Trial · NCT02273596
Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients
NCT02273596, Phase 2, Completed, Alexion Pharmaceuticals, Inc.; results first posted 2021-09-29
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients. Trials, record NCT02273596. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02273596
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients
- NCT number
- NCT02273596
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 29 participants, actual
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Conditions
- Wilson Disease
- Interventions
- ALXN1840
- First posted
- 2014-10-24
- Last updated
- 2021-09-29
- Results first posted
- 2021-09-29
- Limitations and caveats
- Due to low chromatographic resolution of data, quantitative analysis for speciation profiling, as had been planned in the protocol, was not feasible.
Source ClinicalTrials.gov
Registered outcomes23
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage Of Participants With Normalized Concentrations Of NCC | Week 24 | Normalized concentrations of NCC was defined as who achieving or maintaining normalized levels of NCC (0.8 to 2.3 micromole \[μmol\]l/liter \[L\]\]) adjusted for Mo plasma concentration or reaching a reduction of at least 25% in NCC corrected for Mo if above the normal reference range at the time of enrollment. NCC was calculated by subtracting the amount of copper (Cu) bound to ceruloplasmin (CP) from the total plasma Cu concentration. Post-baseline NCC values were adjusted (corrected) to account for Cu bound in tripartite complexes with ALXN1840 and albumin. Descriptive statistics are reported. |
| Secondary | Change From Baseline In NCC Concentrations Adjusted For Mo Plasma Concentration At Week 24 | Baseline, Week 24 | The change from Baseline in NCC adjusted for Mo plasma concentration over 24 weeks were analyzed and descriptive statistics are reported. Change from Baseline = (Week 24 NCC concentrations adjusted for Mo plasma concentration) - (Baseline NCC concentrations). Least square means and their 95% confidence intervals (CIs) were calculated using a restricted maximum likelihood (REML)-based mixed model for repeated measures (MMRM) with fixed effects for Baseline, cohort, visit, Baseline-by-visit, and cohort-by-visit interaction. |
| Secondary | Time To Normalization Of NCC Adjusted For Mo Plasma Concentration In Participants With Elevated Baseline NCC | Up to last assessment (up to Week 176) | For time to normalization of NCC adjusted for Mo plasma concentration, a Kaplan-Meier approach was used where participants not normalized were censored at the latest observed time point. To achieve a normalized NCC concentration, participants must have demonstrated 2 consecutive measures within the normal range (0.8 to 2.3 μmol/L). Analyses includes all data up to the last assessment in the extension period for participants who reached the event of normalization of NCC corrected concentrations. Last Assessment is a summary of the last available post-baseline result for each participant. Analyses includes all data up to the last assessment in the extension period; up to Week 176. |
| Secondary | Change From Baseline In Neurological Status Using The Unified Wilson's Disease Rating Scale (UWDRS) (Neurological Subscore; Part I) At Week 24 | Baseline, Week 24 | The UWDRS is a clinical rating scale designed to evaluate the neurological manifestations of WD. Change from Baseline in the UWDRS I for consciousness is presented. * UWDRS I: range 0 to 3, maximum score of 3 * UWDRS I was assessed by a Neurologist * Change from Baseline was calculated as: Week 24 score - Baseline score. A decrease in score from Baseline is indicative of both an improvement in condition and a better outcome. |
| Secondary | Change From Baseline In Neurological Status Using The UWDRS (Neurological Subscore; Parts II, III, And Total Score) At Week 24 | Baseline, Week 24 | The UWDRS is a clinical rating scale designed to evaluate the neurological manifestations of WD. UWDRS comprises 3 parts: UWDRS I (consciousness), UWDRS II (disability), and UWDRS III (neurological status). Change from Baseline in the UWDRS II, III, and total score are presented. * UWDRS I: range 0 to 3, maximum score of 3 * UWDRS II: range 0 to 40, maximum score of 40 * UWDRS III: range 0 to 175, maximum score of 175 * UWDRS total score: sum of the UWDRS I, II, and III: range 0 to 218, maximum score of 218 * UWDRS I and III were assessed by a neurologist, while UWDRS II was reported by the participant or caregiver. * Change from Baseline was calculated as: Week 24 score - Baseline score. Least square means and their 95% CIs were calculated using a REML-based MMRM with fixed effects for Baseline, cohort, visit, Baseline-by-visit, and cohort-by-visit interaction. A decrease in score from Baseline is indicative of an improvement in condition and a better outcome. |
