Trial · NCT04422431
Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840
NCT04422431, Phase 2, Completed, Alexion Pharmaceuticals, Inc.; results first posted 2023-07-03
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840. Trials, record NCT04422431. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04422431
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840
- NCT number
- NCT04422431
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 31 participants, actual
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Conditions
- Wilson Disease
- Interventions
- Bis-Choline Tetrathiomolybdate
- First posted
- 2020-06-09
- Last updated
- 2024-10-18
- Other registries' IDs
- eudract:2019-003711-60
- Results first posted
- 2023-07-03
Source ClinicalTrials.gov
Registered outcomes16
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in Liver Cu Concentration at Week 48 (Treatment Period) | Baseline, Week 48 (Treatment Period) | Liver biopsy samples were taken for the assessment of liver Cu concentration. Multiple imputation was used to impute missing data at Week 48 due to any reason based on Baseline values. |
| Secondary | Number of Participants With Change From Baseline in Nonalcoholic Steatohepatitis Clinical Research Network (NASH CRN) Fibrosis Stage at Week 48 (Treatment Period) | Baseline, Week 48 (Treatment Period) | Fibrosis from histology was evaluated by NASH CRN Fibrosis Stage, which was scaled from 0 to 4 stages where Score 0: None; Score 1: Perisinusoidal or periportal - 1a - mild, zone 3, perisinusoidal; Score 1: Perisinusoidal or periportal - 1b - moderate, zone 3, perisinusoidal; Score 1: Perisinusoidal or periportal - 1c - portal/periportal; Score 2: Both perisinusoidal and portal/periportal; Score 3: Bridging fibrosis; and Score 4: Cirrhosis. Higher scores indicated greater fibrosis. |
| Secondary | Number of Participants With Change From Baseline in Metavir Fibrosis Score at Week 48 (Treatment Period) | Baseline, Week 48 (Treatment Period) | Fibrosis from histology was evaluated by Metavir Fibrosis Score, which was ranged from 0 to 4 where Score 0: No fibrosis; Score 1: Stellate enlargement of portal tract but without septa formation; Score 2: Enlargement of portal tract with rare septa formation; Score 3: Numerous septa without cirrhosis; and Score 4: Cirrhosis. Higher scores indicated greater fibrosis. |
| Secondary | Number of Participants With Change From Baseline in Ishak Fibrosis Score at Week 48 (Treatment Period) | Baseline, Week 48 (Treatment Period) | Fibrosis from histology was evaluated by Ishak Fibrosis Score, which was ranged from 0 to 6 where Score 0: No fibrosis; Score 1: Fibrous expansion of some portal areas, with or without short fibrous septa; Score 2: Fibrous expansion of most portal areas, with or without short fibrous septa; Score 3: Fibrous expansion of most portal areas with occasional portal to portal (P-P) bridging; and Score 4: Fibrous expansion of portal areas with marked bridging (P-P) as well as portal central (P-C); Score 5: Marked bridging (P-P and/or P-C) with occasional nodules (incomplete cirrhosis); and Score 6: Cirrhosis, probable or definite. Higher scores indicated greater fibrosis. |
| Secondary | Change From Baseline in Hepatic Collagen Content at Week 48 (Treatment Period) | Baseline, Week 48 (Treatment Period) | Fibrosis from histology was evaluated by morphometric quantification of hepatic collagen content. |
| Secondary | Change From Baseline in a-SMA Content at Week 48 (Treatment Period) | Baseline, Week 48 (Treatment Period) | Fibrosis from histology was evaluated by morphometric quantification of hepatic a-SMA content. |
| Secondary | Number of Participants With Change From Baseline in NAS Steatosis Grading Score at Week 48 (Treatment Period) | Baseline, Week 48 (Treatment Period) | Steatosis from histology was evaluated by the steatosis component of the NAS, which was ranged from 0 to 3 where Score 0: \< 5% (minimal); Score 1: 5 - 33% (mild); Score 2: 34 - 66% (moderate); and Score 3: \> 66% (severe). Higher scores indicated greater steatosis. |
| Secondary | Change From Baseline in Hepatic Fat Content at Week 48 (Treatment Period) | Baseline, Week 48 (Treatment Period) | Steatosis from histology was evaluated by morphometric quantification of hepatic fat content. |
| Secondary | Change From Baseline in NAS Total Score at Week 48 (Treatment Period) | Baseline, Week 48 (Treatment Period) | Inflammation was quantified by the NAS total score. The score is defined as the unweighted sum of the scores for steatosis (0 \[minimal\] to 3 \[severe\]), lobular inflammation (0 \[none\] to 3 \[\>4 foci / 200x field\]), and hepatocellular ballooning (0 \[none\] to 2 \[many\]), thus ranging from 0 (no inflammation) to 8 (severe inflammation), with higher scores indicating more severe disease. |
