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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
14/12/2023

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
Percentage Change From Baseline To Week 48 In Non-ceruloplasmin-bound Copper (NCC) In Plasma
Timepoint [1] 0 0
Baseline, Week 48
Secondary outcome [1] 0 0
Area Under The Effect Versus Time Curve (AUEC) For NCC And Plasma Total Copper
Timepoint [1] 0 0
Week 48
Secondary outcome [2] 0 0
Observed Change From Baseline To Week 48 Of Ceruloplasmin-bound Copper And Ceruloplasmin
Timepoint [2] 0 0
Baseline, Week 48
Secondary outcome [3] 0 0
NCC Responder Rate
Timepoint [3] 0 0
Week 48
Secondary outcome [4] 0 0
Change From Baseline To Week 48 In The UWDRS Part II Total Score
Timepoint [4] 0 0
Baseline, Week 48
Secondary outcome [5] 0 0
Change From Baseline To Week 48 In The UWDRS Part III Total Score
Timepoint [5] 0 0
Baseline, Week 48
Secondary outcome [6] 0 0
PK: Maximum Observed Concentration (Cmax) Of ALXN1840 For Plasma Total Molybdenum And Plasma Ultrafiltrate Molybdenum Concentrations
Timepoint [6] 0 0
Up to Week 48
Secondary outcome [7] 0 0
PK: Time To Maximum Concentration (Tmax) Of ALXN1840 For Plasma Total Molybdenum And Plasma Ultrafiltrate Molybdenum Concentrations
Timepoint [7] 0 0
Up to Week 48
Secondary outcome [8] 0 0
PK: 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 Concentrations
Timepoint [8] 0 0
Up to Week 48
Secondary outcome [9] 0 0
Clinical Global Impression-improvement (CGI-I), As Assessed By The Investigator
Timepoint [9] 0 0
Week 48
Secondary outcome [10] 0 0
Change From Baseline To Week 48 In Clinical Global Impression-severity (CGI-S), As Assessed By The Investigator
Timepoint [10] 0 0
Baseline, Week 48
Secondary outcome [11] 0 0
Change From Baseline To Week 48 In Model For End-stage Liver Disease (MELD) Score (Ages 12 Years And Older) Or Pediatric End-stage Liver Disease (PELD) Score (Ages 3 To < 12 Years)
Timepoint [11] 0 0
Baseline, Week 48
Secondary outcome [12] 0 0
Change From Baseline To Week 48 In Modified Nazer Score
Timepoint [12] 0 0
Baseline, 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
6/10/2021
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
Clinical Trial Site - Parkville
Recruitment hospital [2] 0 0
Clinical Trial 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
Bron
Country [2] 0 0
France
State/province [2] 0 0
Lille
Country [3] 0 0
France
State/province [3] 0 0
Paris
Country [4] 0 0
France
State/province [4] 0 0
Toulouse
Country [5] 0 0
Germany
State/province [5] 0 0
Hamburg
Country [6] 0 0
Germany
State/province [6] 0 0
Hanover
Country [7] 0 0
Germany
State/province [7] 0 0
Tuebingen
Country [8] 0 0
Japan
State/province [8] 0 0
Chiba
Country [9] 0 0
Japan
State/province [9] 0 0
Kumamoto
Country [10] 0 0
Japan
State/province [10] 0 0
Kurume
Country [11] 0 0
Japan
State/province [11] 0 0
Meguro-Ku
Country [12] 0 0
Japan
State/province [12] 0 0
Sapporo
Country [13] 0 0
Korea, Republic of
State/province [13] 0 0
Seoul
Country [14] 0 0
Poland
State/province [14] 0 0
Warsaw
Country [15] 0 0
Spain
State/province [15] 0 0
Barcelona
Country [16] 0 0
Spain
State/province [16] 0 0
Las Palmas de Gran Canaria
Country [17] 0 0
Spain
State/province [17] 0 0
Madrid
Country [18] 0 0
Spain
State/province [18] 0 0
Malaga
Country [19] 0 0
Spain
State/province [19] 0 0
Pamplona
Country [20] 0 0
United Kingdom
State/province [20] 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

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT05047523