Trial · NCT02003144
An Open Label Extension Trial of Eculizumab in Relapsing NMO Patients
NCT02003144, Phase 3, Completed, Alexion Pharmaceuticals, Inc.; results first posted 2022-08-23
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. An Open Label Extension Trial of Eculizumab in Relapsing NMO Patients. Trials, record NCT02003144. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02003144
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- An Open Label Extension Trial of Eculizumab in Relapsing NMO Patients
- NCT number
- NCT02003144
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 119 participants, actual
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Conditions
- Neuromyelitis Optica, Neuromyelitis Optica Spectrum Disorder
- Interventions
- eculizumab
- First posted
- 2013-12-06
- Last updated
- 2022-08-23
- Other registries' IDs
- eudract:2013-001151-12
- Results first posted
- 2022-08-23
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events | Baseline up to end of study (up to 6.5 years) | An adverse event (AE) was defined as any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. Treatment-emergent adverse events (TEAEs) were defined as an AE with onset on or after the first study drug dose in Study ECU-NMO-302. A serious adverse event (SAE) was defined as an untoward medical occurrence that at any dose either results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, or is an important medical event. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module. |
| Primary | Number of Participants With At Least 1 Post Baseline Columbia-Suicide Severity Rating Scale (C-SSRS) Assessment (Suicide-Related Thoughts or Behaviours) Abnormality | Baseline up to end of study (up to 6.5 years) | The C-SSRS is a validated questionnaire to capture occurrence, severity, and frequency of suicide-related thoughts and behaviours, and has a binary response (yes/no). Suicidal Ideation: a "yes" answer to any one of 5 suicidal ideation questions: Wish to be Dead; Non-specific Active Suicidal Thoughts; Active Suicidal Ideation with Any Methods (Not Planned) without Intent to Act; Active Suicidal Ideation with Some Intent to Act, without Specific Plan; and Active Suicidal Ideation with Specific Plan and Intent. Suicidal Behaviour: a "yes" answer to any of 5 suicidal behaviour questions: Preparatory Acts or Behaviour, Aborted Attempt, Interrupted Attempt, Actual Attempt (non-fatal), and Completed Suicide. Suicidal Ideation or Behaviour: a "yes" answer to the following question: Self-injurious behaviour without suicidal intent. |
| Primary | Number of Participants With An On-trial Relapse as Determined by The Treating Physician | Baseline up to end of study (up to 6.5 years) | An On-trial Relapse was defined as a new onset of neurologic symptoms or worsening of existing neurologic symptoms with an objective change (clinical sign) on neurologic examination that persisted for more than 24 hours as confirmed by the treating physician. |
| Primary | On-Trial Annualized Relapse Rate (ARR) as Determined by The Treating Physician | Baseline up to end of study (up to 6.5 years) | The On-trial ARR was computed as the total number of relapses divided by the total number of participant years in the study period. |
| Secondary | Change From Baseline in Expanded Disability Status Scale (EDSS) Score | Baseline, Weeks 52, 104 and 156 | Disease-related disability was measured by the EDSS. The EDSS quantifies disability in 8 Functional Systems (FS) and allows neurologists to assign a Functional System Score (FSS) in each of these. The Functional Systems are pyramidal, cerebellar, brainstem, sensory, bowel and bladder, visual, cerebral, and other. The EDSS is an ordinal clinical rating scale that ranges from 0 (normal neurologic examination) to 10 (death) in half-point increments. A decrease in score indicates improvement. Baseline was defined as the last available assessment prior to the first study drug infusion in Study ECU-NMO-302. |
| Secondary | Change From Baseline in Modified Rankin Scale (mRS) Score | Baseline, Weeks 52, 104 and 156 | Disease-related disability was measured by the mRS score. The mRS is a commonly used scale for measuring the degree of disability or dependence in the daily activities of people who have suffered from a neurological disability. The scale ranges from 0 (no symptoms at all) to 6 (death) in whole-point increments. A decrease in score indicates improvement. Baseline was defined as the last available assessment prior to the first study drug infusion in Study ECU-NMO-302. |
| Secondary | Change From Baseline in Hauser Ambulation Index (HAI) in Participants With Abnormal Baseline Ambulatory Function | Baseline, Weeks 52, 104 and 156 | The HAI evaluates gait and was used to assess the time and effort used by the participant to walk 25 feet (8 meters). The scale ranges from 0 to 9, with 0 being the best score (asymptomatic; fully active) and 9 being the worst (restricted to wheelchair; unable to transfer self independently). A decrease in score indicates improvement. Baseline is defined as the last available assessment prior to the first study drug infusion in Study ECU-NMO-302. |
