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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05047523




Registration number
NCT05047523
Ethics application status
Date submitted
9/09/2021
Date registered
17/09/2021
Date last updated
23/10/2024

Titles & IDs
Public title
Study of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease
Scientific title
A Multicenter, Randomized, Controlled, Open-label, Rater-blinded Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease
Secondary ID [1] 0 0
2021-001015-82
Secondary ID [2] 0 0
ALXN1840-WD-302
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
Treatment: Drugs - Standard of Care

Experimental: ALXN1840 - ALXN1840 will be administered at one of two starting doses, with incremental dose increases permitted.

Active comparator: Standard of Care - Participants will receive their current therapy or initiate Standard of Care therapy.


Treatment: Drugs: ALXN1840
Administered as an oral tablet.

Treatment: Drugs: Standard of Care
Depending on the site/region, participants randomized to receive Standard of Care treatment will receive trientine, penicillamine, zinc, or a combination of these medicines, administered according to standard regimens.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percent Change From Baseline to Week 48 in Non-ceruloplasmin-bound Copper in Plasma
Timepoint [1] 0 0
Baseline, Week 48
Secondary outcome [1] 0 0
Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Primary Evaluation Period
Timepoint [1] 0 0
Baseline up to Week 48
Secondary outcome [2] 0 0
Area Under the Effect Versus Time Curve (AUEC) for Plasma Total Copper and Direct NCC
Timepoint [2] 0 0
Week 48
Secondary outcome [3] 0 0
Maximum Observed Concentration (Cmax) of ALXN1840 for Plasma Total Molybdenum and Plasma Ultrafiltrate Molybdenum Concentrations
Timepoint [3] 0 0
Week 48
Secondary outcome [4] 0 0
Area Under the Plasma Concentration Versus Time Curve From Time 0 to the End of the Dosing Interval (AUCtau) of ALXN1840 for Plasma Total Molybdenum and Plasma Ultrafiltrate Molybdenum
Timepoint [4] 0 0
Week 48

Eligibility
Key inclusion criteria
Key

1. Diagnosis of Wilson Disease by Leipzig Score = 4.
2. Adequate venous access to allow collection of required blood samples.
3. Able to swallow intact ALXN1840 tablets or mini-tablets.
4. Willing to avoid intake of foods and drinks with high contents of copper.
5. Willing and able to follow protocol-specified contraception requirements.

Key
Minimum age
3 Years
Maximum age
17 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Decompensated hepatic cirrhosis or MELD score > 13 (ages 12 to <18) or PELD score > 13 (ages 3 to < 12).
2. Modified Nazer score > 7.
3. Clinically significant gastrointestinal bleed within past 3 months.
4. Alanine aminotransferase (ALT) > 2 × upper limit of normal (ULN) for participants treated for > 28 days with WD therapy or ALT > 5 × ULN for treatment-naïve participants or participants who have been treated for = 28 days.
5. Marked neurological disease requiring either nasogastric feeding tube or intensive inpatient medical care.
6. Hemoglobin less than lower limit of the reference range for age and sex.
7. History of seizure activity within 6 months prior to informed consent/assent.
8. Participants in renal failure, defined as in end-stage renal disease on dialysis (chronic kidney disease stage 5) or estimated glomerular filtration rate < 30 milliliters/minute/1.73 meter squared.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled 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
Blinded (masking used)
Who is / are masked / blinded?


The people assessing the outcomes
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
13/09/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
26/06/2023
Sample size
Target
Accrual to date
Final
40
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Research Site - Parkville
Recruitment hospital [2] 0 0
Research Site - South Brisbane
Recruitment postcode(s) [1] 0 0
VIC 3052 - Parkville
Recruitment postcode(s) [2] 0 0
4101 - South Brisbane
Recruitment outside Australia
Country [1] 0 0
France
State/province [1] 0 0
Lille
Country [2] 0 0
France
State/province [2] 0 0
Toulouse
Country [3] 0 0
Germany
State/province [3] 0 0
Hannover
Country [4] 0 0
Germany
State/province [4] 0 0
Tübingen
Country [5] 0 0
Japan
State/province [5] 0 0
Kumamoto-shi
Country [6] 0 0
Japan
State/province [6] 0 0
Kurume-shi
Country [7] 0 0
Japan
State/province [7] 0 0
Meguro-ku
Country [8] 0 0
Japan
State/province [8] 0 0
Sapporo-shi
Country [9] 0 0
Korea, Republic of
State/province [9] 0 0
Seoul
Country [10] 0 0
Poland
State/province [10] 0 0
Warsaw
Country [11] 0 0
Spain
State/province [11] 0 0
Barcelona
Country [12] 0 0
Spain
State/province [12] 0 0
Esplugues de Llobregat
Country [13] 0 0
Spain
State/province [13] 0 0
Las Palmas de Gran Canaria
Country [14] 0 0
Spain
State/province [14] 0 0
Madrid
Country [15] 0 0
Spain
State/province [15] 0 0
Málaga
Country [16] 0 0
Spain
State/province [16] 0 0
Pamplona

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

Ethics approval
Ethics application status

Summary
Brief summary
This study is being conducted to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics of ALXN1840 versus standard of care in pediatric participants with Wilson disease (WD).
Trial website
https://clinicaltrials.gov/study/NCT05047523
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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT05047523