Working… Menu

Efficacy and Safety of ALXN1840 (Formerly Named WTX101) Administered for 48 Weeks Versus Standard of Care in Patients With Wilson Disease With an Extension Period of up to 60 Months

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: NCT03403205
Recruitment Status : Active, not recruiting
First Posted : January 18, 2018
Last Update Posted : April 7, 2020
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Brief Summary:
The purpose of this study is to evaluate the efficacy of ALXN1840 (formerly called WTX101) administered for 48 weeks compared to standard of care (SoC) in WD patients aged 12 and older.

Condition or disease Intervention/treatment Phase
Wilson Disease Drug: ALXN1840 Drug: SoC Therapy Phase 3

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 215 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (Outcomes Assessor)
Masking Description: This study is rater-blinded for the Unified Wilson Disease Rating Scale (UWDRS) assessment only.
Primary Purpose: Treatment
Official Title: A Phase 3, Randomized, Rater-Blinded, Multi-Center Study To Evaluate the Efficacy and Safety of ALXN1840 Administered For 48 Weeks Versus Standard of Care in Patients With Wilson Disease Aged 12 Years and Older With an Extension Period of Up To 60 Months
Actual Study Start Date : February 15, 2018
Estimated Primary Completion Date : February 2021
Estimated Study Completion Date : February 2026

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Wilson Disease

Arm Intervention/treatment
Experimental: ALXN1840 15-60 mg Drug: ALXN1840
ALXN1840 administered orally in 15 mg tablets
Other Name: Formerly named WTX101

Active Comparator: Standard of Care (SoC) Medication Drug: SoC Therapy
Depending on the site/region,subjects randomized to receive SoC treatment will receive trientine, penicillamine, Zinc, or a combination of these medicines, administered according to standard regimens.

Primary Outcome Measures :
  1. Evaluate the efficacy of ALXN1840 using standard of care as comparator and copper as the control assessed in terms of the percentage change from baseline (Day 1) to 48 weeks in non-ceruloplasmin-bound copper levels [ Time Frame: Change from baseline (Day 1) to 48 weeks ]

Secondary Outcome Measures :
  1. Safety and tolerability of individualized dosing of ALXN1840 by descriptive statistics [ Time Frame: Change from baseline (Day 1) to 48 weeks ]
  2. Effects of ALXN1840 on hepatic status [ Time Frame: Change from baseline (Day 1) to 48 weeks ]
  3. Effects of ALXN1840 on disability status [ Time Frame: Change from baseline (Day 1) to 48 weeks ]
  4. Effects of ALXN1840 on neurological status [ Time Frame: Change from baseline (Day 1) to 48 weeks ]
  5. Global effects of ALXN1840 on clinical symptoms as assessed by the Investigator on the Clinical Global Impression-Improvement Scale (CGI-I) and the Clinical Global Impression-Severity Scale (CGI-S) [ Time Frame: Change from baseline (Day 1) to 48 weeks ]
  6. Effects of ALXN1840 on NCC responder rate [ Time Frame: Change from baseline (Day 1) to 48 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.

Layout table for eligibility information
Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Established diagnosis of Wilson disease by Leipzig-Score = or > than 4
  • 12 years of age or older
  • Female patients of childbearing potential, if heterosexually active, must be willing to follow protocol-specified guidance for highly effective contraception starting at least 6 weeks before Day 1 visit and continuing through 28 days after the last dose of either ALXN1840 or SoC
  • Male patients, if heterosexually active, must be willing to follow protocol-specified guidance for highly effective contraception beginning at Day 1 and continuing through 90 days after last dose of either SoC or ALXN1840


  • Decompensated hepatic cirrhosis
  • MELD score > 13
  • Modified Nazer score > 7
  • Clinically significant GI bleed within past 3 months
  • Alanine aminotransferase > 2 X upper limit of normal (ULN) for patients treated for > 28 days with WD therapy (Cohort 1)
  • Alanine aminotransferase > 5 X ULN for treatment naïve patients or patients who have been treated for < or = to 28 days (Cohort 2)
  • Marked neurological disease requiring either nasogastric feeding or intensive inpatient medical care
  • Hemoglobin < 9 g/dL
  • History of seizure activity within 6 months prior to informed consent
  • Pregnant (or women who are planning to become pregnant) or breastfeeding women
  • Active infection with hepatitis B virus (positive hepatitis B surface antigen) or C virus or seropositivity for human immunodeficiency virus (HIV)
  • Previous treatment with tetrathiomolybdate
  • Patients with end-stage renal disease on dialysis (CKD 5) or creatinine clearance < 30 mL/min

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): NCT03403205

Hide Hide 87 study locations
Layout table for location information
United States, California
University of California, Los Angeles (UCLA) - David Geffen School of Medicine
Los Angeles, California, United States, 90095
United States, Connecticut
Yale University School of Medicine
New Haven, Connecticut, United States, 06510
United States, Florida
AdventHealth Medical Group Pediatric Gastroenterology at Orlando
Orlando, Florida, United States, 32803
United States, Illinois
Northwestern Memorial Hospital
Chicago, Illinois, United States, 60611
United States, Michigan
University of Michigan
Ann Arbor, Michigan, United States, 48109
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
United States, Tennessee
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37232
United States, Texas
UT Southwestern Medical Center
Dallas, Texas, United States, 75390-8887
Baylor College of Medicine
Houston, Texas, United States, 77030
Texas Children's Hospital
Houston, Texas, United States, 77030
United States, Washington
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Australia, New South Wales
Royal Prince Alfred Hospital
Camperdown, New South Wales, Australia, 2050
Australia, South Australia
Royal Adelaide Hospital
Adelaide, South Australia, Australia, 5000
Australia, Victoria
Austin Health
Heidelberg, Victoria, Australia, 3084
Alfred Hospital
Melbourne, Victoria, Australia, 3004
Royal Melbourne Hospital
Parkville, Victoria, Australia, 3050
The Royal Children's Hospital, Melbourne: Gastroenterology and Clinical Nutrition
Parkville, Victoria, Australia, 3052
Australia, Western Australia
Sir Charles Gairdner Hospital
Nedlands, Western Australia, Australia, 6009
Concord Repatriation General Hospital
Concord, Australia, NSW 2139
Medical University Graz
Graz, Austria, 8036
Medical University Innsbruck
Innsbruck, Austria, 6020
Medizinische Universitat Wien, Innere Medizin III
Vienna, Austria, 1090
Ghent University Hospital (Universitair Ziekenhuis Gent)
Gent, Oost-Vlaanderen, Belgium, B-9000
University Hospitals Leuven - Gasthuisberg
Leuven, Belgium, 3000
Canada, Ontario
Toronto General Hospital
Toronto, Ontario, Canada, M5G 2C4
Vseobecna fakultni nemocnice v Praze
Prague, Czechia
Aarhus Universitetshospital
Aarhus, Denmark, 8200
CHU Lyon- Hopital Femme- Mere-Enfant
Bron, France, 69677
Hopital Lariboisiere
Paris, France, 75010
Centre Hospitalier Universitaire (CHU) de Toulouse - Hopital Pierre-Paul Riquet
Toulouse, France, 31059
University Hospital Carl Gustav Carus Dresden
Dresden, Germany, 01307
Ifi - Studien und Projekte GmbH
Hamburg, Germany, 20099
University Hospital Heidelberg
Heidelberg, Germany, 69120
University Hospital Leipzig
Leipzig, Germany, 04103
Hong Kong
Queen Mary Hospital
Hong Kong, Hong Kong, 852
Semmelweis University
Budapest, Hungary, 1085
Carmel Medical Center
Haifa, Israel, 3436212
Hadassah University Hospital
Jerusalem, Israel, Q47X+XQ
Sheba Medical Center
Ramat Gan, Israel, 2RWV+M4
Ehime Prefectural Central Hospital
Matsuyama, Ehime, Japan
Kurume University Hospital
Kurume, Fukuoka, Japan
Hokkaido Medical Center
Sapporo, Hokkaido, Japan
Saiseikai Yokohama Tobu Hospital
Yokohama, Kanagawa, Japan
Chiba Children's Hospital
Chiba, Japan
Kumamoto University Hospital
Kumamoto, Japan
Osaka Medical College Hospital
Osaka, Japan
Toho University Ohashi Medical Center
Tokyo, Japan
Korea, Republic of
Kyungpook National University Children's Hospital
Daegu, Korea, Republic of, 41944
Asan Medical Center
Seoul, Korea, Republic of, 05505
Seoul National University
Seoul, Korea, Republic of
New Zealand
Auckland City Hospital
Auckland, New Zealand, 1023
Instytut Pomnik Centrum Zdrowia Dziecka
Warszawa, Woj. Mazowieckie, Poland, 04-730
Instytut Psychiatrii i Neurologii