| Secondary | Change From Baseline in European Quality of Life (EuroQoL) 5-Dimension Visual Analog Scale (EQ-5D VAS) Health Status Score | Baseline, Weeks 52, 104 and 156 | The EQ-5D is a generic, standardized participant self-administered health status instrument. EQ-5D general health status can also be measured by a visual analog scale (EQ-5D VAS). EQ-5D-VAS recorded the participant's self-rated health on a vertical visual analog scale (VAS) that allowed the participants to indicate their health state that ranged from 0 (worst imaginable) to 100 (best imaginable). Baseline is defined as the last available assessment prior to the first study drug infusion in Study ECU-NMO-302. |
| Secondary | Change From Baseline in Kurtzke Visual Functional System Scores (FSS) in Participants With Abnormal Baseline Visual Function | Baseline, Weeks 52, 104 and 156 | The EDSS assesses multiple Kurtzke functional systems in the context of a standard neurological exam, including visual function. The visual score ranges from 0 to 6. A score of 0 implies the participant has normal visual function. Higher scores represent worse disability. Baseline is defined as the last available assessment prior to the first study drug infusion in Study EC-NMO-302. |
Source ClinicalTrials.gov
Outcomes9
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events | Baseline up to end of study (up to 6.5 years) | The Extension Safety Set consisted of all participants who had received at least 1 dose of eculizumab in Study ECU-NMO-302. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With At Least 1 Post Baseline Columbia-Suicide Severity Rating Scale (C-SSRS) Assessment (Suicide-Related Thoughts or Behaviours) Abnormality | Baseline up to end of study (up to 6.5 years) | The Extension Safety Set consisted of all participants who had received at least 1 dose of eculizumab in Study ECU-NMO-302. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With An On-trial Relapse as Determined by The Treating Physician | Baseline up to end of study (up to 6.5 years) | The Extension Full Analysis Set consisted of all participants who had received at least 1 dose of eculizumab in Study ECU-NMO-302 and had a post-IP-infusion efficacy assessment. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | On-Trial Annualized Relapse Rate (ARR) as Determined by The Treating Physician | Baseline up to end of study (up to 6.5 years) | The Extension Full Analysis Set consisted of all participants who had received at least 1 dose of eculizumab in Study ECU-NMO-302 and had a post-IP-infusion efficacy assessment. | relapses/years on study | MEAN |
| SECONDARY | Change From Baseline in Expanded Disability Status Scale (EDSS) Score | Baseline, Weeks 52, 104 and 156 | The Extension Full Analysis Set consisted of all participants who had received at least 1 dose of eculizumab in Study ECU-NMO-302 and had a post-IP-infusion efficacy assessment. Here, Number Analyzed signifies those participants who were evaluable at specified time points. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in Modified Rankin Scale (mRS) Score | Baseline, Weeks 52, 104 and 156 | The Extension Full Analysis Set consisted of all participants who had received at least 1 dose of eculizumab in Study ECU-NMO-302 and had a post-IP-infusion efficacy assessment. Here, Number Analyzed signifies those participants who were evaluable at specified time points. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in Hauser Ambulation Index (HAI) in Participants With Abnormal Baseline Ambulatory Function | Baseline, Weeks 52, 104 and 156 | The Extension Full Analysis Set consisted of all participants who had received at least 1 dose of eculizumab in Study ECU-NMO-302 and had a post-IP-infusion efficacy assessment. Here, Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure and Number Analyzed signifies those participants who were evaluable at specified time points. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in European Quality of Life (EuroQoL) 5-Dimension Visual Analog Scale (EQ-5D VAS) Health Status Score | Baseline, Weeks 52, 104 and 156 | The Extension Full Analysis Set consisted of all participants who had received at least 1 dose of eculizumab in Study ECU-NMO-302 and had a post-IP-infusion efficacy assessment. Here, Number Analyzed signifies those participants who were evaluable at specified time points. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in Kurtzke Visual Functional System Scores (FSS) in Participants With Abnormal Baseline Visual Function | Baseline, Weeks 52, 104 and 156 | The Extension Full Analysis Set consisted of all participants who had received at least 1 dose of eculizumab in Study ECU-NMO-302 and had a post-IP-infusion efficacy assessment. Here, Number of Participants analyzed signifies those participants who were evaluable for this outcome measure and Number Analyzed signifies those participants who were evaluable at specified time points. | units on a scale | MEAN |