| Secondary | Change From Baseline In Psychiatric Status Dimension Using Mini International Neuropsychiatric Interview (M.I.N.I.) Tracking Standardized Scores At Week 24 | Baseline, Week 24 | The M.I.N.I. is a short structured diagnostic interview for the Diagnostic and Statistical Manual of Mental Disorders IV and the 10th revision of the International Statistical Classification of Diseases and Related Health Problems. It was used to "track" the severity of symptoms by using a combination of questions in 16 Psychiatric Status dimensions that are summarized using a single standardized score for each dimension, ranging from 0 (interpreted as "did not occur at all") to 4 (interpreted as "occurred extremely often"). The standardized score is an average of these responses. Change from Baseline = Week 24 standardized score - Baseline standardized score. Least square means and 95% CIs were calculated using an REML-based MMRM with fixed effects for Baseline, cohort, visit, Baseline-by-visit, and cohort-by-visit interaction. Decrease from Baseline (a lower score) is indicative of a decrease in symptoms and a better outcome. |
| Secondary | Clinical Global Impression-Improvement Scale (CGI-I) At Week 24 | Week 24 | 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. Change from Baseline = Week 24 score - Baseline score. An increase in CGI-I score indicates improvement. |
| Secondary | Change From Baseline In Clinical Global Impression Severity Scale (CGI-S) At Week 24 | Baseline, Week 24 | 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. Change from Baseline = Week 24 score - Baseline score. Decrease in CGI-S score and increase indicates improvement. |
| Secondary | Change From Baseline In Quality Of Life (QoL)/Patient Reported Outcome (PRO) Assessed By The European Quality Of Life 5 Dimensions (EQ-5D) Visual Analogue Scale (VAS) At Week 24 | Baseline, Week 24 | The EQ-5D VAS records the participant's self-rated health as indicated on a scale from 0 to 100 with 0 being "the worst health you can imagine" and 100 being "the best health you can imagine, with higher scores for a higher quality of life. Change from Baseline = Week 24 score - Baseline score. An increase in score indicates improvement. The least-square means and their 95% CIs were calculated using a REML-based MMRM with fixed effects for Baseline, cohort, visit, Baseline-by-visit, and cohort-by-visit interaction. |
| Secondary | Change From Baseline In Quality Of Life (QoL)/Patient Reported Outcome (PRO) Assessed By The EQ-5D Descriptive System UK Health Index Scores At Week 24 | Baseline, Week 24 | The EQ-5D-5L Descriptive System provides a simple descriptive profile and a single index value for health status (United Kingdom \[UK\] Health Index Score). The EQ-5D-5L consists of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression), each of which can have 1 of 5 responses that represent 5 levels of severity (no problems, slight problems, moderate problems, severe problems, extreme problems). The participant was asked to indicate his/her health state for each of the 5 dimensions. The 5-item index score was transformed into a utility score between 0 (worst health state) and 1 (best health state). Change from Baseline = Week 24 index score - Baseline index score. An increase in score indicates improvement. The least square means and their 95% CIs were calculated using a REML-based MMRM with fixed effects for Baseline, cohort, visit, Baseline-by-visit, and cohort-by-visit interaction. |
1 to 10 of 23Source ClinicalTrials.gov
See all 23 registered outcomesOutcomes23
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage Of Participants With Normalized Concentrations Of NCC | Week 24 | Participants who were enrolled and received at least 1 dose of study drug. | percentage of participants | NUMBER |
| SECONDARY | Change From Baseline In NCC Concentrations Adjusted For Mo Plasma Concentration At Week 24 | Baseline, Week 24 | Participants who were enrolled and received at least 1 dose of study drug and had analyzable data at the specified timepoint. | μmol/L | LEAST_SQUARES_MEAN |
| SECONDARY | Time To Normalization Of NCC Adjusted For Mo Plasma Concentration In Participants With Elevated Baseline NCC | Up to last assessment (up to Week 176) | This analysis was only performed on the subset of participants with elevated Baseline NCC who reached the event of normalization of NCC corrected concentrations. | days | MEDIAN |
| SECONDARY | Change From Baseline In Neurological Status Using The Unified Wilson's Disease Rating Scale (UWDRS) (Neurological Subscore; Part I) At Week 24 | Baseline, Week 24 | Participants who were enrolled and received at least 1 dose of study drug and had analyzable data at the specified timepoint. | score on a scale | MEAN |
| SECONDARY | Change From Baseline In Neurological Status Using The UWDRS (Neurological Subscore; Parts II, III, And Total Score) At Week 24 | Baseline, Week 24 | Participants who were enrolled and received at least 1 dose of study drug and had analyzable data at the specified timepoint. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline In Psychiatric Status Dimension Using Mini International Neuropsychiatric Interview (M.I.N.I.) Tracking Standardized Scores At Week 24 | Baseline, Week 24 | Participants who were enrolled and received at least 1 dose of study drug and had analyzable data at the specified timepoint. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Clinical Global Impression-Improvement Scale (CGI-I) At Week 24 | Week 24 | Participants who were enrolled and received at least 1 dose of study drug and had analyzable data at the specified timepoint. