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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04573309




Registration number
NCT04573309
Ethics application status
Date submitted
28/09/2020
Date registered
5/10/2020

Titles & IDs
Public title
Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840
Scientific title
A Phase 2, Open-label Study to Assess Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840
Secondary ID [1] 0 0
2020-001104-41
Secondary ID [2] 0 0
ALXN1840-WD-204
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 - ALXN1840

Experimental: ALXN1840 - Participants will be administered ALXN1840 at a dose of 15 milligrams (mg)/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39


Treatment: Drugs: ALXN1840
Administered orally as tablets.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Mean Daily Copper Balance: Day 1 Through Day 8
Timepoint [1] 0 0
Accumulation: Day 1 through Day 8 (ALXN1840 15 mg)
Primary outcome [2] 0 0
Mean Daily Copper Balance: Day 31 Through Day 35
Timepoint [2] 0 0
Accumulation: Day 31 through Day 35 (ALXN1840 30 mg)
Primary outcome [3] 0 0
Mean Daily Copper Balance: Day 25 Through Day 28
Timepoint [3] 0 0
Accumulation: Day 25 through Day 28 (ALXN1840 15 mg)
Primary outcome [4] 0 0
Mean Daily Copper Balance: Day 36 Through Day 39
Timepoint [4] 0 0
Accumulation: Day 36 through Day 39 (ALXN1840 30 mg)
Secondary outcome [1] 0 0
Change From Baseline In Mean Daily Copper Balance
Timepoint [1] 0 0
Accumulation: Baseline, Day 1 through Day 8 (ALXN1840 15 mg) and Day 31 through Day 35 (ALXN1840 30 mg); Steady State: Baseline, Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg)
Secondary outcome [2] 0 0
Copper Quantified In Food, Drink, Feces, And Urine, Including Plasma Total And Labile Bound Copper (LBC)
Timepoint [2] 0 0
Accumulation: Day 1 through Day 8 for 15 mg and Day 31 through Day 35 for 30 mg; Steady state: Day 25 through Day 28 for ALXN1840 15 mg and Day 36 through Day 39 for ALXN1840 30 mg
Secondary outcome [3] 0 0
Molybdenum Specified In ALXN1840 Doses Given And Quantified In Food, Drink, Feces, And Urine, Including Plasma At Steady State
Timepoint [3] 0 0
Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg)
Secondary outcome [4] 0 0
Change From Baseline In Total Molybdenum Excretion In Urine And Feces
Timepoint [4] 0 0
Accumulation: Baseline, Day 1 through Day 8 (ALXN1840 15 mg) and Day 31 through Day 35 (ALXN1840 30 mg); Steady State: Baseline, Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg)
Secondary outcome [5] 0 0
Mean Daily Molybdenum Balance At ALXN1840 Steady State
Timepoint [5] 0 0
Steady state: Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg)
Secondary outcome [6] 0 0
Accumulation Of Molybdenum As Determined By Molybdenum Balance
Timepoint [6] 0 0
Accumulation: Day 1 through Day 8 (ALXN1840 15 mg) and Day 31 through Day 35 ((ALXN1840 30 mg)
Secondary outcome [7] 0 0
Maximum Observed Plasma Concentration (Cmax) of Total Molybdenum and Plasma Ultrafiltrate (PUF) Molybdenum
Timepoint [7] 0 0
Day 1 up to Day 39
Secondary outcome [8] 0 0
Area Under The Concentration Time Curve (AUC0-inf) of Total Molybdenum and Plasma Ultrafiltrate (PUF) Molybdenum
Timepoint [8] 0 0
Day 39

Eligibility
Key inclusion criteria
1. Diagnosis of WD by Leipzig Criteria = 4.
2. Able to reside in the clinical research unit for intensive metabolic monitoring of copper and molybdenum.
3. Participants willing to adhere to copper/molybdenum-controlled diet during the study.
4. Willing and able to follow protocol-specified contraception requirements.
5. Capable of giving signed informed consent.
Minimum age
18 Years
Maximum age
No limit
Sex
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. Hemoglobin less than lower limit of the reference range for age and sex.
6. Significant medical history (current or past).
7. Previous treatment with zinc within 30 days prior to the Screening Visit.
8. Participants in renal failure, defined as in end-stage renal disease on dialysis (chronic kidney disease stage 5) or creatinine clearance < 30 milliliters/minute.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
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
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
7/09/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
7/06/2022
Sample size
Target
Accrual to date
Final
9
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Connecticut
Country [2] 0 0
New Zealand
State/province [2] 0 0
Grafton
Country [3] 0 0
United Kingdom
State/province [3] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Alexion Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Eugene S. Swenson, MD, PhD
Address 0 0
Alexion Pharmaceuticals, Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT04573309