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More info
You can access this
clinical trial
if you have
Diabetes Mellitus, Iron Metabolism Disorders, Gastroduodenal Ulcer, Optic Atrophy, Sensorineural Hearing Loss or Platelet Dysfunction
and you are
over 3
years old
2
3
This is an advanced phase trial assessing
the effectiveness of the new treatment before being released on the market.
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The purpose

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.

Provided treatments

  • Drug: Deferiprone
  • Drug: Acetylcysteine
  • Drug: Sitagliptin and Metformin

Locations near you

Unfortunately, there are no recruiting locations near you. Please check the list with all locations below.
Tris trial is registered with FDA with number: NCT02882477. The sponsor of the trial is Hadassah Medical Organization and it is looking for 20 volunteers for the current phase.
Official trial title:
Treatment of Wolfram Syndrome Type 2 With the Chelator Deferiprone, and Incretin Based Therapy