A Phase 3 clinical study to Evaluate the Efficacy and Safety of ALXN1840 in adult and adolescent patients who suffer from Wilson Disease.

A Phase 3, Randomized, Rater-Blinded, Multi-Center Study To Evaluate The Efficacy And Safety Of ALXN1840 Administered For 48 Weeks Versus Standard Of Care In Patients With Wilson Disease Aged 12 Years And Older With An Extension Period Of Up To 60 Months

Status

Not yet recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2017-004135-36-DE

Enrollment

180

Registered

2017-12-22

Start date

2018-03-16

Completion date

Unknown

Last updated

2021-05-10

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Wilson Disease MedDRA version: 20.0 Level: LLT Classification code 10047988 Term: Wilson's disease System Organ Class: 100000004850

Interventions

Product Name: ALXN1840 Pharmaceutical Form: Tablet INN or Proposed INN: Not applied CAS Number: 649749-10-0 Current Sponsor code: ALXN1840 Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE Concen

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: - Established diagnosis of WD by Leipzig-Score > 4 documented by testing as outlined in the 2012 European Association for the Study of Liver WD Clinical Practice Guidelines; - 12 years of age or older at time of informed consent/assent (18 years and older in Germany); - Willing and able to give written informed consent and comply with the study visit schedule. For patients 48 hours immediately prior to first study assessment on Day 1; - Adequate venous access to allow collection of required blood samples; - Willing to avoid use of vitamins and/or minerals containing copper, zinc, or molybdenum throughout the study duration; - Willing to avoid intake of foods and drinks with high contents of copper throughout the study duration; - Female patients of childbearing potential must be willing to follow guidance for highly effective contraception Please see the study protocol for details Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 170 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 10

Exclusion criteria

Exclusion criteria: - Decompensated hepatic cirrhosis; - MELD score > 13; - Participation in a clinical study of an experimental or unapproved/unlicensed therapy at the same time or within the 4 weeks prior to this Screening Visit; - Pregnant (or women who are planning to become pregnant) or lactating women; - Major systemic disease or other illness that would, in the opinion of the Investigator, compromise subject safety or interfere with the collection or interpretation of study results; - Modified Nazer score > 7 - Hemoglobine 2 × ULN for subjects treated for >28 days with WD therapy (Cohort 1); - Alanine aminotransferase > 5 × ULN for treatment naïve subjects or subjects who have been treated for = 28 days (Cohort 2); - Patients with end-stage renal disease on dialysis (chronic kidney disease stage 5 [CKD 5)]) or creatinine clearance < 30 mL/min Please see study protocol for more details

Design outcomes

Primary

Measure	Time frame
Main Objective: The primary objective is to evaluate the efficacy of ALXN1840 administered for 48 weeks, compared to standard of care (SoC), on copper control in WD subjects aged 12 and older;Secondary Objective: The secondary objectives are to: -Establish the safety and tolerability of individualized dosing of ALXN1840; -Evaluate the effects of ALXN1840 on disability status; -Evaluate the effects of ALXN1840 on neurological status; -Evaluate the global effects of ALXN1840 on global clinical symptoms; -Evaluate the effects of ALXN1840 on hepatic status; -Evaluate the efficacy of ALXN1840 administered for 48 weeks, compared to SoC, on copper control in WD patients aged 12 years and older; -Evaluate the effects of ALXN1840 on the cNCC responder rate Please see the study protocol for more details;Primary end point(s): Daily mean area under the effect-time curve (AUEC) of directly measured non-ceruloplasmin-bound copper (dNCC) from 0 to 48 weeks ;Timepoint(s) of evaluation of this end point: 48 weeks	—

Measure

Time frame

Main Objective: The primary objective is to evaluate the efficacy of ALXN1840 administered for 48 weeks, compared to standard of care (SoC), on copper control in WD subjects aged 12 and older;Secondary Objective: The secondary objectives are to: -Establish the safety and tolerability of individualized dosing of ALXN1840; -Evaluate the effects of ALXN1840 on disability status; -Evaluate the effects of ALXN1840 on neurological status; -Evaluate the global effects of ALXN1840 on global clinical symptoms; -Evaluate the effects of ALXN1840 on hepatic status; -Evaluate the efficacy of ALXN1840 administered for 48 weeks, compared to SoC, on copper control in WD patients aged 12 years and older; -Evaluate the effects of ALXN1840 on the cNCC responder rate Please see the study protocol for more details;Primary end point(s): Daily mean area under the effect-time curve (AUEC) of directly measured non-ceruloplasmin-bound copper (dNCC) from 0 to 48 weeks ;Timepoint(s) of evaluation of this end point: 48 weeks

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Secondary

Measure	Time frame
Secondary end point(s): The secondary endpoints include the following: a. Incidence of • Adverse events/serious adverse events (AE/SAEs) • Clinical laboratory test data • Neurological and physical examination findings • 12-lead electrocardiogram (ECG) data • Vital signs b. Change from baseline in the Unified Wilson Disease Rating Scale (UWDRS) Part II total score; c. Change from baseline in UWDRS Part III total score and individual item/subscales (arising from a chair, gait, handwriting, and speech); d. Clinical Global Impression-Improvement Scale (CGI-I) e. Change from baseline in Clinical Global Impression-Severity Scale (CGI-S) f. Change from baseline in Model for End-Stage Liver Disease (MELD) score; g. Absolute change from baseline (Day 1) to 48 weeks in calculated NCC (cNCC) in plasma and percentage change from baseline in cNCC in plasma. For ALXN1840-treated patients, the cNCC in plasma will be corrected for the amount of copper bound to the ALXN1840 tripartite complex (TPC) (cNCCcorrected); h. cNCC responder rate at 48 weeks;Timepoint(s) of evaluation of this end point: 48 weeks	—

Measure

Time frame

Secondary end point(s): The secondary endpoints include the following: a. Incidence of • Adverse events/serious adverse events (AE/SAEs) • Clinical laboratory test data • Neurological and physical examination findings • 12-lead electrocardiogram (ECG) data • Vital signs b. Change from baseline in the Unified Wilson Disease Rating Scale (UWDRS) Part II total score; c. Change from baseline in UWDRS Part III total score and individual item/subscales (arising from a chair, gait, handwriting, and speech); d. Clinical Global Impression-Improvement Scale (CGI-I) e. Change from baseline in Clinical Global Impression-Severity Scale (CGI-S) f. Change from baseline in Model for End-Stage Liver Disease (MELD) score; g. Absolute change from baseline (Day 1) to 48 weeks in calculated NCC (cNCC) in plasma and percentage change from baseline in cNCC in plasma. For ALXN1840-treated patients, the cNCC in plasma will be corrected for the amount of copper bound to the ALXN1840 tripartite complex (TPC) (cNCCcorrected); h. cNCC responder rate at 48 weeks;Timepoint(s) of evaluation of this end point: 48 weeks

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Countries

Australia, Austria, Belgium, Brazil, Canada, Colombia, Croatia, Czechia, Denmark, France, Germany, Greece, Hong Kong, Hungary, Israel, Japan, Korea, Democratic People's Republic of, Netherlands, New Zealand, Poland, Portugal, Russian Federation, Serbia, Singapore, Spain, Sweden, Taiwan, Turkey, United Kingdom, United States

Contacts

Public ContactEuropean Clinical Trial Information

Alexion Europe SAS

clinicaltrials.eu@alexion.com+33787148158

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026