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Chelating Agent

Efficacy and Safety of ALXN1840 Administered for 48 Weeks Versus Standard of Care in Participants With Wilson Disease

Phase 3

Waitlist Available

Research Sponsored by Alexion

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up week 48

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial tests ALXN1840, a new medicine for Wilson Disease, in patients aged 12 and older. It aims to see if it works better than usual treatments by helping remove excess copper from the body. ALXN1840 is a new treatment for Wilson Disease, which traditionally involves chelation therapy to remove excess copper.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ week 48

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~week 48

This trial's timeline: 3 weeks for screening, Varies for treatment, and week 48 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Daily Mean Area Under The Effect-time Curve (AUEC) of Directly Measured Non-ceruloplasmin-bound Copper (dNCC) From 0 to 48 Weeks (dNCC AUEC0-48W)

Secondary study objectives

Absolute Change From Baseline in Calculated Non-Ceruloplasmin Bound Copper (cNCC) or Calculated Non-Ceruloplasmin Bound Copper Corrected (cNCCcorrected) in Plasma at Week 48

Change From Baseline in Clinical Global Impression Severity Scale (CGI-S) Score at Week 48

Change From Baseline in Model for End-Stage Liver Disease (MELD) Score at Week 48

+8 more

Side effects data

From 2018 Phase 2 trial • 29 Patients • NCT02273596

36%

Alanine aminotransferase increased

32%

Aspartate aminotransferase increased

29%

Urinary tract infection

29%

Gamma-glutamyltransferase increased

21%

Hepatic enzyme increased

21%

Headache

21%

Fatigue

18%

Tremor

14%

Back pain

14%

Constipation

14%

Rash

11%

Insomnia

11%

Sleep disorder

11%

Diarrhoea

11%

Arthralgia

11%

Depression

11%

Cough

11%

Anxiety

11%

Leukopenia

11%

Sinusitis

11%

Blood alkaline phosphatase increased

Blood creatine phosphokinase increased

Dysgeusia

Muscle spasms

Dupuytren's contracture

Vomiting

Dry skin

Decreased appetite

Myalgia

Fall

Erythema

Pain in extremity

Plantar fasciitis

Paraesthesia

Nausea

Liver function test increased

Neck pain

Depressed mood

Influenza

Tonsillitis

Upper respiratory tract infection

Syncope

Mania

Dysphagia

Neutropenia

Psychotic disorder

Neurological decompensation

Abnormal behaviour

Adjustment disorder

Personality disorder

Gait disturbance

Agranulocytosis

Hepato-lenticular degeneration

Affective disorder

100%

80%

60%

40%

20%

Study treatment Arm

ALXN1840

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Active Control

Group I: ALXN1840Experimental Treatment1 Intervention

ALXN1840 was administered orally for 48 weeks at doses ranging from 15 milligrams (mg) every other day (QOD) up to a titrated dose of 60 mg daily. Participants who completed the Primary Evaluation Period had the option to participate in the up to 60-month Extension Period.

Group II: Standard of Care (SoC) MedicationActive Control1 Intervention

SoC medication was administered for 48 weeks. Participants who completed the Primary Evaluation Period had the option to participate in the up to 60-month Extension Period.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

ALXN1840

2020

Completed Phase 2

~340

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