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CompletedNCT04201262Updated Oct 14, 2025Results posted

An Efficacy and Safety Study of Ravulizumab in Adult Participants With NMOSD

A Phase 3 interventional study of Ravulizumab in Neuromyelitis Optica and Neuromyelitis Optica Spectrum Disorder, sponsored by Alexion Pharmaceuticals, Inc.. Completed at 41 sites in 13 countries. Open to participants aged 18 Years to 100 Years. Per ClinicalTrials.gov, last updated 2025-10-14.

Sponsored by Alexion Pharmaceuticals, Inc. · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
58
Allocation
Non-randomized
Ages
18 Years to 100 Years
Sex
All
01

Study summary

The primary purpose of this study is to evaluate the efficacy and safety of ravulizumab for the treatment of adult participants with NMOSD.

02

Conditions studied

  • Neuromyelitis Optica
  • Neuromyelitis Optica Spectrum Disorder

Keywords

  • Neuromyelitis Optica
  • Neuromyelitis Optica Spectrum Disorder
  • Devic's Disease
  • Transverse Myelitis
  • Optic Neuritis
  • Relapse
  • Ravulizumab
  • Ultomiris
  • ALXN1210
  • CNS Autoimmune Disorders
  • Demyelinating Disorders
  • NMO
  • NMOSD
03

Who can participate

Ages eligible
18 Years to 100 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  1. Anti-aquaporin-4 antibody-positive and a diagnosis of NMOSD as defined by the 2015 international consensus diagnostic criteria.
  2. At least 1 attack or relapse in the last 12 months prior to the Screening Period.
  3. Expanded Disability Status Scale score ≤7.
  4. Participants who enter the study receiving supportive immunosuppressive therapy must be on a stable dosing regimen of adequate duration prior to Screening.
  5. Body weight ≥40 kilograms.
  6. Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.

Exclusion criteria

Exclusion Criteria:

  1. History of Neisseria meningitidis infection.
  2. Human immunodeficiency virus (HIV) infection (evidenced by HIV-1 or HIV-2 antibody titer).
  3. Previously or currently treated with a complement inhibitor.
  4. Use of rituximab or mitoxantrone within 3 months prior to Screening.
  5. Use of IV immunoglobulin within 3 weeks prior to Screening.
04

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Single group
Masking
None (open label)
Enrollment
58 participants (actual)

Study arms

  • Experimental
    Ravulizumab

    During the Primary Treatment Period, all participants will receive open-label ravulizumab via intravenous (IV) infusion starting on Day 1. The end of the Primary Treatment Period will be triggered when the last enrolled participant completes between 26 and 50 weeks in the study (depending on the number of adjudicated On-Trial Relapse observed). After completion of the Primary Treatment Period, all participants will have the opportunity to continue receiving ravulizumab in the Long-Term Extension Period of the study. For each participant, the Long-Term Extension Period continues for up to 3 years, or until ravulizumab is approved and/or available (in accordance with country-specific regulations), whichever occurs first.

    Biological: Ravulizumab

Interventions

  • BiologicalRavulizumab

    Participants will receive a weight-based loading dose of ravulizumab via IV infusion on Day 1, followed by weight-based maintenance doses on Day 15, then once every 8 weeks until end of the Long-Term Extension Period.

    Also known as: ALXN1210, Ultomiris

05

What researchers measure

Primary outcomes

  1. Number of Participants With an Adjudicated On-trial Relapse in 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.

    Time frame: Baseline up to 2.25 years (end of the Primary Treatment Period)

Secondary outcomes

  1. Adjudicated On-trial Annualized Relapse Rate (ARR) in 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.

    Time frame: Baseline up to 2.25 years (end of the Primary Treatment Period)

  2. Number of Participants With Clinically Important Change From Baseline in Hauser Ambulation Index (HAI) Score at the End of 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.

    Time frame: Baseline up to 2.25 years (end of the Primary Treatment Period)

  3. Change From Baseline in European Quality of Life Health 5-dimension Questionnaire (EQ-5D) Index Score at the End of 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.

    Time frame: Baseline, up to 2.25 years (end of the Primary Treatment Period)

  4. Change From Baseline in EQ-5D Visual Analog Scale (VAS) Score at the End of 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.

    Time frame: Baseline, up to 2.25 years (end of the Primary Treatment Period)

  5. Number of Participants With Clinically Important Worsening From Baseline in Expanded Disability Status Scale (EDSS) Score at the End of 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.

