Trial · NCT04201262
An Efficacy and Safety Study of Ravulizumab in Adult Participants With NMOSD
NCT04201262, Phase 3, Completed, Alexion Pharmaceuticals, Inc.; results first posted 2023-08-09
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. An Efficacy and Safety Study of Ravulizumab in Adult Participants With NMOSD. Trials, record NCT04201262. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04201262
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- An Efficacy and Safety Study of Ravulizumab in Adult Participants With NMOSD
- NCT number
- NCT04201262
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 58 participants, actual
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Conditions
- Neuromyelitis Optica, Neuromyelitis Optica Spectrum Disorder
- Interventions
- Ravulizumab
- First posted
- 2019-12-17
- Last updated
- 2025-10-14
- Other registries' IDs
- eudract:2019-003352-37
- Results first posted
- 2023-08-09
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With an Adjudicated On-trial Relapse in the Primary Treatment Period | Baseline up to 2.25 years (end of the Primary Treatment Period) | 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 independent relapse adjudication committee. |
| Secondary | Adjudicated On-trial Annualized Relapse Rate (ARR) in the Primary Treatment Period | Baseline up to 2.25 years (end of the Primary Treatment Period) | The adjudicated On-trial ARR was computed as the total number of relapses divided by the total number of participant 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 centered on the mean historical ARR in the 24 months prior to screening. |
| Secondary | Number of Participants With Clinically Important Change From Baseline in Hauser Ambulation Index (HAI) Score at the End of Primary Treatment Period | Baseline up to 2.25 years (end of the Primary Treatment Period) | The HAI is a rating scale developed to assess mobility by evaluating the time and degree of assistance required to walk 25 feet. 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 wheelchair; unable to transfer self independently). Clinically important change is conditional on the baseline value: worsening if the baseline HAI is 0 and at least 2 points increase or if the baseline HAI is \>0 and at least 1 point increase; improvement if the baseline value is at least 2 and at least 1 point decrease; and stable if baseline is 0 or 1 and a 0- or 1-point increase or decrease or baseline is at least 2 and not change. |
| Secondary | Change From Baseline in European Quality of Life Health 5-dimension Questionnaire (EQ-5D) Index Score at the End of Primary Treatment Period | Baseline, up to 2.25 years (end of the Primary Treatment Period) | The EQ-5D is a generic, standardized, self-administered instrument that provides a simple, descriptive profile and a single index value for health status. It consists of 2 parts; the EQ-5D descriptive system and the EQ-5D visual analogue scale (VAS). The EQ-5D descriptive system includes 5 dimensions; mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Each dimension measured on 3 levels: "no problem" (level 1), "some problems" (level 2), "extreme problems" (level 3). The digits for 5 dimensions can be combined in a 5-digit number describing the respondent's health state. The 5 dimensional 3-level systems was converted into single index utility score that ranges from less than 0 to 1, with higher scores representing a better health status. |
| Secondary | Change From Baseline in EQ-5D Visual Analog Scale (VAS) Score at the End of Primary Treatment Period | Baseline, up to 2.25 years (end of the Primary Treatment Period) | The EQ-5D is a generic, standardized, self-administered instrument that provides a simple, descriptive profile and a single index value for health status. It consists of 2 parts; the EQ-5D descriptive system and the EQ-5D visual analogue scale (VAS). The EQ-5D VAS is an overall health state scale where the participant selects a number between 0 to 100 to describe the condition of their health, with 100 being 'The best health state you can imagine' and 0 being 'The worst health state you can imagine'. An increase in score indicates improvement. |
| Secondary | Number of Participants With Clinically Important Worsening From Baseline in Expanded Disability Status Scale (EDSS) Score at the End of Primary Treatment Period | Baseline up to 2.25 years (end of the Primary Treatment Period) | 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. Clinically important worsening was defined as an increase in EDSS score conditional on the baseline value: If the baseline EDSS was 0 and at least 2-point increase; if the baseline is 1 to 5, and at least 1-point increase; if the baseline is \> 5 and at least 0.5 increase. |
