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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. Identifier: NCT02273596
Recruitment Status : Completed
First Posted : October 24, 2014
Last Update Posted : July 9, 2019
Information provided by (Responsible Party):
Alexion Pharmaceuticals

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.

Condition or disease Intervention/treatment Phase
Wilson Disease Drug: WTX101 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 28 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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 Older With an Extension Phase of 12 Months
Actual Study Start Date : November 24, 2014
Actual Primary Completion Date : October 27, 2016
Actual Study Completion Date : November 7, 2018

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Wilson Disease

Arm Intervention/treatment
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
Drug: WTX101
Dosage Form: 15 mg Tablets

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

Secondary Outcome Measures :
  1. Change in and time to normalisation of NCC levels adjusted for Mo plasma concentration [ Time Frame: 24 weeks ]
  2. Neurological status using the Unified Wilson's Disease Rating Scale (UWDRS) [ Time Frame: 24 weeks ]
  3. Psychiatric status using Mini International Neuropsychiatric Interview (M.I.N.I.) Tracking [ Time Frame: 24 weeks ]
  4. 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 ]
  5. Quality of Life / Patient Reported Outcome endpoint measures EQ5D [ Time Frame: 24 weeks ]
  6. Quality of Life / Patient Reported Outcome endpoint measure MMAS-8 [ Time Frame: 24 weeks ]
  7. Quality of Life / Patient Reported Outcome endpoint measure TSQM [ Time Frame: 24 weeks ]
  8. Hepatic measure ALT [ Time Frame: 24 weeks ]
  9. Hepatic measure AST [ Time Frame: 24 weeks ]
  10. Hepatic measure INR [ Time Frame: 24 weeks ]
  11. Hepatic measure bilirubin) [ Time Frame: 24 weeks ]
  12. Copper endpoint: Exchangeable copper [ Time Frame: 24 weeks ]
  13. Copper endpoint: Speciation profiling [ Time Frame: 24 weeks ]
  14. Copper endpoint: 24-hour urinary copper [ Time Frame: 24 weeks ]
  15. Plasma PK parameters. Estimates of individual molybdenum PK parameters, including AUC and Cmax [ Time Frame: 24 weeks ]
  16. Incidence and severity of adverse events (AEs) [ Time Frame: Throughout the study (screening up to follow-up) ]
  17. Copper endpoint: Total copper [ Time Frame: 24 weeks ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02273596

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United States, California
UCLA Department of Neurology
Los Angeles, California, United States, 950095
United States, Connecticut
Yale University Medical Centre
New Haven, Connecticut, United States, 06520
United States, Illinois
Northwestern University
Chicago, Illinois, United States, 60611
United States, Michigan
University of Michigan
Ann Arbor, Michigan, United States, 48109
United States, Tennessee
Vanderbilt Medical Center Department of Neurology Division of Movement Disorders
Nashville, Tennessee, United States, 37232
United States, Washington
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Medical University of Vienna
Vienna, Austria, 1090
Internal Medicine IV University Hospital Heidelberg
Heidelberg, Germany, 69120
Institute of Psychiatry and Neurology 2nd DEPARTMENT OF NEUROLOGY
Warsaw, Poland, 02-957
United Kingdom
The Royal Surrey County Hospital NHS Trust
Guildford, Surrey, United Kingdom, GU27XX
Queen Elizabeth Hospital, Birmingham
Birmingham, United Kingdom, B15 2TH
Sponsors and Collaborators
Alexion Pharmaceuticals
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Study Director: Eugene Swenson, MD, PhD Alexion Pharmaceuticals

Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Alexion Pharmaceuticals Identifier: NCT02273596    
Other Study ID Numbers: WTX101-201
First Posted: October 24, 2014    Key Record Dates
Last Update Posted: July 9, 2019
Last Verified: July 2019
Additional relevant MeSH terms:
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Hepatolenticular Degeneration
Liver Diseases
Digestive System Diseases
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Metal Metabolism, Inborn Errors
Metabolic Diseases