    Time frame: Baseline up to 2.25 years (end of the Primary Treatment Period)

  6. 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

    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.

    Time frame: Baseline up to 2.25 years (end of the Primary Treatment Period)

  7. Serum Ravulizumab Concentration

    Time frame: Predose and end of infusion (EOI) at Week 26

  8. Change From Baseline in Serum Free C5 Concentration at Week 26

    Time frame: Baseline, Week 26 (Predose and EOI)

  9. Number of Participants With Anti-drug Antibodies (ADAs) During the Primary Treatment Period

    Time frame: Baseline, Weeks 26, 50, 82, and 106

06

Results

Posted Aug 9, 2023

Participant flow

This study utilized the placebo group from Study ECU-NMO-301 (NCT01892345) as an external placebo control.

Primary Treatment Period
Participant flow — Primary Treatment Period
MilestoneRavulizumabPlacebo (ECU-NMO-301)
Started5847
Received at least 1 dose of study drug5847
Completed5644
Not completed23
Withdrew: Adverse event12
Withdrew: Physician decision11
Long-term Extension Period
Participant flow — Long-term Extension Period
MilestoneRavulizumabPlacebo (ECU-NMO-301)
Started560
Received at least 1 dose of study drug560
Completed550
Not completed10
Withdrew: Death10

Outcome measures

PrimaryNumber of Participants With an Adjudicated On-trial Relapse in 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.

Time frame:
Baseline up to 2.25 years (end of the Primary Treatment Period)
Reported as:
Count of participants · Participants
Number of Participants With an Adjudicated On-trial Relapse in the Primary Treatment Period
ParticipantsRavulizumabPlacebo (ECU-NMO-301)
Number of Participants With an Adjudicated On-trial Relapse in the Primary Treatment Period020
Statistical analysis
  • Ravulizumab vs Placebo (ECU-NMO-301) · Log Rank · p = < 0.0001 · Hazard ratio (hr): 0.014 · 95% CI 0.000 to 0.103HR based on a Cox proportional hazards model, with Firth's adjustment. Confidence interval (CI)= Wald CI or Profile Likelihood CI Limits. HR for ravulizumab compared with placebo presented a 98.6% reduction in risk of relapse, 95% CI (89.7%, 100.0%).
SecondaryAdjudicated On-trial Annualized Relapse Rate (ARR) in 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.

Time frame:
Baseline up to 2.25 years (end of the Primary Treatment Period)
Reported as:
Number · relapses/year on study
Adjudicated On-trial Annualized Relapse Rate (ARR) in the Primary Treatment Period
relapses/year on studyRavulizumabPlacebo (ECU-NMO-301)
Adjudicated On-trial Annualized Relapse Rate (ARR) in the Primary Treatment Period0.000 (NA to NA)0.350 (0.199 to 0.616)
SecondaryNumber of Participants With Clinically Important Change From Baseline in Hauser Ambulation Index (HAI) Score at the End of 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.

Time frame:
Baseline up to 2.25 years (end of the Primary Treatment Period)
Reported as:
Count of participants · Participants
Number of Participants With Clinically Important Change From Baseline in Hauser Ambulation Index (HAI) Score at the End of Primary Treatment Period
ParticipantsRavulizumabPlacebo (ECU-NMO-301)
Clinical Improvement44
Stable5232
Clinical Worsening211
Statistical analysis
  • Ravulizumab vs Placebo (ECU-NMO-301) · Univariate models · p = 0.0122 (Proportional Odds p-value)
SecondaryChange From Baseline in European Quality of Life Health 5-dimension Questionnaire (EQ-5D) Index Score at the End of 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.

Time frame:
Baseline, up to 2.25 years (end of the Primary Treatment Period)
Reported as:
Mean · units on a scale
Change From Baseline in European Quality of Life Health 5-dimension Questionnaire (EQ-5D) Index Score at the End of Primary Treatment Period
units on a scaleRavulizumabPlacebo (ECU-NMO-301)
Change From Baseline in European Quality of Life Health 5-dimension Questionnaire (EQ-5D) Index Score at the End of Primary Treatment Period0.005 ± 0.1522-0.043 ± 0.2115
SecondaryChange From Baseline in EQ-5D Visual Analog Scale (VAS) Score at the End of 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.

