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Efficacy and Safety Study of WTX101 in Adult Wilson Disease Patients

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02273596
Recruitment Status : Active, not recruiting
First Posted : October 24, 2014
Last Update Posted : December 21, 2017
Sponsor:
Information provided by (Responsible Party):

October 20, 2014
October 24, 2014
December 21, 2017
November 2014
June 2018   (Final data collection date for primary outcome measure)
Nonceruloplasmin-bound copper (NCC) levels adjusted for molybdenum (Mo) plasma concentration [ Time Frame: 24 weeks ]
Same as current
Complete list of historical versions of study NCT02273596 on ClinicalTrials.gov Archive Site
  • 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 ]
  • 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) ]
Not Provided
Not Provided
 
Efficacy and Safety Study of WTX101 in Adult Wilson Disease Patients
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 Older With an Extension Phase of 12 Months
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.
Not Provided
Interventional
Phase 2
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Wilson Disease
Drug: WTX101
Dosage Form: 15 mg Tablets
Experimental: WTX101
WTX101 Dosage Form: Enteric Coated Tablet WTX101 Dose: 15 - 60 mg, individualized dosing, WTX101 Frequency: QOD, QD or BID, individualized dosing WTX101 Duration: 76 weeks
Intervention: Drug: WTX101
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
30
August 2018
June 2018   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • Willing and able to give informed consent for participation in the study.
  • Male or female patients, aged 18 years or older as of signing the ICF.
  • Able to understand and willing to comply with study procedures, restrictions and requirements, as judged by the Investigator.
  • Established diagnosis of Wilson Disease by Leipzig-Score ≥ 4 (Ferenci et al 2003) documented by testing as outlined in 2012 EASL WD Clinical Practice Guidelines.
  • NCC levels within or above the normal reference range (0.8 - 2.3 μM).
  • Willing to undergo 48 hour washout from current Wilson Disease treatment

Exclusion Criteria:

  • Treatment for greater than 24 months for Wilson Disease with chelation therapy (i.e. penicillamine, trientine hydrochloride) or zinc therapy.
  • Decompensated hepatic cirrhosis.
  • Model for End-Stage Liver Disease (MELD) score > 11.
  • Modified Nazer score > 6 (Dhawan et al. Liver Transplant 2005).
  • GI bleed within past 6 months.
  • ALT > 5x upper limit of normal (ULN).
  • Marked neurological disease requiring either nasogastric (NG) feeding or intensive in-patient medical care.
  • Severe anaemia with a haemoglobin < 9 mg/dL.
Sexes Eligible for Study: All
18 Years and older   (Adult, Senior)
No
Contact information is only displayed when the study is recruiting subjects
Austria,   Germany,   Poland,   United Kingdom,   United States
 
 
NCT02273596
WTX101-201
Yes
Not Provided
Not Provided
Wilson Therapeutics AB
Wilson Therapeutics AB
Not Provided
Study Director: Carl Bjartmar, MD, PhD Wilson Therapeutics AB
Wilson Therapeutics AB
December 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP