Efficacy and Safety of ALXN1840 Administered for 48 Weeks Versus Standard of Care in Participants With Wilson Disease
Launched by ALEXION PHARMACEUTICALS, INC. · Jan 10, 2018
Trial Information
Current as of May 28, 2025
Terminated
Keywords
ClinConnect Summary
The study consists of 2 cohorts. Cohort 1: Participants who have received SoC therapy for \> 28 days and Cohort 2: Participants who are treatment-naïve or who have received SoC therapy for ≤ 28 days.
All enrolled participants were randomized by cohort in a 2:1 ratio to treatment with ALXN1840 or SoC (either as continued therapy in Cohort 1 or as continued or initial therapy in Cohort 2).
Gender
ALL
Eligibility criteria
- Key Inclusion Criteria:
- • Established diagnosis of WD by Leipzig-Score ≥ 4
- • Female participants of childbearing potential, if heterosexually active, must be willing to follow protocol-specified guidance for highly effective contraception starting at least 6 weeks before the Day 1 visit and continuing through 28 days after the last dose of either ALXN1840 or SoC
- • Male participants, if heterosexually active, must be willing to follow protocol-specified guidance for highly effective contraception beginning at Day 1 visit and continuing through 90 days after last dose of either ALXN1840 or SoC
- Key Exclusion Criteria:
- • Decompensated hepatic cirrhosis
- • MELD score \> 13
- • Modified Nazer score \> 7
- • Clinically significant gastrointestinal bleed within past 3 months
- • Alanine aminotransferase \> 2 X upper limit of normal (ULN) for participants treated for \> 28 days with WD therapy (Cohort 1)
- • Alanine aminotransferase \> 5 X ULN for treatment-naïve participants or participants who have been treated for ≤ 28 days (Cohort 2)
- • Marked neurological disease requiring either nasogastric feeding or intensive inpatient medical care
- • Hemoglobin \< 9 grams/deciliter
- • 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
- • Participants with end-stage renal disease on dialysis (chronic kidney disease stage 5) or creatinine clearance \< 30 milliliter/minute
About Alexion Pharmaceuticals, Inc.
Alexion Pharmaceuticals, Inc. is a global biopharmaceutical company dedicated to the discovery, development, and commercialization of innovative therapies for patients with rare and complex diseases. With a strong focus on addressing unmet medical needs, Alexion leverages advanced science and technology to create transformative treatments that improve the quality of life for patients suffering from conditions such as complement-mediated disorders, neurology, hematology, and metabolic diseases. Committed to patient-centric approaches, Alexion collaborates with healthcare professionals, patients, and advocacy groups to drive clinical research and foster advancements in rare disease treatment.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
Chicago, Illinois, United States
Ann Arbor, Michigan, United States
Adelaide, South Australia, Australia
Hong Kong, , Hong Kong
Concord, New South Wales, Australia
Paris, , France
Toronto, Ontario, Canada
New Haven, Connecticut, United States
Seattle, Washington, United States
Toronto, Ontario, Canada
Los Angeles, California, United States
Chicago, Illinois, United States
Houston, Texas, United States
Camperdown, New South Wales, Australia
Los Angeles, California, United States
Praha 2, , Czechia
Belgrade, , Serbia
Chiba, , Japan
Matsuyama Shi, Ehime, Japan
Barcelona, , Spain
Seattle, Washington, United States
Paris, , France
Budapest, , Hungary
Heidelberg, , Germany
