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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT04422431




Registration number
NCT04422431
Ethics application status
Date submitted
22/05/2020
Date registered
9/06/2020
Date last updated
9/06/2020

Titles & IDs
Public title
Copper Concentration and Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840
Scientific title
A Phase 2, Single-arm, Pathologist-blinded Study Using Liver Biopsy Specimens to Assess Copper Concentration and Histopathologic Changes in Patients With Wilson Disease Who Are Treated With ALXN1840 for 48 Weeks Followed by an Extension Treatment Period With ALXN1840 for up to an Additional 48 Weeks
Secondary ID [1] 0 0
2019-003711-60
Secondary ID [2] 0 0
ALXN1840-WD-205
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Wilson Disease 0 0
Condition category
Condition code
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Neurological 0 0 0 0
Other neurological disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Bis-Choline Tetrathiomolybdate

Experimental: ALXN1840 - Participants will receive ALXN1840.


Treatment: Drugs: 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.

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change From Baseline At Week 48 In Liver Cu Concentration
Timepoint [1] 0 0
Baseline, Week 48
Secondary outcome [1] 0 0
Change From Baseline At Week 48 In Liver Histopathology As Assessed by Liver Biopsy
Timepoint [1] 0 0
Baseline, Week 48
Secondary outcome [2] 0 0
Pre-dose Trough ALXN1840 Concentrations In Plasma At Each Study Site Visit
Timepoint [2] 0 0
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
Secondary outcome [3] 0 0
Clinical Global Impression-Improvement Scale At Week 48
Timepoint [3] 0 0
Baseline, Week 48
Secondary outcome [4] 0 0
Change From Baseline At Week 48 In The Clinical Global Impression-Severity Scale
Timepoint [4] 0 0
Baseline, Week 48

Eligibility
Key inclusion criteria
1. Diagnosis of WD by Leipzig Criteria > 4.

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.
Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Clinical Trial Site - Concord
Recruitment postcode(s) [1] 0 0
- Concord
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Connecticut
Country [2] 0 0
United States of America
State/province [2] 0 0
Michigan
Country [3] 0 0
Austria
State/province [3] 0 0
Wien
Country [4] 0 0
Belgium
State/province [4] 0 0
Gent
Country [5] 0 0
Germany
State/province [5] 0 0
Heidelberg
Country [6] 0 0
Germany
State/province [6] 0 0
Leipzig
Country [7] 0 0
Korea, Republic of
State/province [7] 0 0
Seoul
Country [8] 0 0
New Zealand
State/province [8] 0 0
Auckland
Country [9] 0 0
Poland
State/province [9] 0 0
Warszawa
Country [10] 0 0
Russian Federation
State/province [10] 0 0
Saint Petersburg
Country [11] 0 0
Singapore
State/province [11] 0 0
Singapore
Country [12] 0 0
Spain
State/province [12] 0 0
Barcelona

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Alexion Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
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 website
https://clinicaltrials.gov/show/NCT04422431
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Alexion Pharmaceuticals Inc.
Address 0 0
Country 0 0
Phone 0 0
855-752-2356
Fax 0 0
Email 0 0
clinicaltrials@alexion.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT04422431