Wilson DiseasePhase 3NCT05047523
Study of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease
A Phase 3 study testing ALXN1840 in people with Wilson Disease, run by Alexion Pharmaceuticals, Inc. at 20 sites in 7 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 3 to 17 years
- Conditions the study is for
- Wilson Disease
- What is being tested?
- ALXN1840
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 20 sitesin 7 countries
- Study dates
- 13 Sep 2021to 26 Jun 2023
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
20 sites in 7 countries.
20 sites in 7 countries
Spain6 sites
Research Site
Barcelona, 08035
Research Site
Esplugues de Llobregat, 8950
Research Site
Las Palmas de Gran Canaria, 35016
Research Site
Madrid, 28041
Research Site
Málaga, 29011
Research Site
Pamplona, 31008
Japan4 sites
Research Site
Kumamoto, 860-8556
Research Site
Kurume-shi, 830-0011
Research Site
Meguro-ku, 153-8515
Research Site
Sapporo, 063-0005
South Korea3 sites
Research Site
Seoul, 03080
Research Site
Seoul, 03722
Research Site
Seoul, 06351
Australia2 sites
Research Site
Parkville, VIC 3052
Research Site
South Brisbane, 4101
About this study
Official 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
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).
Read the detailed description
Participants who complete the 48 weeks of treatment in Period 1 will have the option to receive ALXN1840 for 24 weeks in Period 2 (open-label extension).
Safety will be monitored throughout the study.
- Conditions
- Wilson Disease
- Being tested
- ALXN1840, Standard of Care
- Keywords
- Wilson Disease, Pediatric
- Medical subject headings
- Hepatolenticular Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALXN1840Drug
Also called Formerly WTX101
Administered as an oral tablet.
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.
| Study group | Role | What they receive |
|---|---|---|
| ALXN1840 | Experimental | ALXN1840 will be administered at one of two starting doses, with incremental dose increases permitted. Receives: Drug: ALXN1840 |
| Standard of Care | Active comparator | Participants will receive their current therapy or initiate Standard of Care therapy. Receives: Drug: Standard of Care |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline, Week 48
Secondary outcomes (4)
Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Primary Evaluation Period
An adverse event (AE) was any untoward medical occurrence in a participant administered the study drug and which did not necessarily have a causal relationship with this treatment. TEAEs were defined as AEs with onset after the first dose of study intervention or existing events that worsened in severity after the first dose of study intervention. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
Measured: Baseline up to Week 48
Area Under the Effect Versus Time Curve (AUEC) for Plasma Total Copper and Direct NCC
Measured: Week 48
Maximum Observed Concentration (Cmax) of ALXN1840 for Plasma Total Molybdenum and Plasma Ultrafiltrate Molybdenum Concentrations
Measured: 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
Measured: Week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Sep 2021
- Study start13 Sep 2021
- Primary completion26 Jun 2023
- Study completion26 Jun 2023
Why it stopped: Sponsor decision to terminate the program
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Wilson DiseaseStudies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Eugene S. Swenson, MD, PhD, Study director, Alexion Pharmaceuticals, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05047523
- Sponsor's protocol ID
- ALXN1840-WD-302
- Other IDs
- 2021-001015-82
- Status last verified
- Oct 2024
- First submitted
- 9 Sep 2021
- Last updated
- 23 Oct 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT05047523
- Acronym / sponsor ID
- ALXN1840-WD-302
- Conditions
- Wilson Disease
- Keywords
- Wilson Disease, Pediatric
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor decision to terminate the program
- Enrolment
- 40 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 13 Sep 2021 (actual)
- Primary completion
- 26 Jun 2023 (actual)
- Study completion
- 26 Jun 2023 (actual)
- First posted
- 17 Sep 2021 (actual)
- Results
- Posted 15 Oct 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
Participants who complete the 48 weeks of treatment in Period 1 will have the option to receive ALXN1840 for 24 weeks in Period 2 (open-label extension).
Safety will be monitored throughout the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ALXN1840 | Experimental | Drug: ALXN1840ALXN1840 will be administered at one of two starting doses, with incremental dose increases permitted. |
| Standard of Care | Active comparator | Drug: Standard of CareParticipants will receive their current therapy or initiate Standard of Care therapy. |
Who may qualify
- Ages
- 3 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ALXN1840Also: Formerly WTX101Administered as an oral tablet. | Drug | ALXN1840 |
| Standard of CareDepending 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. | Drug | Standard of Care |
Primary outcome
Percent Change From Baseline to Week 48 in Non-ceruloplasmin-bound Copper in Plasma
Time frame: Baseline, Week 48
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.
4 secondary outcomes
Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Primary Evaluation Period
Time frame: Baseline up to Week 48
Area Under the Effect Versus Time Curve (AUEC) for Plasma Total Copper and Direct NCC
Time frame: Week 48
Maximum Observed Concentration (Cmax) of ALXN1840 for Plasma Total Molybdenum and Plasma Ultrafiltrate Molybdenum Concentrations
Time frame: 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
Time frame: Week 48
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Oct 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Related InfoLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Eugene S. Swenson, MD, PhD study director
Alexion Pharmaceuticals, Inc.
20 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing ALXN1840 in people with Wilson Disease, run by Alexion Pharmaceuticals, Inc. at 20 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 23 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in Spain, Japan, South Korea, Australia, France and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 15 Oct 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percent Change From Baseline to Week 48 in Non-ceruloplasmin-bound Copper in Plasma (time frame: Baseline, Week 48). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Oct 2024.