Wilson DiseasePhase 2NCT04573309
Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840
A Phase 2 study testing ALXN1840 in people with Wilson Disease, run by Alexion Pharmaceuticals, Inc. at 3 sites in 3 countries.
Trial summary
This exploratory study will investigate the effects of ALXN1840 on copper balance in participants with Wilson disease (WD). Read more about this study
- Who is it for?
- Ages 18 years and older
- 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
- 9 participants
- Where
- 3 sitesin 3 countries
- Study dates
- 7 Sep 2020to 7 Jun 2022
- Phase
- Phase 2
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of WD by Leipzig Criteria ≥ 4.
- Able to reside in the clinical research unit for intensive metabolic monitoring of copper and molybdenum.
- Participants willing to adhere to copper/molybdenum-controlled diet during the study.
- Willing and able to follow protocol-specified contraception requirements.
- Capable of giving signed informed consent.
Who cannot join
- Decompensated cirrhosis or model for end stage liver disease score > 13.
- Modified Nazer score > 7.
- Clinically significant gastrointestinal bleed within past 3 months.
- Alanine aminotransferase > 2 × upper limit of normal.
- Hemoglobin less than lower limit of the reference range for age and sex.
- Significant medical history (current or past).
- Previous treatment with zinc within 30 days prior to the Screening Visit.
- Participants in renal failure, defined as in end-stage renal disease on dialysis (chronic kidney disease stage 5) or creatinine clearance < 30 milliliters/minute.
Trial locations and contacts
3 sites in 3 countries.
3 sites in 3 countries
About this study
Official title: A Phase 2, Open-label Study to Assess Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840
This exploratory study will investigate the effects of ALXN1840 on copper balance in participants with Wilson disease (WD).
Read the detailed description
Participants who are treatment experienced (which includes standard-of-care therapies or ALXN1840) and treatment naïve are eligible for this study.
- Conditions
- Wilson Disease
- Being tested
- ALXN1840
- Keywords
- Copper Balance, Molybdenum Balance, Wilson Disease, ALXN1840
- 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 orally as tablets.
| Study group | Role | What they receive |
|---|---|---|
| ALXN1840 | Experimental | Participants will be administered ALXN1840 at a dose of 15 milligrams (mg)/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39 Receives: Drug: ALXN1840 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean Daily Copper Balance: Day 1 Through Day 8
Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period.
Measured: Accumulation: Day 1 through Day 8 (ALXN1840 15 mg)
Mean Daily Copper Balance: Day 31 Through Day 35
Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period.
Measured: Accumulation: Day 31 through Day 35 (ALXN1840 30 mg)
Mean Daily Copper Balance: Day 25 Through Day 28
Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period.
Measured: Accumulation: Day 25 through Day 28 (ALXN1840 15 mg)
Mean Daily Copper Balance: Day 36 Through Day 39
Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period.
Measured: Accumulation: Day 36 through Day 39 (ALXN1840 30 mg)
Secondary outcomes (8)
Change From Baseline In Mean Daily Copper Balance
Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period.
Measured: Accumulation: Baseline, Day 1 through Day 8 (ALXN1840 15 mg) and Day 31 through Day 35 (ALXN1840 30 mg); Steady State: Baseline, Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg)
Copper Quantified In Food, Drink, Feces, And Urine, Including Plasma Total And Labile Bound Copper (LBC)
Copper was assessed through measurement of copper intake (in food and drink), and copper output (in feces and urine) as well as plasma total and labile bound copper.
Measured: Accumulation: Day 1 through Day 8 for 15 mg and Day 31 through Day 35 for 30 mg; Steady state: Day 25 through Day 28 for ALXN1840 15 mg and Day 36 through Day 39 for ALXN1840 30 mg
Molybdenum Specified In ALXN1840 Doses Given And Quantified In Food, Drink, Feces, And Urine, Including Plasma At Steady State
The amount of molybdenum in food, drink, feces and urine is reported in this outcome measure.
Measured: Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg)
Change From Baseline In Total Molybdenum Excretion In Urine And Feces
Measured: Accumulation: Baseline, Day 1 through Day 8 (ALXN1840 15 mg) and Day 31 through Day 35 (ALXN1840 30 mg); Steady State: Baseline, Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg)
Mean Daily Molybdenum Balance At ALXN1840 Steady State
Molybdenum balance at steady state was assessed through measurement of molybdenum intake (in food, drink, and ALXN1840), and molybdenum output (in feces and urine). Steady state is defined as molybdenum (out) equal to molybdenum (in).
Measured: Steady state: Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg)
Accumulation Of Molybdenum As Determined By Molybdenum Balance
Molybdenum balance was assessed through measurement of molybdenum intake (in food, drink, and ALXN1840), and molybdenum output (in feces and urine).