Time frame:
Baseline, up to 2.25 years (end of the Primary Treatment Period)
Reported as:
Mean · units on a scale
Change From Baseline in EQ-5D Visual Analog Scale (VAS) Score at the End of Primary Treatment Period
units on a scaleRavulizumabPlacebo (ECU-NMO-301)
Change From Baseline in EQ-5D Visual Analog Scale (VAS) Score at the End of Primary Treatment Period2.6 ± 14.070.6 ± 16.39
SecondaryNumber of Participants With Clinically Important Worsening From Baseline in Expanded Disability Status Scale (EDSS) Score at the End of 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.

Time frame:
Baseline up to 2.25 years (end of the Primary Treatment Period)
Reported as:
Count of participants · Participants
Number of Participants With Clinically Important Worsening From Baseline in Expanded Disability Status Scale (EDSS) Score at the End of Primary Treatment Period
ParticipantsRavulizumabPlacebo (ECU-NMO-301)
No clinically important worsening5236
Clinically important worsening611
SecondaryNumber 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

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.

Time frame:
Baseline up to 2.25 years (end of the Primary Treatment Period)
Reported as:
Count of participants · Participants
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
ParticipantsRavulizumabPlacebo (ECU-NMO-301)
Any TEAEs5345
TESAEs826
TEAEs Leading to Study Drug Discontinuation12
SecondarySerum Ravulizumab Concentration
Time frame:
Predose and end of infusion (EOI) at Week 26
Reported as:
Mean · micrograms (µg)/milliliter (mL)
Serum Ravulizumab Concentration
micrograms (µg)/milliliter (mL)Ravulizumab
Week 26: Predose760.3 ± 202.75
Week 26: EOI1836.4 ± 355.39
SecondaryChange From Baseline in Serum Free C5 Concentration at Week 26
Time frame:
Baseline, Week 26 (Predose and EOI)
Reported as:
Mean · µg/mL
Change From Baseline in Serum Free C5 Concentration at Week 26
µg/mLRavulizumab
Change at Week 26: Predose-119.02 ± 42.857
Change at Week 26: EOI-119.32 ± 42.512
SecondaryNumber of Participants With Anti-drug Antibodies (ADAs) During the Primary Treatment Period
Time frame:
Baseline, Weeks 26, 50, 82, and 106
Reported as:
Count of participants · Participants
Number of Participants With Anti-drug Antibodies (ADAs) During the Primary Treatment Period
ParticipantsRavulizumab
Baseline — Positive5
Baseline — Negative53
Week 26 — Positive1
Week 26 — Negative54
Week 50 — Positive0
Week 50 — Negative52
Week 82 — Positive0
Week 82 — Negative15
Week 106 — Positive0
Week 106 — Negative1

Adverse events

Collected over Up to approximately 4.9 years.. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Primary Treatment Period: Placebo0/47 (0%)26/47 (55.3%)43/47 (91.5%)
Primary Treatment Period: Ravulizumab0/58 (0%)8/58 (13.8%)53/58 (91.4%)
Long-Term Extension: Ravulizumab1/56 (1.8%)9/56 (16.1%)49/56 (87.5%)
Most frequent serious events
Showing 10 of 42
Most frequent serious events
EventPrimary Treatment Period: PlaceboPrimary Treatment Period: RavulizumabLong-Term Extension: Ravulizumab
Neuromyelitis optica spectrum disorderNervous system disorders16/470/580/56
Ankle fractureInjury, poisoning and procedural complications0/470/582/56
PancytopeniaBlood and lymphatic system disorders1/470/580/56
Myocardial ischaemiaCardiac disorders1/470/580/56
Abdominal painGastrointestinal disorders1/470/580/56
PancreatitisGastrointestinal disorders1/470/580/56
Cholecystitis acuteHepatobiliary disorders1/470/580/56
PneumoniaInfections and infestations1/471/581/56
BronchitisInfections and infestations1/470/580/56
Gastroenteritis viralInfections and infestations1/470/580/56
Most frequent other events
Showing 10 of 394
Most frequent other events
EventPrimary Treatment Period: PlaceboPrimary Treatment Period: RavulizumabLong-Term Extension: Ravulizumab
COVID-19Infections and infestations0/4714/5816/56
HeadacheNervous system disorders10/4715/585/56
NauseaGastrointestinal disorders12/472/583/56
Pain in extremityMusculoskeletal and connective tissue disorders10/472/582/56
Urinary tract infectionInfections and infestations9/476/586/56
VomitingGastrointestinal disorders8/474/581/56
NasopharyngitisInfections and infestations8/473/584/56
DiarrhoeaGastrointestinal disorders6/473/585/56
Upper respiratory tract infectionInfections and infestations6/475/586/56
Back painMusculoskeletal and connective tissue disorders6/477/582/56

Baseline characteristics

The safety set included all participants who received at least 1 dose of study drug (ravulizumab or placebo).

