Decentralised study in Wilson's Disease

• Research type

  Research Study

• Full title

  Decentralized study to assess patient treatment preference comparing their current standard-of-care Wilson’s Disease (WD) treatment to a new once-daily Trientine (TETA) 4HCl formulation.

• IRAS ID

  1011374

• Contact name

  Benedicte Deloux

• Contact email

  regulatory@orphalan.com

• Sponsor organisation

  Orphalan SA

• Research summary

  Wilson’s Disease (WD) is a life-threatening genetic condition that occurs in approximately 1 in 30,000 – 40,000 people worldwide. The body only needs a very small amount of copper to function normally. In WD the body can’t process and remove copper normally leading to a build-up of copper in the body, particularly in the liver and brain. Excess copper in the liver causes inflammation, damage and scarring, eventually leading to liver failure. Copper build-up in the brain can cause confusion, physical tremors and problems with coordination and clumsiness.
  There are no cures for WD, but there are treatments to stop copper building up. WD patients are not happy with the treatments are currently available and approximately 30% of patients don’t take their medications as they should, leading to worsening of symptoms. The current treatments are multiple tablets / capsules taken multiple times a day that can’t be taken at the same time as meals or other medications. Long term use of current treatment also has side effects such as nausea, stomach pain, rashes and diarrhoea.
  This study aims to assess and compare patient preference and convenience between the currently available treatments and a new once daily treatment. Adult patients who have been on a stable course of treatment for at least 3 months will be eligible for this decentralised study. Participants will receive the new once daily treatment for 28 days and will then receive their standard of care treatment for 4 weeks. Safety blood tests will be conducted fortnightly. Participants will complete regular questionnaires about their treatment satisfaction and whether they took the medication each day. The safety assessments and patient questionnaires will be compared between the two different treatments to determine which treatment patients prefer.

• REC name

  Wales REC 1

• REC reference

  25/WA/0070

• Date of REC Opinion

  23 Apr 2025

• REC opinion

  Further Information Favourable Opinion