NCT05047523

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 Health

63
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
40

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Sep 2021

Geographic Reach
7 countries

20 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

September 9, 2021

Completed
4 days until next milestone

Study Start

First participant enrolled

September 13, 2021

Completed
4 days until next milestone

First Posted

Study publicly available on registry

September 17, 2021

Completed
1.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 26, 2023

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 26, 2023

Completed
1.3 years until next milestone

Results Posted

Study results publicly available

October 15, 2024

Completed
Last Updated

October 23, 2024

Status Verified

October 1, 2024

Enrollment Period

1.8 years

First QC Date

September 9, 2021

Results QC Date

June 20, 2024

Last Update Submit

October 21, 2024

Conditions

Wilson Disease

Keywords

Wilson DiseasePediatric

Outcome Measures

Primary Outcomes (1)

  • Percent Change From Baseline to Week 48 in Non-ceruloplasmin-bound Copper in Plasma

    Plasma samples were planned to be collected to measure ceruloplasmin-bound copper. Due to the early termination of the study, data for this Outcome Measure were not collected for any of the cohorts.

    Baseline, Week 48

Secondary Outcomes (4)

  • Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Primary Evaluation Period

    Baseline up to Week 48

  • Area Under the Effect Versus Time Curve (AUEC) for Plasma Total Copper and Direct NCC

    Week 48

  • Maximum Observed Concentration (Cmax) of ALXN1840 for Plasma Total Molybdenum and Plasma Ultrafiltrate Molybdenum Concentrations

    Week 48

  • 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

    Week 48

Study Arms (2)

ALXN1840

EXPERIMENTAL

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

Drug: ALXN1840

Standard of Care

ACTIVE COMPARATOR

Participants will receive their current therapy or initiate Standard of Care therapy.

Drug: Standard of Care

Interventions

ALXN1840DRUG

Administered as an oral tablet.

Also known as: Formerly WTX101
ALXN1840
Standard of CareDRUG

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.

Standard of Care

Eligibility Criteria

Age3 Years - 17 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

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

You may not qualify if:

  • Decompensated hepatic cirrhosis or MELD score \> 13 (ages 12 to \<18) or PELD score \> 13 (ages 3 to \< 12).
  • Modified Nazer score \> 7.
  • Clinically significant gastrointestinal bleed within past 3 months.
  • 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.
  • Marked neurological disease requiring either nasogastric feeding tube or intensive inpatient medical care.
  • Hemoglobin less than lower limit of the reference range for age and sex.
  • History of seizure activity within 6 months prior to informed consent/assent.
  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (20)

Research Site

Parkville, VIC 3052, Australia

Location

Research Site

South Brisbane, 4101, Australia

Location

Research Site

Lille, 59037, France

Location

Research Site

Toulouse, 31059, France

Location

Research Site

Hanover, 30625, Germany

Location

Research Site

Tübingen, 72076, Germany

Location

Research Site

Kumamoto, 860-8556, Japan

Location

Research Site

Kurume-shi, 830-0011, Japan

Location

Research Site

Meguro-ku, 153-8515, Japan

Location

Research Site

Sapporo, 063-0005, Japan

Location

Research Site

Warsaw, 04-730, Poland

Location

Research Site

Seoul, 03080, South Korea

Location

Research Site

Seoul, 03722, South Korea

Location

Research Site

Seoul, 06351, South Korea

Location

Research Site

Barcelona, 08035, Spain

Location

Research Site

Esplugues de Llobregat, 8950, Spain

Location

Research Site

Las Palmas de Gran Canaria, 35016, Spain

Location

Research Site

Madrid, 28041, Spain

Location

Research Site

Málaga, 29011, Spain

Location

Research Site

Pamplona, 31008, Spain

Location

Related Links

MeSH Terms

Conditions

Hepatolenticular Degeneration

Interventions

Standard of Care

Condition Hierarchy (Ancestors)

Liver DiseasesDigestive System DiseasesBasal Ganglia DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicMovement DisordersHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolism, Inborn ErrorsMetal Metabolism, Inborn ErrorsMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Quality Indicators, Health CareQuality of Health CareHealth Services AdministrationHealth Care Quality, Access, and Evaluation

Limitations and Caveats

Due to early termination of the study, data for the efficacy endpoints were not collected.

Results Point of Contact

Title
Alexion Pharmaceuticals, Inc.
Organization
Alexion Pharmaceuticals, Inc.
clinicaltrials@alexion.com
+1.855.752.2356

Study Officials

  • Eugene S. Swenson, MD, PhD

    Alexion Pharmaceuticals, Inc.

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
SINGLE
Who Masked
OUTCOMES ASSESSOR
Masking Details
This study is rater-blinded for the Unified Wilson Disease Rating Scale (UWDRS) assessment only.
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 9, 2021

First Posted

September 17, 2021

Study Start

September 13, 2021

Primary Completion

June 26, 2023

Study Completion

June 26, 2023

Last Updated

October 23, 2024

Results First Posted

October 15, 2024

Record last verified: 2024-10

Locations

ES(6)DE(2)JP(4)PL(1)AU(2)FR(2)KR(3)