Source ClinicalTrials.gov
Results by group54
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 and Serious Adverse Events | Placebo/Eculizumab | TEAEs | 41 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events | Eculizumab/Eculizumab | TEAEs | 70 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events | Placebo/Eculizumab | SAEs | 14 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events | Eculizumab/Eculizumab | SAEs | 26 | Participants | |
| Number of Participants With At Least 1 Post Baseline Columbia-Suicide Severity Rating Scale (C-SSRS) Assessment (Suicide-Related Thoughts or Behaviours) Abnormality | Placebo/Eculizumab | Suicidal Ideation | 4 | Participants | |
| Number of Participants With At Least 1 Post Baseline Columbia-Suicide Severity Rating Scale (C-SSRS) Assessment (Suicide-Related Thoughts or Behaviours) Abnormality | Eculizumab/Eculizumab | Suicidal Ideation | 5 | Participants | |
| Number of Participants With At Least 1 Post Baseline Columbia-Suicide Severity Rating Scale (C-SSRS) Assessment (Suicide-Related Thoughts or Behaviours) Abnormality | Placebo/Eculizumab | Suicidal Behavior | 0 | Participants | |
| Number of Participants With At Least 1 Post Baseline Columbia-Suicide Severity Rating Scale (C-SSRS) Assessment (Suicide-Related Thoughts or Behaviours) Abnormality | Eculizumab/Eculizumab | Suicidal Behavior | 1 | Participants | |
| Number of Participants With At Least 1 Post Baseline Columbia-Suicide Severity Rating Scale (C-SSRS) Assessment (Suicide-Related Thoughts or Behaviours) Abnormality | Placebo/Eculizumab | Suicidal Ideation or Behavior | 4 | Participants | |
| Number of Participants With At Least 1 Post Baseline Columbia-Suicide Severity Rating Scale (C-SSRS) Assessment (Suicide-Related Thoughts or Behaviours) Abnormality | Eculizumab/Eculizumab | Suicidal Ideation or Behavior | 5 | Participants |
1 to 10 of 54Source ClinicalTrials.gov
See all 54 results by groupParticipant flow16
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Blind Induction Phase | STARTED | Placebo/Eculizumab | 41 | |
| Blind Induction Phase | STARTED | Eculizumab/Eculizumab | 78 | |
| Blind Induction Phase | Received at Least 1 Dose of Study Drug | Placebo/Eculizumab | 41 | |
| Blind Induction Phase | Received at Least 1 Dose of Study Drug | Eculizumab/Eculizumab | 78 | |
| Blind Induction Phase | COMPLETED | Placebo/Eculizumab | 41 | |
| Blind Induction Phase | COMPLETED | Eculizumab/Eculizumab | 78 | |
| Blind Induction Phase | NOT COMPLETED | Placebo/Eculizumab | 0 | |
| Blind Induction Phase | NOT COMPLETED | Eculizumab/Eculizumab | 0 | |
| Open Label Maintenance Phase | STARTED | Placebo/Eculizumab | 41 | |
| Open Label Maintenance Phase | STARTED | Eculizumab/Eculizumab | 78 |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 participant flowAdverse events333
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 | Anaemia | Blood and lymphatic system disorders | Placebo/Eculizumab | 3 | 41 | 3 |
| other | Anaemia | Blood and lymphatic system disorders | Eculizumab/Eculizumab | 6 | 78 | 7 |
| other | Anaemia | Blood and lymphatic system disorders | Eculizumab (Combined Total) | 9 | 119 | 10 |
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | Placebo/Eculizumab | 2 | 41 | 2 |
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | Eculizumab/Eculizumab | 5 | 78 | 5 |
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | Eculizumab (Combined Total) | 7 | 119 | 7 |
| other | Diarrhoea | Gastrointestinal disorders | Placebo/Eculizumab | 8 | 41 | 17 |
| other | Diarrhoea | Gastrointestinal disorders | Eculizumab/Eculizumab | 6 | 78 | 11 |
| other | Diarrhoea | Gastrointestinal disorders | Eculizumab (Combined Total) | 14 | 119 | 28 |
| other | Constipation | Gastrointestinal disorders | Placebo/Eculizumab | 4 | 41 | 5 |
1 to 10 of 333Source ClinicalTrials.gov
See all 333 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Placebo/Eculizumab | 0 | 41 |
| Total, all-cause mortality | Eculizumab/Eculizumab | 0 | 78 |
| Total, all-cause mortality | Eculizumab (Combined Total) | 0 | 119 |
| Total, other adverse events | Placebo/Eculizumab | 40 | 41 |
| Total, other adverse events | Eculizumab/Eculizumab | 70 | 78 |
| Total, other adverse events | Eculizumab (Combined Total) | 110 | 119 |
| Total, serious adverse events | Placebo/Eculizumab | 14 | 41 |
| Total, serious adverse events | Eculizumab/Eculizumab | 26 | 78 |
| Total, serious adverse events | Eculizumab (Combined Total) | 40 | 119 |
Source ClinicalTrials.gov
Papers3
The papers the trial lists on ClinicalTrials.gov.
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 comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| Eculizumab/Eculizumab vs Placebo/Eculizumab: Number of Participants With An On-trial Relapse as Determined by The Treating Physician |
Source 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.
GET https://conductscience.org/api/trials/v1/NCT02003144
GET https://conductscience.org/api/trials/v1/NCT02003144/results?page=2