Clinical Trial: Efficacy and Safety Study of WTX101 in Adult Wilson Disease Patients

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: A Phase 2, Multi-centre, Open-label, Study to Evaluate the Efficacy and Safety of WTX101 Administered for 24 Weeks in Newly Diagnosed Wilson Disease Patients Aged 18 and O

Brief Summary: The purpose of the study is to evaluate the efficacy and safety of WTX101 administered for 24 weeks in newly diagnosed Wilson Disease (WD) patients aged 18 and older with Nonceruloplasmin-bound copper (NCC) levels within or above the normal reference range at the time of enrollment. Subjects with Wilson Disease who have received either no prior Wilson Disease treatments, or have been treated for up to and including 24 months prior to study enrollment with chelation therapy (e.g. trientine, penicillamine), zinc therapy or combination therapy are eligible to participate, if all other inclusion and no exclusion criteria are met. The purpose of the 12 month Extension Phase is to evaluate the durability, and establish long-term safety and efficacy of WTX101.

Detailed Summary:
Sponsor: Wilson Therapeutics AB

Current Primary Outcome: Nonceruloplasmin-bound copper (NCC) levels adjusted for molybdenum (Mo) plasma concentration [ Time Frame: 24 weeks ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Change in and time to normalisation of NCC levels adjusted for Mo plasma concentration [ Time Frame: 24 weeks ]
  • Neurological status using the Unified Wilson's Disease Rating Scale (UWDRS) [ Time Frame: 24 weeks ]
  • Psychiatric status using Mini International Neuropsychiatric Interview (M.I.N.I.) Tracking [ Time Frame: 24 weeks ]
  • Clinical symptoms as assessed by the Investigators on the Clinician Global Impression (CGI) scale items 1 (severity of illness) and 2 (global improvement) [ Time Frame: 24 weeks ]
  • Quality of Life / Patient Reported Outcome endpoint measures EQ5D [ Time Frame: 24 weeks ]
  • Quality of Life / Patient Reported Outcome endpoint measure MMAS-8 [ Time Frame: 24 weeks ]
  • Quality of Life / Patient Reported Outcome endpoint measure TSQM [ Time Frame: 24 weeks ]
  • Hepatic measure ALT [ Time Frame: 24 weeks ]
  • Hepatic measure AST [ Time Frame: 24 weeks ]
  • Hepatic measure INR [ Time Frame: 24 weeks ]
  • Hepatic measure bilirubin) [ Time Frame: 24 weeks ]
  • Copper endpoint: Exchangeable copper [ Time Frame: 24 weeks ]
  • Copper endpoint: Speciation profiling [ Time Frame: 24 weeks ]
  • Copper endpoint: 24-hour urinary copper [ Time Frame: 24 weeks ]
  • Plasma PK parameters. Estimates of individual molybdenum PK parameters, including AUC and Cmax [ Time Frame: 24 weeks ]
  • Incidence and severity of adverse events (AEs) [ Time Frame: Throughout the study (screening up to follow-up) ]
  • Copper endpoint: Total copper [ Time Frame: 24 weeks ]


Original Secondary Outcome:

  • Change in and time to normalisation of NCC levels adjusted for Mo plasma concentration [ Time Frame: 24 weeks ]
  • Neurological status using the Unified Wilson's Disease Rating Scale (UWDRS) [ Time Frame: 24 weeks ]
  • Psychiatric status using Mini International Neuropsychiatric Interview (M.I.N.I.) Tracking [ Time Frame: 24 weeks ]
  • Clinical symptoms as assessed by the Investigators on the Clinician Global Impression (CGI) scale items 1 (severity of illness) and 2 (global improvement) [ Time Frame: 24 weeks ]
  • Quality of Life / Patient Reported Outcome endpoint measures EQ5D [ Time Frame: 24 weeks ]
  • Quality of Life / Patient Reported Outcome endpoint measure MMAS-8 [ Time Frame: 24 weeks ]
  • Quality of Life / Patient Reported Outcome endpoint measure TSQM [ Time Frame: 24 weeks ]
  • Hepatic measure ALT [ Time Frame: 24 weeks ]
  • Hepatic measure AST [ Time Frame: 24 weeks ]
  • Hepatic measure INR [ Time Frame: 24 weeks ]
  • Hepatic measure bilirubin) [ Time Frame: 24 weeks ]
  • Copper endpoint: Exchangeable copper [ Time Frame: 24 weeks ]
  • Copper endpoint: Speciation profiling [ Time Frame: 24 weeks ]
  • Copper endpoint: 24-hour urinary copper [ Time Frame: 24 weeks ]
  • Plasma PK parameters. Estimates of individual molybdenum PK parameters, including AUC and Cmax [ Time Frame: 24 weeks ]
  • Incidence and severity of adverse events (AEs) [ Time Frame: Throughout the study (screening up to follow-up) ]


Information By: Wilson Therapeutics AB

Dates:
Date Received: October 20, 2014
Date Started: November 2014
Date Completion: August 2017
Last Updated: November 22, 2016
Last Verified: November 2016