Chelating Agent
ALXN1840 for Wilson's Disease
Clinical Study Site, Kaohsiung, Taiwan
ALXN1840 +1 morePhase 3Waitlist AvailableResearch Sponsored by AlexionEligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Must not have
Timeline
Screening 3 weeks
Treatment Varies
Follow Upprimary evaluation period: baseline through 48 weeks; extension period: baseline through up to 60 months
Awards & highlights
No Placebo-Only Group
Pivotal Trial
Study Summary
This trial will test a new treatment for Wilson Disease, comparing it to the current standard of care. The trial will last 48 weeks, with an optional 60-month extension period.
Eligible Conditions
- Wilson's Disease
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ primary evaluation period: baseline through 48 weeks; extension period: baseline through up to 60 months
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~primary evaluation period: baseline through 48 weeks; extension period: baseline through up to 60 months
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Daily Mean Area Under The Effect-time Curve (AUEC) Of Directly Measured Non-ceruloplasmin-bound Copper (dNCC)
Secondary outcome measures
Plasma Exchange
Change From Baseline In Clinical Global Impression Severity Scale (CGI-S)
Change From Baseline In Model For End-Stage Liver Disease (MELD) Score
+11 moreSide effects data
From 2018 Phase 2 trial • 29 Patients • NCT0227359636%
Alanine aminotransferase increased
32%
Aspartate aminotransferase increased
29%
Urinary tract infection
29%
Gamma-glutamyltransferase increased
21%
Fatigue
21%
Headache
21%
Hepatic enzyme increased
18%
Tremor
14%
Rash
14%
Constipation
14%
Back pain
11%
Sinusitis
11%
Blood alkaline phosphatase increased
11%
Depression
11%
Insomnia
11%
Sleep disorder
11%
Arthralgia
11%
Leukopenia
11%
Anxiety
11%
Cough
11%
Diarrhoea
7%
Pain in extremity
7%
Plantar fasciitis
7%
Nausea
7%
Blood creatine phosphokinase increased
7%
Liver function test increased
7%
Decreased appetite
7%
Dupuytren's contracture
7%
Muscle spasms
7%
Depressed mood
7%
Dry skin
7%
Neck pain
7%
Vomiting
7%
Fall
7%
Erythema
7%
Tonsillitis
7%
Upper respiratory tract infection
7%
Influenza
7%
Myalgia
7%
Dysgeusia
7%
Paraesthesia
4%
Psychotic disorder
4%
Agranulocytosis
4%
Abnormal behaviour
4%
Gait disturbance
4%
Dysphagia
4%
Affective disorder
4%
Mania
4%
Hepato-lenticular degeneration
4%
Neurological decompensation
4%
Neutropenia
4%
Adjustment disorder
4%
Personality disorder
4%
Syncope
100%
80%
60%
40%
20%
0%
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
Find a site
Who is running the clinical trial?
AlexionLead Sponsor
242 Previous Clinical Trials
39,185 Total Patients Enrolled
Alexion PharmaceuticalsLead Sponsor
230 Previous Clinical Trials
37,565 Total Patients Enrolled
Eugene Swenson, MD, PhDStudy Director
Alexion Pharmaceuticals1 Previous Clinical Trials
29 Total Patients Enrolled
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Could you tell me how many hospitals are testing this procedure?
"In addition to Children's Hospital of Philadelphia in Los Angeles, California, Texas Children's Hospital in Philadelphia, Pennsylvania, and Yale University School of Medicine in Nashville, Tennessee, this study is also enrolling at 20 other locations."
Answered by AI
Could you please elaborate on the safety of ALXN1840 for human patients?
"There is enough evidence from prior clinical trials to give ALXN1840 a score of 3 for safety."
Answered by AI