Measured: Accumulation: Day 1 through Day 8 (ALXN1840 15 mg) and Day 31 through Day 35 ((ALXN1840 30 mg)
Maximum Observed Plasma Concentration (Cmax) of Total Molybdenum and Plasma Ultrafiltrate (PUF) Molybdenum
Measured: Day 1 up to Day 39
Area Under The Concentration Time Curve (AUC0-inf) of Total Molybdenum and Plasma Ultrafiltrate (PUF) Molybdenum
Measured: Day 39
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Oct 2020
- Study start7 Sep 2020
- Primary completion7 Jun 2022
- Study completion7 Jun 2022
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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 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.Peter Ksenuk, MD, Study director, Alexion Pharmaceuticals, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04573309
- Sponsor's protocol ID
- ALXN1840-WD-204
- Other IDs
- 2020-001104-41
- Status last verified
- Jun 2024
- First submitted
- 28 Sep 2020
- Last updated
- 24 Jun 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Open-label Study to Assess Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840
- ClinicalTrials.gov ID
- NCT04573309
- Acronym / sponsor ID
- ALXN1840-WD-204
- Conditions
- Wilson Disease
- Keywords
- Copper Balance, Molybdenum Balance, Wilson Disease, ALXN1840
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 7 Sep 2020 (actual)
- Primary completion
- 7 Jun 2022 (actual)
- Study completion
- 7 Jun 2022 (actual)
- First posted
- 5 Oct 2020 (actual)
- Results
- Posted 27 Oct 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This exploratory study will investigate the effects of ALXN1840 on copper balance in participants with Wilson disease (WD).
Detailed description
Participants who are treatment experienced (which includes standard-of-care therapies or ALXN1840) and treatment naïve are eligible for this study.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ALXN1840 | Experimental | Drug: ALXN1840Participants will be administered ALXN1840 at a dose of 15 milligrams (mg)/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Diagnosis of WD by Leipzig Criteria ≥ 4.
- Able to reside in the clinical research unit for intensive metabolic monitoring of copper and molybdenum.
- Participants willing to adhere to copper/molybdenum-controlled diet during the study.
- Willing and able to follow protocol-specified contraception requirements.
- Capable of giving signed informed consent.
Key exclusion criteria (8 listed)
- Decompensated cirrhosis or model for end stage liver disease score > 13.
- Modified Nazer score > 7.
- Clinically significant gastrointestinal bleed within past 3 months.
- Alanine aminotransferase > 2 × upper limit of normal.
- Hemoglobin less than lower limit of the reference range for age and sex.
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 orally as tablets. | Drug | ALXN1840 |
Primary outcome
Mean Daily Copper Balance: Day 1 Through Day 8
Time frame: Accumulation: Day 1 through Day 8 (ALXN1840 15 mg)
Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period.
Mean Daily Copper Balance: Day 31 Through Day 35
Time frame: Accumulation: Day 31 through Day 35 (ALXN1840 30 mg)
Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period.
Mean Daily Copper Balance: Day 25 Through Day 28
Time frame: Accumulation: Day 25 through Day 28 (ALXN1840 15 mg)
Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period.
Mean Daily Copper Balance: Day 36 Through Day 39
Time frame: Accumulation: Day 36 through Day 39 (ALXN1840 30 mg)
Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period.
8 secondary outcomes
Change From Baseline In Mean Daily Copper Balance
Time frame: Accumulation: Baseline, Day 1 through Day 8 (ALXN1840 15 mg) and Day 31 through Day 35 (ALXN1840 30 mg); Steady State: Baseline, Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg)
Copper Quantified In Food, Drink, Feces, And Urine, Including Plasma Total And Labile Bound Copper (LBC)
Time frame: Accumulation: Day 1 through Day 8 for 15 mg and Day 31 through Day 35 for 30 mg; Steady state: Day 25 through Day 28 for ALXN1840 15 mg and Day 36 through Day 39 for ALXN1840 30 mg
Molybdenum Specified In ALXN1840 Doses Given And Quantified In Food, Drink, Feces, And Urine, Including Plasma At Steady State
Time frame: Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg)
Change From Baseline In Total Molybdenum Excretion In Urine And Feces
Time frame: Accumulation: Baseline, Day 1 through Day 8 (ALXN1840 15 mg) and Day 31 through Day 35 (ALXN1840 30 mg); Steady State: Baseline, Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg)
Mean Daily Molybdenum Balance At ALXN1840 Steady State
Time frame: Steady state: Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg)
Accumulation Of Molybdenum As Determined By Molybdenum Balance
Time frame: Accumulation: Day 1 through Day 8 (ALXN1840 15 mg) and Day 31 through Day 35 ((ALXN1840 30 mg)
Maximum Observed Plasma Concentration (Cmax) of Total Molybdenum and Plasma Ultrafiltrate (PUF) Molybdenum
Time frame: Day 1 up to Day 39
Area Under The Concentration Time Curve (AUC0-inf) of Total Molybdenum and Plasma Ultrafiltrate (PUF) Molybdenum
Time frame: Day 39
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jun 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
Peter Ksenuk, MD study director
Alexion Pharmaceuticals, Inc.
3 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ALXN1840 in people with Wilson Disease, run by Alexion Pharmaceuticals, Inc. at 3 sites in 3 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: finished, as of the sponsor's last update on 24 Jun 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States, New Zealand, United Kingdom. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 27 Oct 2023). 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?
Mean Daily Copper Balance: Day 1 Through Day 8 (time frame: Accumulation: Day 1 through Day 8 (ALXN1840 15 mg)). The study lists 4 primary outcomes in all. 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 24 Jun 2024.