Trial · NCT03403205
Efficacy and Safety of ALXN1840 Administered for 48 Weeks Versus Standard of Care in Participants With Wilson Disease
NCT03403205, Phase 3, Terminated, Alexion Pharmaceuticals, Inc.; results first posted 2023-10-10
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Efficacy and Safety of ALXN1840 Administered for 48 Weeks Versus Standard of Care in Participants With Wilson Disease. Trials, record NCT03403205. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03403205
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Efficacy and Safety of ALXN1840 Administered for 48 Weeks Versus Standard of Care in Participants With Wilson Disease
- NCT number
- NCT03403205
- Status
- Terminated
- Phases
- Phase 3
- Enrollment
- 214 participants, actual
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Conditions
- Wilson Disease
- Interventions
- ALXN1840, SoC Therapy
- First posted
- 2018-01-18
- Last updated
- 2024-06-17
- Other registries' IDs
- eudract:2017-004135-36
- Results first posted
- 2023-10-10
Source ClinicalTrials.gov
Registered outcomes12
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | 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) | Baseline to Week 48 | dNCC is the directly quantified copper not bound to ceruloplasmin, obtained by inductively coupled plasma mass spectrometry after immunocapture and removal of ceruloplasmin. Baseline was defined as last non-missing value on or before first study drug administration. Least square (LS) mean and standard error (SE) was calculated using analysis of covariance (ANCOVA). |
| Secondary | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Baseline up to Week 48 | An 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 those AEs with onset after the first dose of randomized treatment or existing events that worsened in severity after the first dose of randomized treatment. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. |
| Secondary | Change From Baseline in the Unified Wilson Disease Rating Scale (UWDRS) Part II Total Score at Week 48 | Baseline, Week 48 | The UWDRS comprises 3 parts: UWDRS Part I (level of consciousness, item 1), UWDRS Part II (a patient-reported review of daily activity items \[disability\], items 2 to 11 \[10 items in total\]), and UWDRS Part III (a detailed neurological examination, items 12 to 34 \[23 items in total\]). The UWDRS Part II total score was calculated as the sum of Question 2 to Question 11 (each question has range 0 \[none\] to 4 \[severe\]). The UWDRS Part II total score ranges from 0 (no disability) to 40 (severe disability), with lower score indicating improvement in condition and a better outcome. Change from baseline was calculated as: postbaseline assessment value - baseline assessment value when both values were not missing. |
| Secondary | Change From Baseline in UWDRS Part III Total Score at Week 48 | Baseline, Week 48 | The UWDRS comprises 3 parts: UWDRS Part I (level of consciousness, item 1), UWDRS Part II (a patient-reported review of daily activity items \[disability\], items 2 to 11 \[10 items in total\]), and UWDRS Part III (a detailed neurological examination, items 12 to 34 \[23 items in total\]). The UWDRS Part I and III was assessed by a neurologist who was blinded to the treatment randomization. The UWDRS Part III total score was calculated as the sum of Question 12 to Question 34. The UWDRS Part III total score ranges from 0 (normal) to 175 (severe disease), with lower score indicating improvement in condition and a better outcome. Change from baseline was calculated as: postbaseline assessment value - baseline assessment value when both values were not missing. |
| Secondary | Change From Baseline in UWDRS Part III Functional Subscale Score at Week 48 | Baseline, Week 48 | UWDRS Part III Functional Subscale consists of speech, handwriting, arising from chair, and gait from UWDRS Part III. The standardized score of the first 3 items ranges from 0 (normal) to 10 (worst), and standardized transformed score of gait ranges from 0 (normal) to 10 (worst). The average of these scores was used to create the Part III Functional Subscale with a range of 0 (normal) - 10 (worst) with higher scores indicating more functional disability. |
| Secondary | Change From Baseline in UWDRS Part III Individual Items/Subscales (Speech, Handwriting, Arising From a Chair, and Gait) Score at Week 48 | Baseline, Week 48 | UWDRS Part III individual items speech, handwriting, arising from chair, and gait are reported here. For speech (Question 12), original score ranges from 0 (normal) to 4 (unintelligible). For handwriting (Question 20), original score ranges from 0 (normal) to 4 (cannot hold a pen). For arising from chair (Question 27), original score ranges from 0 (normal) to 4 (unable to arise without help). For gait (Question 29), the original score (range: 0 \[normal\] to 10 \[severe condition\]) was calculated by summing subscores (0 \[normal\] to 4 \[severe\]) of Part A (Right and Left Leg dystonia), B (Ataxia), and C (Parkinsonism). |