| Secondary | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and TEAEs Leading to Study Drug Discontinuation in the Primary Treatment Period | Baseline up to 2.25 years (end of the Primary Treatment Period) | An AE was 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. TEAEs were AEs with a start date on or after the date of the first dose of study drug. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. |
| Secondary | Serum Ravulizumab Concentration | Predose and end of infusion (EOI) at Week 26 | |
| Secondary | Change From Baseline in Serum Free C5 Concentration at Week 26 | Baseline, Week 26 (Predose and EOI) | |
| Secondary | Number of Participants With Anti-drug Antibodies (ADAs) During the Primary Treatment Period | Baseline, Weeks 26, 50, 82, and 106 |
Source ClinicalTrials.gov
Outcomes10
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 an Adjudicated On-trial Relapse in the Primary Treatment Period | Baseline up to 2.25 years (end of the Primary Treatment Period) | The full analysis set (FAS) included all participants who had received at least 1 dose of study drug (ravulizumab or placebo). | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Adjudicated On-trial Annualized Relapse Rate (ARR) in the Primary Treatment Period | Baseline up to 2.25 years (end of the Primary Treatment Period) | The FAS included all participants who had received at least 1 dose of study drug (ravulizumab or placebo). | relapses/year on study | NUMBER |
| SECONDARY | Number of Participants With Clinically Important Change From Baseline in Hauser Ambulation Index (HAI) Score at the End of Primary Treatment Period | Baseline up to 2.25 years (end of the Primary Treatment Period) | The FAS included all participants who had received at least 1 dose of study drug (ravulizumab or placebo). | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in European Quality of Life Health 5-dimension Questionnaire (EQ-5D) Index Score at the End of Primary Treatment Period | Baseline, up to 2.25 years (end of the Primary Treatment Period) | The FAS included all participants who had received at least 1 dose of study drug (ravulizumab or placebo). | units on a scale | MEAN |
| SECONDARY | Change From Baseline in EQ-5D Visual Analog Scale (VAS) Score at the End of Primary Treatment Period | Baseline, up to 2.25 years (end of the Primary Treatment Period) | The FAS included all participants who had received at least 1 dose of study drug (ravulizumab or placebo). | units on a scale | MEAN |
| SECONDARY | Number of Participants With Clinically Important Worsening From Baseline in Expanded Disability Status Scale (EDSS) Score at the End of Primary Treatment Period | Baseline up to 2.25 years (end of the Primary Treatment Period) | The FAS included all participants who had received at least 1 dose of study drug (ravulizumab or placebo). | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and TEAEs Leading to Study Drug Discontinuation in the Primary Treatment Period | Baseline up to 2.25 years (end of the Primary Treatment Period) | The safety set included all participants who received at least 1 dose of study drug (ravulizumab or placebo). | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Serum Ravulizumab Concentration | Predose and end of infusion (EOI) at Week 26 | Pharmacokinetics/pharmacodynamics (PK/PD) analysis set included participants who received at least 1 dose of study drug and who had at least 1 evaluable PK or PD result. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. 'Number analyzed' = participants evaluable at specified timepoint. | micrograms (µg)/milliliter (mL) | MEAN |
| SECONDARY | Change From Baseline in Serum Free C5 Concentration at Week 26 | Baseline, Week 26 (Predose and EOI) | The PK/PD analysis set included participants who received at least 1 dose of study drug and who had at least 1 evaluable PK or PD result. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. 'Number analyzed' = participants evaluable at specified timepoint. | µg/mL | MEAN |
| SECONDARY | Number of Participants With Anti-drug Antibodies (ADAs) During the Primary Treatment Period | Baseline, Weeks 26, 50, 82, and 106 | The safety set included all participants who received at least 1 dose of study drug (ravulizumab or placebo). Here, 'Number analyzed' = participants evaluable at specified timepoint. | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group38
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 an Adjudicated On-trial Relapse in the Primary Treatment Period | Ravulizumab | 0 | Participants | ||
| Number of Participants With an Adjudicated On-trial Relapse in the Primary Treatment Period | Placebo (ECU-NMO-301) | 20 | Participants | ||
