Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840
A Phase 2, Single-arm Pathologist-blinded 48-week Study Using Liver Biopsy Specimens to Assess Copper Concentration and Histopathologic Changes in ALXN1840-treated Patients With Wilson Disease Followed by an up to 48-weeks Extension Period
2 other identifiers
interventional
31
8 countries
12
Brief Summary
The main objective of the study is to evaluate the change in liver copper (Cu) concentration following 48 weeks of treatment with ALXN1840 in adult participants with Wilson Disease (WD) who have been previously treated for at least 1 year with standard of care (that is, trientine, penicillamine, or zinc). In the Treatment Period, efficacy and safety of ALXN1840 will be assessed at Week 48.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
Started Dec 2020
12 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
May 22, 2020
CompletedFirst Posted
Study publicly available on registry
June 9, 2020
CompletedStudy Start
First participant enrolled
December 2, 2020
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 21, 2022
CompletedStudy Completion
Last participant's last visit for all outcomes
May 17, 2023
CompletedResults Posted
Study results publicly available
July 3, 2023
CompletedOctober 18, 2024
October 1, 2024
1.5 years
May 22, 2020
May 18, 2023
October 16, 2024
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Change From Baseline in Liver Cu Concentration at Week 48 (Treatment Period)
Liver biopsy samples were taken for the assessment of liver Cu concentration. Multiple imputation was used to impute missing data at Week 48 due to any reason based on Baseline values.
Baseline, Week 48 (Treatment Period)
Secondary Outcomes (15)
Number of Participants With Change From Baseline in Nonalcoholic Steatohepatitis Clinical Research Network (NASH CRN) Fibrosis Stage at Week 48 (Treatment Period)
Baseline, Week 48 (Treatment Period)
Number of Participants With Change From Baseline in Metavir Fibrosis Score at Week 48 (Treatment Period)
Baseline, Week 48 (Treatment Period)
Number of Participants With Change From Baseline in Ishak Fibrosis Score at Week 48 (Treatment Period)
Baseline, Week 48 (Treatment Period)
Change From Baseline in Hepatic Collagen Content at Week 48 (Treatment Period)
Baseline, Week 48 (Treatment Period)
Change From Baseline in a-SMA Content at Week 48 (Treatment Period)
Baseline, Week 48 (Treatment Period)
- +10 more secondary outcomes
Study Arms (1)
ALXN1840
EXPERIMENTALParticipants will receive ALXN1840.
Interventions
Participants will be initiated at 15 milligrams once daily, then the dose will be increased to 30 milligrams once daily at Week 6.
Eligibility Criteria
You may qualify if:
- Diagnosis of WD by Leipzig Criteria ≥ 4 or by historical test results.
- Continuous treatment for WD with penicillamine, trientine or zinc for at least 1 year prior to screening.
- Body mass index \< 30 kilograms/meter squared.
- Able to cooperate with a percutaneous liver biopsy.
- Willing and able to follow protocol-specified contraception requirements.
- Capable of giving signed informed consent.
You may not qualify if:
- Decompensated cirrhosis or Model for End Stage Liver Disease score \> 13.
- Modified Nazer score \> 7.
- Clinically significant gastrointestinal bleed within past 3 months.
- Alanine aminotransferase \> 2 × upper limit of normal.
- History of bleeding abnormality or known coagulopathy, including platelet count \< 100,000, and international normalized ratio for prothrombin time ≥ 1.5.
- Participant unwilling to accept blood products, if required.
- Marked neurological disease requiring either nasogastric feeding tube or intensive inpatient medical care.
- Hemoglobin less than lower limit of the reference range for age and sex.
- Participants in renal failure, defined as in end-stage renal disease on dialysis (chronic kidney disease 5) or creatinine clearance \< 30 milliliters/minute.
- Lymphoma, leukemia, or any malignancy within the past 5 years.
- Current or chronic history of liver disease not associated with WD.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (12)
Research Site
Sacramento, California, 95817, United States
Research Site
Ann Arbor, Michigan, 48109, United States
Research Site
Dallas, Texas, 75235, United States
Research Site
Toronto, Ontario, M5G 2C4, Canada
Research Site
Århus N, 8200, Denmark
Research Site
Grafton, 1010, New Zealand
Research Site
Moscow, 119021, Russia
Research Site
Saint Petersburg, 194017, Russia
Research Site
Singapore, 169608, Singapore
Research Site
Barcelona, 08036, Spain
Research Site
Valencia, 46026, Spain
Research Site
London, SE5 9RS, United Kingdom
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Alexion Pharmaceuticals, Inc.
- Organization
- Alexion Pharmaceuticals, Inc.
Study Officials
- STUDY DIRECTOR
Eugene S. Swenson, MD, PhD
Alexion Pharmaceuticals, Inc.
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Masking Details
- This study is only pathologist-blinded for the assessments of liver histology samples.
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
May 22, 2020
First Posted
June 9, 2020
Study Start
December 2, 2020
Primary Completion
May 21, 2022
Study Completion
May 17, 2023
Last Updated
October 18, 2024
Results First Posted
July 3, 2023
Record last verified: 2024-10
Data Sharing
- IPD Sharing
- Will not share