| Secondary | Clinical Global Impression-Improvement Scale (CGI-I) Score at Week 48 | Week 48 | The CGI-I is a 7-point scale where the clinician assessed how much participant's illness improved or worsened relative to a Baseline state at the beginning of the intervention and rated as: 1, very much improved; 2, much improved; 3, minimally improved; 4, no change; 5, minimally worse; 6, much worse; or 7, very much worse. |
| Secondary | Change From Baseline in Clinical Global Impression Severity Scale (CGI-S) Score at Week 48 | Baseline, Week 48 | The CGI-S is a 7-point scale where the investigator rated severity of participant's illness at the time of assessment, relative to the investigator's past experience with participants who have the same diagnosis. Considering total clinical experience, a participant was assessed on severity of illness at time of rating as: 1, normal, not at all ill; 2, borderline ill; 3, mildly ill; 4, moderately ill; 5, markedly ill; 6, severely ill; or 7, extremely ill. |
| Secondary | Change From Baseline in Model for End-Stage Liver Disease (MELD) Score at Week 48 | Baseline, Week 48 | The MELD score uses the participant's values for bilirubin, creatinine, and the international normalized ratio (INR). The initial MELD score (MELD\[i\]) is calculated according to the following formula: MELD(i) = 3.78\*ln\[serum bilirubin (mg/dL)\] + 11.2\*ln\[INR\] + 9.57\*ln\[serum creatinine (mg/dL)\] + 6.43. Creatinine, bilirubin, and INR values less than 1.0 are set to 1.0 and creatinine values greater than 4.0 are set to 4.0 when calculating MELD(i). Additionally, creatinine, bilirubin, and INR are rounded to the 10th decimal place prior to performing the calculation. The initial MELD score is then rounded to the nearest integer. The MELD score ranges from 6 (least sick) - 40 (most sick), with higher values indicating more advanced disease. |
| Secondary | Absolute Change From Baseline in Calculated Non-Ceruloplasmin Bound Copper (cNCC) or Calculated Non-Ceruloplasmin Bound Copper Corrected (cNCCcorrected) in Plasma at Week 48 | Baseline, Week 48 | cNCC = Plasma Total Copper (Cu) \[micrograms (µg)/L\]-(3.15\*ceruloplasmin \[milligrams (mg)/L\])/63.5 \[µg/µmol\] For ALXN1840-treated participants, cNCC in plasma corrected for amount of Cu bound to ALXN1840 tripartite complex (TPC) cNCCcorrected = (√cNCC- 0.993)2√Mo, (Mo= molybdenum). In calculation of cNCC and cNCCcorrected following rules apply: - For plasma total Cu concentration \<lower limit of quantification (LLOQ), cNCC was considered missing (LLOQ = 20 nanograms \[ng\]/mL); - Serum ceruloplasmin concentration values \<LLOQ are set to 0 (LLOQ = 22.5 mg/L); - Plasma total Mo concentration values \<LLOQ are set to 0 (LLOQ = 1 ng/L); - If cNCC calculation produces a negative result, cNCC was considered missing and cNCCcorrected was not derived; - cNCCcorrected was set to 0 when 0.993√Mo \> √cNCC. |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 registered outcomesOutcomes12
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | 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) | Baseline to Week 48 | Full analysis set included all randomized participants who received at least 1 dose of randomized treatment. | micromoles (µmol)*hours (hr)/liter (L) | LEAST_SQUARES_MEAN |
| SECONDARY | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Baseline up to Week 48 | Safety analysis set included all participants who received at least 1 dose of randomized treatment. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in the Unified Wilson Disease Rating Scale (UWDRS) Part II Total Score at Week 48 | Baseline, Week 48 | Full analysis set included all randomized participants who received at least 1 dose of randomized treatment. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in UWDRS Part III Total Score at Week 48 | Baseline, Week 48 | Full analysis set included all randomized participants who received at least 1 dose of randomized treatment. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in UWDRS Part III Functional Subscale Score at Week 48 | Baseline, Week 48 | Full analysis set included all randomized participants who received at least 1 dose of randomized treatment. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in UWDRS Part III Individual Items/Subscales (Speech, Handwriting, Arising From a Chair, and Gait) Score at Week 48 | Baseline, Week 48 | Full analysis set included all randomized participants who received at least 1 dose of randomized treatment. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. 'Number analyzed' = participants evaluable for specified category. | units on a scale | MEAN |
| SECONDARY | Clinical Global Impression-Improvement Scale (CGI-I) Score at Week 48 | Week 48 | Full analysis set included all randomized participants who received at least 1 dose of randomized treatment. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in Clinical Global Impression Severity Scale (CGI-S) Score at Week 48 | Baseline, Week 48 | Full analysis set included all randomized participants who received at least 1 dose of randomized treatment. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in Model for End-Stage Liver Disease (MELD) Score at Week 48 | Baseline, Week 48 | Full analysis set included all randomized participants who received at least 1 dose of randomized treatment. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. | units on a scale | MEAN |