Warsaw, Poland, 02-957
Russian Federation
National Scientific Institute of Nutrition
Moscow, Russian Federation, 115446
Federal State Autonomous Institution "National Medical Research Center of Children's Health" of the Ministry of Health of the Russian Federation
Moscow, Russian Federation, 119991
University Clinic Hospital Number 2, Federal State Autonomous Institution of Higher Education, First Moscow State Medical University n. a. I. M. Sechenov of the Ministry of Healthcare of the Russian Federation
Moscow, Russian Federation, 119991
University Clinic Hospital Number 3, Federal State Autonomous Institution of Higher Education, First Moscow State Medical University n. a. I. M. Sechenov of the Ministry of Healthcare of the Russian Federation
Moscow, Russian Federation, 119991
Privolzhsky Research Medical University of the Ministry of Healthcare of the Russian Federation
Nizhny Novgorod, Russian Federation, 603005
St. Petersburg Clinical Hospital of the Russian Academy of Sciences
Saint Petersburg, Russian Federation, 194021
Federal State Budgetary Institution "Child Research Clinical Infectious Disease Center" of the Medical Biology Agency
Saint Petersburg, Russian Federation, 197022
Children Hospital in Belgrade
Belgrade, Serbia, 11000
Clinic for Gastroenterohepatology, Clinical Center of Serbia
Belgrade, Serbia, 11000
Clinical Centre of Serbia
Belgrade, Serbia, 11000
National University Hospital
Singapore, Singapore, 119074
Singapore General Hospital (SGH)
Singapore, Singapore, 169608
KK Women's and Children's Hospital
Singapore, Singapore, 229899
Hospital Sant Joan de Déu Barcelona
Esplugues De Llobregat, Bareclona, Spain, 8950
Hospital Materno-Infantil Las Palmas de Gran Canaria
Las Palmas de Gran Canaria, Las Palmas, Spain, 35016
Hospital Universitari Vall d'Hebron
Barcelona,, Spain, 08035
Hospital Universitari Vall Hebron Barcelona
Barcelona, Spain, 08035
Hospital Clínic i Provincial de Barcelona
Barcelona, Spain, 08036
Hospital Parc Tauli de Sabadell
Barcelona, Spain, H446+P9
Hospital Universitario Puerta de Hierro Majadahonda
Madrid, Spain, 28222
Hospital Materno Infantil de Málaga
Málaga, Spain, 29011
Chang Gung Memorial Hospital (CGMH) - Kaohsiung Branch
Kaohsiung, Taiwan, 833
National Taiwan University Hospital
Taipei, Taiwan, 100
Chang Gung Memorial Hospital-Linkou Branch
Taoyuan, Taiwan, 333
Ege University School of Medicine
Izmir, Bornova, Turkey, 35100
Ege Universitesi Tip Fakultesi Çocuk Hastanesi (Ege University Medical Faculty Pediatric Hospital)
Bornova, Izmir, Turkey, 35100
Hacettepe Universitesi - Tip Fakultesi (Hacettepe University Faculty of Medicine)
Ankara, Turkey, 06620
Koc University Medical Faculty
Istanbul, Turkey, 1111
Bezmialem Foundation University Medical Faculty
Istanbul, Turkey, 34093
Istanbul Uni. Istanbul Medical Faculty Hospital
Istanbul, Turkey, 34093
United Kingdom
Queen Elizabeth Hospital Birmingham
Birmingham, England, United Kingdom, B15 2TH
Cambridge University - Addenbrooke's Hospital
Cambridge, England, United Kingdom, CB2 0QQ
Royal Surrey County Hospital
Guildford, England, United Kingdom, GU2 7XX
Kings College Hospital
Brixton, London, United Kingdom, SE5 9RS
Sponsors and Collaborators
Alexion Pharmaceuticals
Layout table for investigator information
Study Director: Eugene Swenson, MD, PhD Alexion Pharmaceuticals
Layout table for additonal information
Responsible Party: Alexion Pharmaceuticals Identifier: NCT03403205    
Other Study ID Numbers: WTX101-301
First Posted: January 18, 2018    Key Record Dates
Last Update Posted: April 7, 2020
Last Verified: April 2020

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
Layout table for 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
Metabolic Diseases
Metabolism, Inborn Errors
Metal Metabolism, Inborn Errors