| Secondary | Treatment Period: Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Day 1 (Treatment Period) up to Week 48 (Treatment Period) | An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), important medical event or reaction. The TEAEs were AEs with onset on or after the first study drug dose. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 registered outcomesOutcomes16
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in Liver Cu Concentration at Week 48 (Treatment Period) | Baseline, Week 48 (Treatment Period) | The Full Analysis Set in the Treatment Period included all participants who received at least 1 dose of study drug in the Treatment Period and had at least a Baseline liver Cu value evaluable. | μg/g | MEAN |
| SECONDARY | Number of Participants With Change From Baseline in Nonalcoholic Steatohepatitis Clinical Research Network (NASH CRN) Fibrosis Stage at Week 48 (Treatment Period) | Baseline, Week 48 (Treatment Period) | The Full Analysis Set in the Treatment Period included all participants who received at least 1 dose of study drug in the Treatment Period and had at least a Baseline liver Cu value evaluable. 'Number analyzed' signifies participants evaluable at specified timepoint. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Change From Baseline in Metavir Fibrosis Score at Week 48 (Treatment Period) | Baseline, Week 48 (Treatment Period) | The Full Analysis Set in the Treatment Period included all participants who received at least 1 dose of study drug in the Treatment Period and had at least a Baseline liver Cu value evaluable. 'Number analyzed' signifies participants evaluable at specified timepoint. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Change From Baseline in Ishak Fibrosis Score at Week 48 (Treatment Period) | Baseline, Week 48 (Treatment Period) | The Full Analysis Set in the Treatment Period included all participants who received at least 1 dose of study drug in the Treatment Period and had at least a Baseline liver Cu value evaluable. 'Number analyzed' signifies participants evaluable at specified timepoint. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in Hepatic Collagen Content at Week 48 (Treatment Period) | Baseline, Week 48 (Treatment Period) | The Full Analysis Set in the Treatment Period included all participants who received at least 1 dose of study drug in the Treatment Period and had at least a Baseline liver Cu value evaluable. 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. | percentage of collagen | MEAN |
| SECONDARY | Change From Baseline in a-SMA Content at Week 48 (Treatment Period) | Baseline, Week 48 (Treatment Period) | The Full Analysis Set in the Treatment Period included all participants who received at least 1 dose of study drug in the Treatment Period and had at least a Baseline liver Cu value evaluable. 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. | percentage of a-SMA | MEAN |
| SECONDARY | Number of Participants With Change From Baseline in NAS Steatosis Grading Score at Week 48 (Treatment Period) | Baseline, Week 48 (Treatment Period) | The Full Analysis Set in the Treatment Period included all participants who received at least 1 dose of study drug in the Treatment Period and had at least a Baseline liver Cu value evaluable. 'Number analyzed' signifies participants evaluable at specified timepoint. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in Hepatic Fat Content at Week 48 (Treatment Period) | Baseline, Week 48 (Treatment Period) | The Full Analysis Set in the Treatment Period included all participants who received at least 1 dose of study drug in the Treatment Period and had at least a Baseline liver Cu value evaluable. 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. | percentage of fat | MEAN |
| SECONDARY | Change From Baseline in NAS Total Score at Week 48 (Treatment Period) | Baseline, Week 48 (Treatment Period) | The Full Analysis Set in the Treatment Period included all participants who received at least 1 dose of study drug in the Treatment Period and had at least a Baseline liver Cu value evaluable. 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. | units on a scale | MEAN |
| SECONDARY | Treatment Period: Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Day 1 (Treatment Period) up to Week 48 (Treatment Period) | Safety Analysis Set in the Treatment Period included all participants who received at least 1 dose of treatment in the Treatment Period. | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 outcomesResults by group66
