 times daily

Timeframe: 5 months

C-Peptide levels in blood in response to Glucose Challenge - Oral Glucose tolerance test ( OGTT ) and Intra venous glucose tolerance test IVGTT

Timeframe: 5 months