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Efficacy and Safety Study of WTX101 in Adult Wilson Disease Patients

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ClinicalTrials.gov Identifier: NCT02273596
Recruitment Status : Completed
First Posted : October 24, 2014
Last Update Posted : December 10, 2018
Sponsor:
Information provided by (Responsible Party):
Wilson Therapeutics AB

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

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

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

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 ClinicalTrials.gov identifier (NCT number): NCT02273596


Locations
United States, California
Clinical Trial Site
Los Angeles, California, United States, 950095
United States, Connecticut
Clinical Trial Site
New Haven, Connecticut, United States, 06520
United States, Illinois
Clinical Trial Site
Chicago, Illinois, United States, 60611
United States, Michigan
Clinical Trial Site
Ann Arbor, Michigan, United States, 48109
United States, Tennessee
Clinical Trial Site
Nashville, Tennessee, United States, 37232
United States, Washington
Clinical Trial Site
Seattle, Washington, United States, 98105
Austria
Clinical Trial Site
Vienna, Austria, 1090
Germany
Clinical Trial Site
Heidelberg, Germany, 69120
Poland
Clinical Trial Site
Warsaw, Poland, 02-957
United Kingdom
Clinical Trial Site
Guildford, Surrey, United Kingdom, GU27XX
Clinical Trial Site
Birmingham, United Kingdom, B15 2TH
Sponsors and Collaborators
Wilson Therapeutics AB
Investigators
Study Director: Eugene Swenson, MD, PhD Alexion Pharmaceuticals

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Wilson Therapeutics AB
ClinicalTrials.gov Identifier: NCT02273596     History of Changes
Other Study ID Numbers: WTX101-201
First Posted: October 24, 2014    Key Record Dates
Last Update Posted: December 10, 2018
Last Verified: November 2018

Additional relevant MeSH terms:
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