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in Liver Cu Concentration at Week 48 (Treatment Period) | ALXN1840 | 92.8 | Standard Error 56.34 | μg/g | |
| Number of Participants With Change From Baseline in Nonalcoholic Steatohepatitis Clinical Research Network (NASH CRN) Fibrosis Stage at Week 48 (Treatment Period) | ALXN1840 | Baseline / Score 0 | 7 | Participants | |
| Number of Participants With Change From Baseline in Nonalcoholic Steatohepatitis Clinical Research Network (NASH CRN) Fibrosis Stage at Week 48 (Treatment Period) | ALXN1840 | Baseline / Score 1a | 1 | Participants | |
| Number of Participants With Change From Baseline in Nonalcoholic Steatohepatitis Clinical Research Network (NASH CRN) Fibrosis Stage at Week 48 (Treatment Period) | ALXN1840 | Baseline / Score 1b | 0 | Participants | |
| Number of Participants With Change From Baseline in Nonalcoholic Steatohepatitis Clinical Research Network (NASH CRN) Fibrosis Stage at Week 48 (Treatment Period) | ALXN1840 | Baseline / Score 1c | 7 | Participants | |
| Number of Participants With Change From Baseline in Nonalcoholic Steatohepatitis Clinical Research Network (NASH CRN) Fibrosis Stage at Week 48 (Treatment Period) | ALXN1840 | Baseline / Score 2 | 3 | Participants | |
| Number of Participants With Change From Baseline in Nonalcoholic Steatohepatitis Clinical Research Network (NASH CRN) Fibrosis Stage at Week 48 (Treatment Period) | ALXN1840 | Baseline / Score 3 | 8 | Participants | |
| Number of Participants With Change From Baseline in Nonalcoholic Steatohepatitis Clinical Research Network (NASH CRN) Fibrosis Stage at Week 48 (Treatment Period) | ALXN1840 | Baseline / Score 4 | 2 | Participants | |
| Number of Participants With Change From Baseline in Nonalcoholic Steatohepatitis Clinical Research Network (NASH CRN) Fibrosis Stage at Week 48 (Treatment Period) | ALXN1840 | Baseline / Not evaluable | 1 | Participants | |
| Number of Participants With Change From Baseline in Nonalcoholic Steatohepatitis Clinical Research Network (NASH CRN) Fibrosis Stage at Week 48 (Treatment Period) | ALXN1840 | Week 48 / Score 0 | 5 | Participants |
1 to 10 of 66Source ClinicalTrials.gov
See all 66 results by groupParticipant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Treatment Period (48 Weeks) | STARTED | ALXN1840 | 31 | |
| Treatment Period (48 Weeks) | Received at Least 1 Dose of Study Drug | ALXN1840 | 31 | |
| Treatment Period (48 Weeks) | COMPLETED | ALXN1840 | 26 | |
| Treatment Period (48 Weeks) | NOT COMPLETED | ALXN1840 | 5 | |
| Extension Period (48 Weeks) | STARTED | ALXN1840 | 25 | |
| Extension Period (48 Weeks) | Received at Least 1 Dose of Study Drug | ALXN1840 | 25 | |
| Extension Period (48 Weeks) | COMPLETED | ALXN1840 | 21 | |
| Extension Period (48 Weeks) | NOT COMPLETED | ALXN1840 | 4 |
Source ClinicalTrials.gov
Adverse events354
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 | Treatment Period: ALXN1840 | 1 | 31 | |
| other | Leukopenia | Blood and lymphatic system disorders | Extension Period: ALXN1840 | 0 | 25 | |
| other | Neutropenia | Blood and lymphatic system disorders | Treatment Period: ALXN1840 | 1 | 31 | |
| other | Neutropenia | Blood and lymphatic system disorders | Extension Period: ALXN1840 | 1 | 25 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Treatment Period: ALXN1840 | 1 | 31 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Extension Period: ALXN1840 | 0 | 25 | |
| other | Tinnitus | Ear and labyrinth disorders | Treatment Period: ALXN1840 | 2 | 31 | |
| other | Tinnitus | Ear and labyrinth disorders | Extension Period: ALXN1840 | 2 | 25 | |
| other | Chalazion | Eye disorders | Treatment Period: ALXN1840 | 1 | 31 | |
| other | Chalazion | Eye disorders | Extension Period: ALXN1840 | 1 | 25 |
1 to 10 of 354Source ClinicalTrials.gov
See all 354 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Treatment Period: ALXN1840 | 0 | 31 |
| Total, all-cause mortality | Extension Period: ALXN1840 | 0 | 25 |
| Total, other adverse events | Treatment Period: ALXN1840 | 30 | 31 |
| Total, other adverse events | Extension Period: ALXN1840 | 24 | 25 |
| Total, serious adverse events | Treatment Period: ALXN1840 | 3 | 31 |
| Total, serious adverse events | Extension Period: ALXN1840 | 2 | 25 |
Source ClinicalTrials.gov
Papers0
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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Treatment Period: ALXN1840 vs Extension Period: ALXN1840: deaths from any cause | Trial comparisons |
| Treatment Period: ALXN1840 vs Extension Period: ALXN1840: serious adverse events | Trial comparisons |
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.
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