Trial · NCT01892345
A Randomized Controlled Trial of Eculizumab in AQP4 Antibody-positive Participants With NMO (PREVENT Study)
NCT01892345, Phase 3, Terminated, Alexion Pharmaceuticals, Inc.; results first posted 2019-06-26
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Randomized Controlled Trial of Eculizumab in AQP4 Antibody-positive Participants With NMO (PREVENT Study). Trials, record NCT01892345. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01892345
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Randomized Controlled Trial of Eculizumab in AQP4 Antibody-positive Participants With NMO (PREVENT Study)
- NCT number
- NCT01892345
- Status
- Terminated
- Phases
- Phase 3
- Enrollment
- 143 participants, actual
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Conditions
- Neuromyelitis Optica, Neuromyelitis Optica Spectrum Disorder
- Interventions
- Eculizumab, Placebo
- First posted
- 2013-07-04
- Last updated
- 2019-06-26
- Results first posted
- 2019-06-26
- Limitations and caveats
After rigorous review of blinded study data, the Sponsor terminated the study at 23 adjudicated events, not the protocol-specified 24. This was not driven by safety or efficacy concerns, but rather by uncertainty in estimating final event occurrence.
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Participants With An Adjudicated On-trial Relapse | Baseline, Up To 211 Weeks (End of Study) | 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. An adjudicated On-trial Relapse was defined by the protocol and positively adjudicated by the relapse adjudication committee. |
| Secondary | Adjudicated On-trial Annualized Relapse Rate (ARR) | Baseline, Up To 211 Weeks (End of Study) | The adjudicated On-trial ARR was computed as the total number of relapses divided by the total number of patient years in the study period. A central independent committee was used to adjudicate all On-trial Relapses as determined by the treating physician. Results reported as adjusted adjudicated On-trial ARR based on a Poisson regression adjusted for randomization strata and historical ARR in 24 months prior to Screening. |
| Secondary | Change From Baseline In EDSS At End Of Study | Baseline, Up To 211 Weeks (End of Study) | Disease-related disability was measured by the EDSS. 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. |
| Secondary | Change From Baseline In Modified Rankin Scale (mRS) Score At End Of Study | Baseline, Up To 211 Weeks (End of Study) | 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 disability) to 6 (death) in whole-point increments. A decrease in score indicates improvement. |
| Secondary | Change From Baseline In Hauser Ambulation Index (HAI) Score At End of Study | Baseline, Up To 211 Weeks (End of Study) | 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 ambulatory with no assistance) and 9 being the worst (restricted to wheel chair; unable to transfer self independently). A decrease in score indicates improvement. |
| Secondary | Change From Baseline In European Quality Of Life (EuroQoL) Health 5-Dimension Questionnaire (EQ-5D) Visual Analogue Scale At End Of Study | Baseline, Up To 211 Weeks (End of Study) | The EuroQoL EQ-5D is a generic, standardized, self-administered instrument that provides a simple, descriptive profile and a single index value for health status. Assessments were made using the EQ-5D Visual Analogue Scale, which captures the self-rating of current health status using a visual "thermometer" with the endpoints of 100 (best imaginable health state) at the top and zero (worst imaginable health state) at the bottom. An increase in score indicates improvement. |
| Secondary | Change From Baseline In EuroQoL EQ-5D Index Score At End Of Study | Baseline, Up To 211 Weeks (End of Study) | The EuroQoL EQ-5D is a generic, standardized, self-administered instrument that provides a simple, descriptive profile and a single index value for health status. Index scores range from less than 0 to 1, with higher scores representing a better health status. |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Participants With An Adjudicated On-trial Relapse | Baseline, Up To 211 Weeks (End of Study) | Full Analysis Set (FAS): all participants who were randomized to treatment, received at least 1 dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Adjudicated On-trial Annualized Relapse Rate (ARR) | Baseline, Up To 211 Weeks (End of Study) | Full Analysis Set (FAS): all participants who were randomized to treatment and who received at least 1 dose of study drug. | relapses/years on study | NUMBER |
| SECONDARY | Change From Baseline In EDSS At End Of Study | Baseline, Up To 211 Weeks (End of Study) | Full Analysis Set (FAS): all participants who were randomized to treatment and who received at least 1 dose of study drug. | units on a scale | MEAN |