| Adjudicated On-trial Annualized Relapse Rate (ARR) in the Primary Treatment Period | Ravulizumab | 0.000 | 95% Confidence Interval NA to NA | relapses/year on study | |
| Adjudicated On-trial Annualized Relapse Rate (ARR) in the Primary Treatment Period | Placebo (ECU-NMO-301) | 0.350 | 95% Confidence Interval 0.199 to 0.616 | relapses/year on study | |
| Number of Participants With Clinically Important Change From Baseline in Hauser Ambulation Index (HAI) Score at the End of Primary Treatment Period | Ravulizumab | Clinical Improvement | 4 | Participants | |
| Number of Participants With Clinically Important Change From Baseline in Hauser Ambulation Index (HAI) Score at the End of Primary Treatment Period | Placebo (ECU-NMO-301) | Clinical Improvement | 4 | Participants | |
| Number of Participants With Clinically Important Change From Baseline in Hauser Ambulation Index (HAI) Score at the End of Primary Treatment Period | Ravulizumab | Stable | 52 | Participants | |
| Number of Participants With Clinically Important Change From Baseline in Hauser Ambulation Index (HAI) Score at the End of Primary Treatment Period | Placebo (ECU-NMO-301) | Stable | 32 | Participants | |
| Number of Participants With Clinically Important Change From Baseline in Hauser Ambulation Index (HAI) Score at the End of Primary Treatment Period | Ravulizumab | Clinical Worsening | 2 | Participants | |
| Number of Participants With Clinically Important Change From Baseline in Hauser Ambulation Index (HAI) Score at the End of Primary Treatment Period | Placebo (ECU-NMO-301) | Clinical Worsening | 11 | Participants |
1 to 10 of 38Source ClinicalTrials.gov
See all 38 results by groupParticipant flow16
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Primary Treatment Period | STARTED | Ravulizumab | 58 | |
| Primary Treatment Period | STARTED | Placebo (ECU-NMO-301) | 47 | |
| Primary Treatment Period | Received at Least 1 Dose of Study Drug | Ravulizumab | 58 | |
| Primary Treatment Period | Received at Least 1 Dose of Study Drug | Placebo (ECU-NMO-301) | 47 | |
| Primary Treatment Period | COMPLETED | Ravulizumab | 56 | |
| Primary Treatment Period | COMPLETED | Placebo (ECU-NMO-301) | 44 | |
| Primary Treatment Period | NOT COMPLETED | Ravulizumab | 2 | |
| Primary Treatment Period | NOT COMPLETED | Placebo (ECU-NMO-301) | 3 | |
| Long-term Extension Period | STARTED | Ravulizumab | 56 | |
| Long-term Extension Period | STARTED | Placebo (ECU-NMO-301) | 0 |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 participant flowAdverse events1,308
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 | Lymphadenopathy | Blood and lymphatic system disorders | Primary Treatment Period: Placebo | 1 | 47 | 2 |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Primary Treatment Period: Ravulizumab | 3 | 58 | 3 |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Long-Term Extension: Ravulizumab | 1 | 56 | 1 |
| other | Anaemia | Blood and lymphatic system disorders | Primary Treatment Period: Placebo | 1 | 47 | 1 |
| other | Anaemia | Blood and lymphatic system disorders | Primary Treatment Period: Ravulizumab | 1 | 58 | 1 |
| other | Anaemia | Blood and lymphatic system disorders | Long-Term Extension: Ravulizumab | 0 | 56 | 0 |
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | Primary Treatment Period: Placebo | 1 | 47 | 1 |
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | Primary Treatment Period: Ravulizumab | 0 | 58 | 0 |
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | Long-Term Extension: Ravulizumab | 1 | 56 | 1 |
| other | Leukopenia | Blood and lymphatic system disorders | Primary Treatment Period: Placebo | 1 | 47 | 1 |
1 to 10 of 1,308Source ClinicalTrials.gov
See all 1,308 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Primary Treatment Period: Placebo | 0 | 47 |
| Total, all-cause mortality | Primary Treatment Period: Ravulizumab | 0 | 58 |
| Total, all-cause mortality | Long-Term Extension: Ravulizumab | 1 | 56 |
| Total, other adverse events | Primary Treatment Period: Placebo | 43 | 47 |
| Total, other adverse events | Primary Treatment Period: Ravulizumab | 53 | 58 |
| Total, other adverse events | Long-Term Extension: Ravulizumab | 49 | 56 |
| Total, serious adverse events | Primary Treatment Period: Placebo | 26 | 47 |
| Total, serious adverse events | Primary Treatment Period: Ravulizumab | 8 | 58 |
| Total, serious adverse events | Long-Term Extension: Ravulizumab | 9 | 56 |
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 |
|---|
| Ravulizumab vs Placebo (ECU-NMO-301): Number of Participants With an Adjudicated On-trial Relapse in the Primary Treatment Period |
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/NCT04201262
GET https://conductscience.org/api/trials/v1/NCT04201262/results?page=2