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Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840

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.
 
ClinicalTrials.gov Identifier: NCT04422431
Recruitment Status : Active, not recruiting
First Posted : June 9, 2020
Last Update Posted : January 27, 2023
Sponsor:
Information provided by (Responsible Party):
Alexion

Study Description
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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.

Condition or disease Intervention/treatment Phase
Wilson Disease Drug: Bis-Choline Tetrathiomolybdate Phase 2

Detailed Description:
Participants who complete the 48-week Treatment Period will be offered the opportunity to continue their treatment in a 48-week Extension Period that will offer additional time for evaluation of long-term efficacy and safety of ALXN1840. There will be no liver biopsies during the Extension Period.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 31 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Masking Description: This study is only pathologist-blinded for the assessments of liver histology samples.
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : December 2, 2020
Actual Primary Completion Date : May 17, 2022
Estimated Study Completion Date : May 27, 2023

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: ALXN1840
Participants will receive ALXN1840.
 Drug: Bis-Choline Tetrathiomolybdate
Participants will be initiated at 15 milligrams once daily, then the dose will be increased to 30 milligrams once daily at Week 6.
Other Name: ALXN1840


Outcome Measures
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Primary Outcome Measures :
  1. Change From Baseline At Week 48 In Liver Cu Concentration [ Time Frame: Baseline, Week 48 ]

Secondary Outcome Measures :
  1. Change From Baseline At Week 48 In Liver Histopathology As Assessed by Liver Biopsy [ Time Frame: Baseline, Week 48 ]
  2. Pre-dose Trough ALXN1840 Concentrations In Plasma At Each Study Site Visit [ Time Frame: Pre-dose at 0 hour, and at 2- and 4-hours post-dose at the Week 6 (Day 43) and Week 36 (Day 253) visits ]
  3. Clinical Global Impression-Improvement Scale At Week 48 [ Time Frame: Baseline, Week 48 ]
  4. Change From Baseline At Week 48 In The Clinical Global Impression-Severity Scale [ Time Frame: Baseline, Week 48 ]

Eligibility Criteria
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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:

  1. Diagnosis of WD by Leipzig Criteria ≥ 4 or by historical test results.
  2. Continuous treatment for WD with penicillamine, trientine or zinc for at least 1 year prior to screening.
  3. Body mass index < 30 kilograms/meter squared.
  4. Able to cooperate with a percutaneous liver biopsy.
  5. Willing and able to follow protocol-specified contraception requirements.
  6. Capable of giving signed informed consent.

Exclusion Criteria:

  1. Decompensated cirrhosis or Model for End Stage Liver Disease score > 13.
  2. Modified Nazer score > 7.
  3. Clinically significant gastrointestinal bleed within past 3 months.
  4. Alanine aminotransferase > 2 × upper limit of normal.
  5. History of bleeding abnormality or known coagulopathy, including platelet count < 100,000, and international normalized ratio for prothrombin time ≥ 1.5.
  6. Participant unwilling to accept blood products, if required.
  7. Marked neurological disease requiring either nasogastric feeding tube or intensive inpatient medical care.
  8. Hemoglobin less than lower limit of the reference range for age and sex.
  9. Participants in renal failure, defined as in end-stage renal disease on dialysis (chronic kidney disease 5) or creatinine clearance < 30 milliliters/minute.
  10. Lymphoma, leukemia, or any malignancy within the past 5 years.
  11. Current or chronic history of liver disease not associated with WD.
Contacts and Locations
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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): NCT04422431


Locations
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United States, California
Clinical Trial Site
Sacramento, California, United States, 95817
United States, Michigan
Clinical Trial Site
Ann Arbor, Michigan, United States, 48109
United States, Texas
Clinical Trial Site
Dallas, Texas, United States, 75390
Denmark
Clinical Trial Site
Aarhus, Denmark
New Zealand
Clinical Trial Site
Auckland, New Zealand
Russian Federation
Clinical Trial Site
Moscow, Russian Federation
Clinical Trial Site
St. Petersburg, Russian Federation
Singapore
Clinical Trial Site
Singapore, Singapore, 169608
Spain
Clinical Trial Site
Barcelona, Spain, 08036
Clinical Trial Site
Valencia, Spain, 46026
United Kingdom
Clinical Trial Site
London, United Kingdom
Sponsors and Collaborators
Alexion
Investigators
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Study Director: Eugene S. Swenson, MD, PhD Alexion
More Information
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Responsible Party: Alexion
ClinicalTrials.gov Identifier: NCT04422431    
Other Study ID Numbers: ALXN1840-WD-205
2019-003711-60 ( EudraCT Number )
First Posted: June 9, 2020    Key Record Dates
Last Update Posted: January 27, 2023
Last Verified: January 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Alexion:
Copper
ALXN1840
Histology
Liver Biopsy
Additional relevant MeSH terms:
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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
Tetrathiomolybdate
Choline
Lipotropic Agents
Hypolipidemic Agents
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Gastrointestinal Agents
Lipid Regulating Agents
Nootropic Agents
Chelating Agents
Sequestering Agents
Enzyme Inhibitors
Angiogenesis Inhibitors
Angiogenesis Modulating Agents