| SECONDARY | Change From Baseline In Modified Rankin Scale (mRS) Score At End Of Study | Baseline, Up To 211 Weeks (End of Study) | Full Analysis Set (FAS): all participants who were randomized to treatment and who received at least 1 dose of study drug. | units on a scale | MEAN |
| SECONDARY | Change From Baseline In Hauser Ambulation Index (HAI) Score At End of Study | Baseline, Up To 211 Weeks (End of Study) | Full Analysis Set (FAS): all participants who were randomized to treatment and who received at least 1 dose of study drug. | units on a scale | MEAN |
| SECONDARY | Change From Baseline In European Quality Of Life (EuroQoL) Health 5-Dimension Questionnaire (EQ-5D) Visual Analogue Scale At End Of Study | Baseline, Up To 211 Weeks (End of Study) | Full Analysis Set (FAS): all participants who were randomized to treatment and who received at least 1 dose of study drug. | units on a scale | MEAN |
| SECONDARY | Change From Baseline In EuroQoL EQ-5D Index Score At End Of Study | Baseline, Up To 211 Weeks (End of Study) | Full Analysis Set (FAS): all participants who were randomized to treatment and who received at least 1 dose of study drug. | units on a scale | MEAN |
Source ClinicalTrials.gov
Results by group14
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Participants With An Adjudicated On-trial Relapse | Eculizumab | 3 | Participants | ||
| Participants With An Adjudicated On-trial Relapse | Placebo | 20 | Participants | ||
| Adjudicated On-trial Annualized Relapse Rate (ARR) | Eculizumab | 0.016 | 95% Confidence Interval 0.005 to 0.050 | relapses/years on study | |
| Adjudicated On-trial Annualized Relapse Rate (ARR) | Placebo | 0.350 | 95% Confidence Interval 0.199 to 0.616 | relapses/years on study | |
| Change From Baseline In EDSS At End Of Study | Eculizumab | -0.18 | Standard Deviation 0.814 | units on a scale | |
| Change From Baseline In EDSS At End Of Study | Placebo | 0.12 | Standard Deviation 0.945 | units on a scale | |
| Change From Baseline In Modified Rankin Scale (mRS) Score At End Of Study | Eculizumab | -0.2 | Standard Deviation 0.72 | units on a scale | |
| Change From Baseline In Modified Rankin Scale (mRS) Score At End Of Study | Placebo | 0.1 | Standard Deviation 0.75 | units on a scale | |
| Change From Baseline In Hauser Ambulation Index (HAI) Score At End of Study | Eculizumab | -0.4 | Standard Deviation 1.08 | units on a scale | |
| Change From Baseline In Hauser Ambulation Index (HAI) Score At End of Study | Placebo | 0.5 | Standard Deviation 1.61 | units on a scale |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 results by groupParticipant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Eculizumab | 96 | |
| Overall Study | STARTED | Placebo | 47 | |
| Overall Study | Received At Least 1 Dose Of Study Drug | Eculizumab | 96 | |
| Overall Study | Received At Least 1 Dose Of Study Drug | Placebo | 47 | |
| Overall Study | COMPLETED | Eculizumab | 80 | |
| Overall Study | COMPLETED | Placebo | 44 | |
| Overall Study | NOT COMPLETED | Eculizumab | 16 | |
| Overall Study | NOT COMPLETED | Placebo | 3 |
Source ClinicalTrials.gov
Adverse 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 | Leukopenia | Blood and lymphatic system disorders | Eculizumab | 5 | 96 | |
| other | Leukopenia | Blood and lymphatic system disorders | Placebo | 1 | 47 | |
| other | Lymphopenia | Blood and lymphatic system disorders | Eculizumab | 5 | 96 | |
| other | Lymphopenia | Blood and lymphatic system disorders | Placebo | 0 | 47 | |
| other | Cataract | Eye disorders | Eculizumab | 6 | 96 | |
| other | Cataract | Eye disorders | Placebo | 2 | 47 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Eculizumab | 5 | 96 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Placebo | 3 | 47 | |
| other | Constipation | Gastrointestinal disorders | Eculizumab | 9 | 96 | |
| other | Constipation | Gastrointestinal disorders | Placebo | 3 | 47 |
1 to 10 of 228Source ClinicalTrials.gov
See all 228 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Eculizumab | 1 | 96 |
| Total, all-cause mortality | Placebo | 0 | 47 |
| Total, other adverse events | Eculizumab | 86 | 96 |
| Total, other adverse events | Placebo | 43 | 47 |
| Total, serious adverse events | Eculizumab | 30 | 96 |
| Total, serious adverse events | Placebo | 26 | 47 |
Source ClinicalTrials.gov
Papers6
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 comparisons3
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Eculizumab vs Placebo: Participants With An Adjudicated On-trial Relapse | Trial comparisons |
| Eculizumab vs Placebo: deaths from any cause | Trial comparisons |
| Eculizumab vs Placebo: 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.
GET https://conductscience.org/api/trials/v1/NCT01892345
GET https://conductscience.org/api/trials/v1/NCT01892345/events?page=2