Meguro Ku, Tokyo, Japan
Graz, , Austria
Innsbruck, , Austria
Vienna, , Austria
Parkville, , Australia
Jerusalem, , Israel
New Haven, Connecticut, United States
London, , United Kingdom
Toronto, Ontario, Canada
Heidelberg, , Germany
St. Petersburg, , Russian Federation
Ann Arbor, Michigan, United States
Barcelona, , Spain
Ramat Gan, , Israel
Concord, , Australia
Málaga, , Spain
Adelaide, , Australia
Hamburg, , Germany
Leipzig, , Germany
Jerusalem, , Israel
Warszawa, , Poland
Belgrade, , Serbia
Prague, , Czechia
Kurume Shi, Fukuoka, Japan
Taipei, , Taiwan
Chicago, Illinois, United States
Houston, Texas, United States
Izmir, , Turkey
Houston, Texas, United States
Takatsuki Shi, Osaka, Japan
Ramat Gan, , Israel
Kumamoto Shi, , Japan
Warszawa, , Poland
Sapporo Shi, , Japan
Sabadell, , Spain
London, , United Kingdom
Takatsuki Shi, , Japan
Yokohama Shi, Kanagawa, Japan
Ankara, , Turkey
Moscow, , Russian Federation
Moscow, , Russian Federation
Singapore, , Singapore
Kurume Shi, , Japan
Daegu, , Korea, Republic Of
århus N, , Denmark
Meguro Ku, , Japan
Grafton, , New Zealand
Ann Arbor, Michigan, United States
Los Angeles, California, United States
Heidelberg, , Germany
Taoyuan City, , Taiwan
Budapest, , Hungary
Málaga, , Spain
Barcelona, , Spain
Taoyuan City, , Taiwan
Heidelberg, , Germany
Aarhus, , Denmark
Nizhniy Novgorod, , Russian Federation
Warszawa, , Poland
Toronto, Ontario, Canada
Los Angeles, California, United States
Houston, Texas, United States
Graz, Styria, Austria
Innsbruck, Tyrol, Austria
Wien, Vienna, Austria
Bron, , France
Leipzig, Sachsen, Germany
Hamburg, , Germany
Jerusalem, , Israel
Tel Hashomer, , Israel
Chiba Shi, Chiba, Japan
Sapporo Shi, Hokkaido, Japan
Daegu, , Korea, Republic Of
Auckland, , New Zealand
Warszawa, Woj. Mazowieckie, Poland
Moscow, , Russian Federation
Moscow, , Russian Federation
Nizhny Novgorod, , Russian Federation
Saint Petersburg, , Russian Federation
Sabadell, Barcelona, Spain
Ankara, , Turkey
Istanbul, , Turkey
Istanbul, , Turkey
Izmir, , Turkey
Birmingham, England, United Kingdom
Guildford, England, United Kingdom
London, England, United Kingdom
Yokohama Shi, , Japan
Aarhus, , Denmark
Paris, , France
Budapest, , Hungary
Guildford, , United Kingdom
New Haven, Connecticut, United States
Istanbul, , Turkey
Edgbaston, , United Kingdom
Singapore, , Singapore
Bron, , France
Taoyuan City, , Taiwan
Daegu, , Korea, Republic Of
Singapore, , Singapore
Adelaide, South Australia, Australia
Matsuyama City, , Japan
Seattle, Washington, United States
Concord, New South Wales, Australia
Graz, Styria, Austria
Innsbruck, Tyrol, Austria
Wien, Vienna, Austria
Prague, , Czechia
Leipzig, Sachsen, Germany
Hamburg, , Germany
Hong Kong, , Hong Kong
Jerusalem, , Israel
Tel Hashomer, , Israel
Chiba Shi, Chiba, Japan
Matsuyama Shi, Ehime, Japan
Kurume Shi, Fukuoka, Japan
Sapporo Shi, Hokkaido, Japan
Yokohama Shi, Kanagawa, Japan
Takatsuki Shi, Osaka, Japan
Meguro Ku, Tokyo, Japan
Auckland, , New Zealand
Warszawa, Woj. Mazowieckie, Poland
Moscow, , Russian Federation
Nizhny Novgorod, , Russian Federation
Saint Petersburg, , Russian Federation
Belgrade, , Serbia
Sabadell, Barcelona, Spain
Málaga, , Spain
Istanbul, , Turkey
Izmir, , Turkey
Birmingham, England, United Kingdom
Guildford, England, United Kingdom
London, England, United Kingdom
Bron, , France
Hong Kong, , Hong Kong
Chiba, , Japan
Warsaw, , Poland
Patients applied
Trial Officials
Eugene S. Swenson, M.D., Ph.D.
Study Director
Alexion Pharmaceuticals, Inc.
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported
Similar Trials