| SECONDARY | Absolute Change From Baseline in Calculated Non-Ceruloplasmin Bound Copper (cNCC) or Calculated Non-Ceruloplasmin Bound Copper Corrected (cNCCcorrected) in Plasma at Week 48 | Baseline, Week 48 | Full analysis set: all randomized participants who received at least 1 dose of study drug. 'Overall number of participants analyzed'= participants evaluable for this outcome measure. | µmol/L | MEAN |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 outcomesResults by group60
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| 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) | Cohort 1: ALXN1840 | 2.50 | Standard Error 0.150 | micromoles (µmol)*hours (hr)/liter (L) | |
| 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) | Cohort 1: SoC Therapy | 0.87 | Standard Error 0.204 | micromoles (µmol)*hours (hr)/liter (L) | |
| 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) | Cohort 2: ALXN1840 | 4.76 | Standard Error 0.319 | micromoles (µmol)*hours (hr)/liter (L) | |
| 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) | Cohort 2: SoC Therapy | 0.96 | Standard Error 0.487 | micromoles (µmol)*hours (hr)/liter (L) | |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Cohort 1: ALXN1840 | 89 | Participants | ||
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Cohort 1: SoC Therapy | 41 | Participants | ||
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Cohort 2: ALXN1840 | 30 | Participants | ||
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Cohort 2: SoC Therapy | 12 | Participants | ||
| Change From Baseline in the Unified Wilson Disease Rating Scale (UWDRS) Part II Total Score at Week 48 | Cohort 1: ALXN1840 | -0.7 | Standard Deviation 2.75 | units on a scale | |
| Change From Baseline in the Unified Wilson Disease Rating Scale (UWDRS) Part II Total Score at Week 48 | Cohort 1: SoC Therapy | 0.0 | Standard Deviation 2.31 | units on a scale |
1 to 10 of 60Source ClinicalTrials.gov
See all 60 results by groupParticipant flow54
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 | 105 | |
| Primary Evaluation Period (48 Weeks) | STARTED | Cohort 1: SoC Therapy | 56 | |
| Primary Evaluation Period (48 Weeks) | STARTED | Cohort 2: ALXN1840 | 37 | |
| Primary Evaluation Period (48 Weeks) | STARTED | Cohort 2: SoC Therapy | 16 | |
| Primary Evaluation Period (48 Weeks) | STARTED | Cohort 1: WTX101-201- ALXN1840 | 0 | |
| Primary Evaluation Period (48 Weeks) | STARTED | Cohort 2: WTX101-201- ALXN1840 | 0 | |
| Primary Evaluation Period (48 Weeks) | Received at Least 1 Dose of Study Drug | Cohort 1: ALXN1840 | 104 | |
| Primary Evaluation Period (48 Weeks) | Received at Least 1 Dose of Study Drug | Cohort 1: SoC Therapy | 56 | |
| Primary Evaluation Period (48 Weeks) | Received at Least 1 Dose of Study Drug | Cohort 2: ALXN1840 | 33 | |
| Primary Evaluation Period (48 Weeks) | Received at Least 1 Dose of Study Drug | Cohort 2: SoC Therapy | 14 |
1 to 10 of 54Source ClinicalTrials.gov
See all 54 participant flowAdverse events1,030
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 | Nausea | Gastrointestinal disorders | PEP Cohort 1: ALXN1840 | 6 | 104 | 6 |
| other | Nausea | Gastrointestinal disorders | PEP Cohort 1: SoC Therapy | 1 | 56 | 1 |
| other | Nausea | Gastrointestinal disorders | PEP Cohort 2: ALXN1840 | 2 | 33 | 2 |
| other | Nausea | Gastrointestinal disorders | PEP Cohort 2: SoC Therapy | 2 | 14 | 2 |
| other | Nausea | Gastrointestinal disorders | OLE Cohort 1: ALXN1840 | 6 | 89 | 10 |
| other | Nausea | Gastrointestinal disorders | OLE Cohort 1: SoC Therapy | 3 | 49 | 5 |
| other | Nausea | Gastrointestinal disorders | OLE Cohort 2: ALXN1840 | 1 | 28 | 1 |
| other | Nausea | Gastrointestinal disorders | OLE Cohort 2: SoC Therapy | 1 | 12 | 1 |
| other | Nausea | Gastrointestinal disorders | Cohort 1: WTX101-201- ALXN1840 | 1 | 8 | 1 |
| other | Nausea | Gastrointestinal disorders | Cohort 2: WTX101-201- ALXN1840 | 0 | 11 | 0 |
1 to 10 of 1,030Source ClinicalTrials.gov
See all 1,030 adverse eventsAdverse event totals30
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | PEP Cohort 1: ALXN1840 | 1 | 104 |
| Total, all-cause mortality | PEP Cohort 1: SoC Therapy | 0 | 56 |
| Total, all-cause mortality | PEP Cohort 2: ALXN1840 | 1 | 33 |
| Total, all-cause mortality | PEP Cohort 2: SoC Therapy | 0 | 14 |
| Total, all-cause mortality | OLE Cohort 1: ALXN1840 | 1 | 89 |
| Total, all-cause mortality | OLE Cohort 1: SoC Therapy | 0 | 49 |
| Total, all-cause mortality | OLE Cohort 2: ALXN1840 | 0 | 28 |
| Total, all-cause mortality | OLE Cohort 2: SoC Therapy | 0 | 12 |
| Total, all-cause mortality | Cohort 1: WTX101-201- ALXN1840 | 0 | 8 |
| Total, all-cause mortality | Cohort 2: WTX101-201- ALXN1840 | 0 | 11 |
1 to 10 of 30Source ClinicalTrials.gov
See all 30 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.
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