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Trial record 1 of 2 for:    WTx101
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Efficacy and Safety Study of WTX101 in Adult Wilson Disease Patients

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Wilson Therapeutics AB
ClinicalTrials.gov Identifier:
NCT02273596
First received: October 20, 2014
Last updated: November 22, 2016
Last verified: November 2016
  Purpose
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 Intervention Phase
Wilson Disease
Drug: WTX101
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by Wilson Therapeutics AB:

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

Secondary Outcome Measures:
  • 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 ]

Estimated Enrollment: 30
Study Start Date: November 2014
Estimated Study Completion Date: August 2017
Estimated Primary Completion Date: August 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

  Eligibility

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
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.
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02273596

Locations
United States, California
UCLA Medical Center
Los Angeles, California, United States, 950095
United States, Connecticut
Yale University School of Medicine
New Haven, Connecticut, United States, 06520
United States, Illinois
Northwestern University
Chicago, Illinois, United States, 60611
United States, Michigan
University of Michigan Hospital
Ann Arbor, Michigan, United States, 48109
United States, Tennessee
Vanderbilt Medical Center
Nashville, Tennessee, United States, 37232
United States, Washington
Seattle Children's
Seattle, Washington, United States, 98105
Austria
University Klinik fur Innere Medizin IV / Medical University of Vienna
Vienna, Austria, 1090
Germany
Universitätsklinikum Heidelberg Medizinische Klinik / Internal Medicine IV University Hospital Heidelberg
Heidelberg, Germany, 69120
Poland
Institute of Psychiatry and Neurology
Warsaw, Poland, 02-957
United Kingdom
The Royal Surrey County Hospital NHS Foundation Trust
Guildford, Surrey, United Kingdom, GU27XX
Sandwell and West Birmingham Hospitals Nhs Trust
Birmingham, United Kingdom, B15 2TH
Sponsors and Collaborators
Wilson Therapeutics AB
Investigators
Study Director: Carl Bjartmar, MD, PhD Wilson Therapeutics AB
  More Information

Responsible Party: Wilson Therapeutics AB
ClinicalTrials.gov Identifier: NCT02273596     History of Changes
Other Study ID Numbers: WTX101-201
Study First Received: October 20, 2014
Last Updated: November 22, 2016

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

ClinicalTrials.gov processed this record on March 29, 2017