Trial · NCT05047523
Study of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease
NCT05047523, Phase 3, Terminated, Alexion Pharmaceuticals, Inc.; results first posted 2024-10-15
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease. Trials, record NCT05047523. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05047523
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease
- NCT number
- NCT05047523
- Status
- Terminated
- Phases
- Phase 3
- Enrollment
- 40 participants, actual
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Conditions
- Wilson Disease
- Interventions
- ALXN1840, Standard of Care
- First posted
- 2021-09-17
- Last updated
- 2024-10-23
- Other registries' IDs
- eudract:2021-001015-82
- Results first posted
- 2024-10-15
- Limitations and caveats
- Due to early termination of the study, data for the efficacy endpoints were not collected.
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percent Change From Baseline to Week 48 in Non-ceruloplasmin-bound Copper in Plasma | 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. |
| Secondary | Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Primary Evaluation Period | Baseline up to Week 48 | 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. |
| Secondary | Area Under the Effect Versus Time Curve (AUEC) for Plasma Total Copper and Direct NCC | Week 48 | |
| Secondary | Maximum Observed Concentration (Cmax) of ALXN1840 for Plasma Total Molybdenum and Plasma Ultrafiltrate Molybdenum Concentrations | Week 48 | |
| Secondary | 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 | Week 48 |
Source ClinicalTrials.gov
Outcomes5
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percent Change From Baseline to Week 48 in Non-ceruloplasmin-bound Copper in Plasma | Baseline, Week 48 | FAS included all participants who received at least 1 dose of ALXN1840 or SoC treatment. Due to the early termination of the study, data for this Outcome Measure were not collected for any of the cohorts. | ||
| SECONDARY | Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Primary Evaluation Period | Baseline up to Week 48 | Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Area Under the Effect Versus Time Curve (AUEC) for Plasma Total Copper and Direct NCC | Week 48 | Pharmacodynamic (PD) analysis set included all participants who had sufficient plasma samples to enable the calculation of pharmacokinetic (PK) parameters and provide PK/PD profiles. Here, 'Overall number of participants analyzed' = participants evaluable for this Outcome Measure. No participants were evaluable for this Outcome Measure for Cohort 2: ALXN1840. | nanograms (ng)*hours (hr)/milliliter(mL) | MEAN |
| SECONDARY | Maximum Observed Concentration (Cmax) of ALXN1840 for Plasma Total Molybdenum and Plasma Ultrafiltrate Molybdenum Concentrations | Week 48 | PK analysis set included all participants who had sufficient plasma samples to enable the calculation of PK parameters and provide PK/PD profiles. Here, 'Overall number of participants analyzed' = participants evaluable for this Outcome Measure. No participants were evaluable for this Outcome Measure for Cohort 2: ALXN1840. | ng/mL | MEAN |
| SECONDARY | 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 | Week 48 | PK analysis set included all participants who had sufficient plasma samples to enable the calculation of PK parameters and provide PK/PD profiles. Here, 'Overall number of participants analyzed' = participants evaluable for this Outcome Measure. No participants were evaluable for this Outcome Measure for Cohort 2: ALXN1840. | ng*hr/mL | MEAN |
Source ClinicalTrials.gov
Results by group22
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Primary Evaluation Period | Cohort 1: ALXN1840 | 13 | Participants | ||
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Primary Evaluation Period | Cohort 1: SoC Therapy | 13 | Participants | ||
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Primary Evaluation Period | Cohort 2: ALXN1840 | 3 | Participants | ||
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Primary Evaluation Period | Cohort 2: SoC Therapy | 4 | Participants | ||