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline In Clinical Global Impression Severity Scale (CGI-S) At Week 24 | Baseline, Week 24 | Participants who were enrolled and received at least 1 dose of study drug and had analyzable data at the specified timepoint. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline In Quality Of Life (QoL)/Patient Reported Outcome (PRO) Assessed By The European Quality Of Life 5 Dimensions (EQ-5D) Visual Analogue Scale (VAS) At Week 24 | Baseline, Week 24 | Participants who were enrolled and received at least 1 dose of study drug and had analyzable data at the specified timepoint. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline In Quality Of Life (QoL)/Patient Reported Outcome (PRO) Assessed By The EQ-5D Descriptive System UK Health Index Scores At Week 24 | Baseline, Week 24 | Participants who were enrolled and received at least 1 dose of study drug and had analyzable data at the specified timepoint. | EQ-5D-5L index score | LEAST_SQUARES_MEAN |
1 to 10 of 23Source ClinicalTrials.gov
See all 23 outcomesResults by group60
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage Of Participants With Normalized Concentrations Of NCC | ALXN1840 | 85.7 | 95% Confidence Interval 70.8 to 97.6 | percentage of participants | |
| Change From Baseline In NCC Concentrations Adjusted For Mo Plasma Concentration At Week 24 | ALXN1840 | -2.56 | 95% Confidence Interval -3.13 to -1.98 | μmol/L | |
| Time To Normalization Of NCC Adjusted For Mo Plasma Concentration In Participants With Elevated Baseline NCC | ALXN1840 | 147.5 | Inter-Quartile Range 37.0 to 211.0 | days | |
| Change From Baseline In Neurological Status Using The Unified Wilson's Disease Rating Scale (UWDRS) (Neurological Subscore; Part I) At Week 24 | ALXN1840 | 0.0 | Standard Deviation 0.0 | score on a scale | |
| Change From Baseline In Neurological Status Using The UWDRS (Neurological Subscore; Parts II, III, And Total Score) At Week 24 | ALXN1840 | UWDRS Part II | -2.5 | 95% Confidence Interval -4.6 to -0.4 | score on a scale |
| Change From Baseline In Neurological Status Using The UWDRS (Neurological Subscore; Parts II, III, And Total Score) At Week 24 | ALXN1840 | UWDRS Part III | -5.75 | 95% Confidence Interval -10.74 to -0.77 | score on a scale |
| Change From Baseline In Neurological Status Using The UWDRS (Neurological Subscore; Parts II, III, And Total Score) At Week 24 | ALXN1840 | Total Score | -8.23 | 95% Confidence Interval -15.42 to -1.04 | score on a scale |
| Change From Baseline In Psychiatric Status Dimension Using Mini International Neuropsychiatric Interview (M.I.N.I.) Tracking Standardized Scores At Week 24 | ALXN1840 | Major Depressive Episode Standardized | -0.39 | 95% Confidence Interval -0.62 to -0.16 | score on a scale |
| Change From Baseline In Psychiatric Status Dimension Using Mini International Neuropsychiatric Interview (M.I.N.I.) Tracking Standardized Scores At Week 24 | ALXN1840 | Suicidality Tracking Scale Standardized | -0.07 | 95% Confidence Interval -0.09 to -0.04 | score on a scale |
| Change From Baseline In Psychiatric Status Dimension Using Mini International Neuropsychiatric Interview (M.I.N.I.) Tracking Standardized Scores At Week 24 | ALXN1840 | Manic/Hypomanic Episode Standardized | -0.09 | 95% Confidence Interval -0.23 to 0.05 | score on a scale |
1 to 10 of 60Source ClinicalTrials.gov
See all 60 results by groupParticipant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| 24-Week Treatment Period | STARTED | ALXN1840 | 29 | |
| 24-Week Treatment Period | Received at Least 1 Dose of Study Drug | ALXN1840 | 28 | |
| 24-Week Treatment Period | COMPLETED | ALXN1840 | 22 | |
| 24-Week Treatment Period | NOT COMPLETED | ALXN1840 | 7 | |
| Extension Period | STARTED | ALXN1840 | 22 | |
| Extension Period | Received at Least 1 Dose of Study Drug | ALXN1840 | 22 | |
| Extension Period | COMPLETED | ALXN1840 | 0 | |
| Extension Period | NOT COMPLETED | ALXN1840 | 22 |
Source ClinicalTrials.gov
Adverse events57
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 | Leukopenia | Blood and lymphatic system disorders | ALXN1840 | 3 | 28 | |
| other | Constipation | Gastrointestinal disorders | ALXN1840 | 4 | 28 | |
| other | Diarrhoea | Gastrointestinal disorders | ALXN1840 | 3 | 28 | |
| other | Nausea | Gastrointestinal disorders | ALXN1840 | 2 | 28 | |
| other | Vomiting | Gastrointestinal disorders | ALXN1840 | 2 | 28 | |
| other | Fatigue | General disorders | ALXN1840 | 6 | 28 | |
| other | Urinary tract infection | Infections and infestations | ALXN1840 | 8 | 28 | |
| other | Sinusitis | Infections and infestations | ALXN1840 | 3 | 28 | |
| other | Influenza | Infections and infestations | ALXN1840 | 2 | 28 | |
| other | Tonsillitis | Infections and infestations | ALXN1840 | 2 | 28 |
1 to 10 of 57Source ClinicalTrials.gov
See all 57 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | ALXN1840 | 0 | 28 |
| Total, other adverse events | ALXN1840 | 26 | 28 |
| Total, serious adverse events | ALXN1840 | 11 | 28 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 28988934 | RESULT |
Source PubMed
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.
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GET https://conductscience.org/api/trials/v1/NCT02273596
GET https://conductscience.org/api/trials/v1/NCT02273596/registered?page=2