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

NCT02882477 · Status: UNKNOWN · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2016-08-29

No results posted yet for this study

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.

Conditions

  • Diabetes Mellitus
  • Iron Metabolism Disorders
  • Gastroduodenal Ulcer
  • Optic Atrophy
  • Sensorineural Hearing Loss
  • Platelet Dysfunction

Interventions

DRUG

Deferiprone

DRUG

Acetylcysteine

DRUG

Sitagliptin and Metformin

Sponsors & Collaborators

  • Hadassah Medical Organization

    lead OTHER

Principal Investigators

  • David Zangen, Professor · Head of pediatric endocrinology department

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
3 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-12-31
Primary Completion
2018-05-31
Completion
2018-12-31

Countries

  • Israel

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02882477 on ClinicalTrials.gov