| Area Under the Effect Versus Time Curve (AUEC) for Plasma Total Copper and Direct NCC | Cohort 1: ALXN1840 | Total Copper | 6342.909 | Standard Deviation 3038.3317 | nanograms (ng)*hours (hr)/milliliter(mL) |
| Area Under the Effect Versus Time Curve (AUEC) for Plasma Total Copper and Direct NCC | Cohort 1: SoC Therapy | Total Copper | 7528.864 | Standard Deviation 4167.2110 | nanograms (ng)*hours (hr)/milliliter(mL) |
| Area Under the Effect Versus Time Curve (AUEC) for Plasma Total Copper and Direct NCC | Cohort 2: SoC Therapy | Total Copper | 16369.000 | Standard Deviation NA | nanograms (ng)*hours (hr)/milliliter(mL) |
| Area Under the Effect Versus Time Curve (AUEC) for Plasma Total Copper and Direct NCC | Cohort 1: ALXN1840 | Direct NCC | 3882.473 | Standard Deviation 1716.4565 | nanograms (ng)*hours (hr)/milliliter(mL) |
| Area Under the Effect Versus Time Curve (AUEC) for Plasma Total Copper and Direct NCC | Cohort 1: SoC Therapy | Direct NCC | 3067.864 | Standard Deviation 1524.9183 | nanograms (ng)*hours (hr)/milliliter(mL) |
| Area Under the Effect Versus Time Curve (AUEC) for Plasma Total Copper and Direct NCC | Cohort 2: SoC Therapy | Direct NCC | 4214.000 | Standard Deviation NA | nanograms (ng)*hours (hr)/milliliter(mL) |
1 to 10 of 22Source ClinicalTrials.gov
See all 22 results by groupParticipant flow48
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Primary Evaluation Period (48 Weeks) | STARTED | Cohort 1: ALXN1840 | 15 | |
| Primary Evaluation Period (48 Weeks) | STARTED | Cohort 1: SoC Therapy | 16 | |
| Primary Evaluation Period (48 Weeks) | STARTED | Cohort 2: ALXN1840 | 4 | |
| Primary Evaluation Period (48 Weeks) | STARTED | Cohort 2: SoC Therapy | 5 | |
| Primary Evaluation Period (48 Weeks) | STARTED | ALXN1840/ALXN1840 | 0 | |
| Primary Evaluation Period (48 Weeks) | STARTED | SoC Therapy/ALXN1840 | 0 | |
| Primary Evaluation Period (48 Weeks) | Received at Least 1 Dose of Study Drug | Cohort 1: ALXN1840 | 15 | |
| Primary Evaluation Period (48 Weeks) | Received at Least 1 Dose of Study Drug | Cohort 1: SoC Therapy | 16 | |
| Primary Evaluation Period (48 Weeks) | Received at Least 1 Dose of Study Drug | Cohort 2: ALXN1840 | 4 | |
| Primary Evaluation Period (48 Weeks) | Received at Least 1 Dose of Study Drug | Cohort 2: SoC Therapy | 5 |
1 to 10 of 48Source ClinicalTrials.gov
See all 48 participant flowAdverse events228
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Neutropenia | Blood and lymphatic system disorders | Cohort 1: ALXN1840 | 1 | 15 | 1 |
| other | Neutropenia | Blood and lymphatic system disorders | Cohort 1: SoC Therapy | 1 | 16 | 1 |
| other | Neutropenia | Blood and lymphatic system disorders | Cohort 2: ALXN1840 | 1 | 4 | 1 |
| other | Neutropenia | Blood and lymphatic system disorders | Cohort 2: SoC Therapy | 0 | 5 | 0 |
| other | Neutropenia | Blood and lymphatic system disorders | ALXN1840/ALXN1840 | 0 | 12 | 0 |
| other | Neutropenia | Blood and lymphatic system disorders | SoC Therapy/ALXN1840 | 0 | 12 | 0 |
| other | Abdominal pain | Gastrointestinal disorders | Cohort 1: ALXN1840 | 3 | 15 | 5 |
| other | Abdominal pain | Gastrointestinal disorders | Cohort 1: SoC Therapy | 0 | 16 | 0 |
| other | Abdominal pain | Gastrointestinal disorders | Cohort 2: ALXN1840 | 0 | 4 | 0 |
| other | Abdominal pain | Gastrointestinal disorders | Cohort 2: SoC Therapy | 0 | 5 | 0 |
1 to 10 of 228Source ClinicalTrials.gov
See all 228 adverse eventsAdverse event totals18
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cohort 1: ALXN1840 | 0 | 15 |
| Total, all-cause mortality | Cohort 1: SoC Therapy | 0 | 16 |
| Total, all-cause mortality | Cohort 2: ALXN1840 | 0 | 4 |
| Total, all-cause mortality | Cohort 2: SoC Therapy | 0 | 5 |
| Total, all-cause mortality | ALXN1840/ALXN1840 | 0 | 12 |
| Total, all-cause mortality | SoC Therapy/ALXN1840 | 0 | 12 |
| Total, other adverse events | Cohort 1: ALXN1840 | 13 | 15 |
| Total, other adverse events | Cohort 1: SoC Therapy | 12 | 16 |
| Total, other adverse events | Cohort 2: ALXN1840 | 2 | 4 |
| Total, other adverse events | Cohort 2: SoC Therapy | 4 | 5 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 adverse event totalsPapers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT05047523
GET https://conductscience.org/api/trials/v1/NCT05047523/results?page=2