Age, Categorical
Age, Categorical(Participants)RavulizumabPlacebo (ECU-NMO-301)Total
<=18 years000
Between 18 and 65 years514495
>=65 years7310
Sex: Female, Male
Sex: Female, Male(Participants)RavulizumabPlacebo (ECU-NMO-301)Total
Female524294
Male6511
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)RavulizumabPlacebo (ECU-NMO-301)Total
Hispanic or Latino9312
Not Hispanic or Latino454186
Unknown or Not Reported437
Race (NIH/OMB)
Race (NIH/OMB)(Participants)RavulizumabPlacebo (ECU-NMO-301)Total
American Indian or Alaska Native000
Asian211536
Native Hawaiian or Other Pacific Islander000
Black or African American6814
White292453
More than one race000
Unknown or Not Reported202
07

Study locations

41 sites
  • Research Site
    Aurora, Colorado 80045, United States
  • Research Site
    Fort Collins, Colorado 80528, United States
  • Research Site
    Washington D.C., District of Columbia 20007, United States
  • Research Site
    Miami, Florida 33136, United States
  • Research Site
    Boston, Massachusetts 02114, United States
  • Research Site
    Rochester, Minnesota 55905, United States
  • Research Site
    Jackson, Mississippi 39216, United States
  • Research Site
    St Louis, Missouri 63110, United States
  • Research Site
    Cincinnati, Ohio 45219, United States
  • Research Site
    Houston, Texas 77030, United States
  • Research Site
    Camperdown, 2050, Australia
  • Research Site
    Fitzroy, 3065, Australia
  • Research Site
    Vienna, 1090, Austria
  • Research Site
    Burnaby, British Columbia V5G 2X6, Canada
  • Research Site
    Aarhus, 8200, Denmark
  • Research Site
    Strasbourg, 67098, France
  • Research Site
    Berlin, 10117, Germany
  • Research Site
    Leipzig, 04103, Germany
  • Research Site
    München, 81675, Germany
  • Research Site
    Cefalù, 90015, Italy
  • Research Site
    Gallarate, 21013, Italy
  • Research Site
    Naples, 80131, Italy
  • Research Site
    Roma, 00133, Italy
  • Research Site
    Rome, 00178, Italy
  • Research Site
    Chiba, 260-0877, Japan
  • Research Site
    Fukuoka, 812-8582, Japan
  • Research Site
    Kawagoe-shi, 350-8550, Japan
  • Research Site
    Niigata, 951-8585, Japan
  • Research Site
    Sendai, 980-8574, Japan
  • Research Site
    Sendai, 983-8512, Japan
  • Research Site
    Katowice, 40-571, Poland
  • Research Site
    Lódz, 90-324, Poland
  • Research Site
    Goyang-si, 10408, South Korea
  • Research Site
    Seoul, 02841, South Korea
  • Research Site
    Seoul, 03722, South Korea
  • Research Site
    Seoul, 06351, South Korea
  • Research Site
    Seoul, 143-729, South Korea
  • Research Site
    Barcelona, 08036, Spain
  • Research Site
    Madrid, 28040, Spain
  • Research Site
    Málaga, 29010, Spain
  • Research Site
    Oxford, OX3 9DU, United Kingdom
08

References and documents

Publications

  • Pittock SJ, Barnett MH, Bennett JL, Berthele A, de Seze J, Levy M, Nakashima I, Oreja-Guevara C, Palace J, Paul F, Pozzilli C, Pathak R, Allen K, Parks B, Kim HJ. Long-Term Ravulizumab Efficacy and Safety in AQP4 Antibody-Positive Neuromyelitis Optica Spectrum Disorder: Final CHAMPION-NMOSD Results. Neurol Neuroimmunol Neuroinflamm. 2026 Sep;13(5):e200609. doi: 10.1212/NXI.0000000000200609. Epub 2026 Jul 13. PubMed 42441931 ↗
  • Bennett JL, Bhattacharyya S, Zabeti A, Levy M, de Seze J, Taney MJ, Parks B, Allen K, Akpoji U, Pittock SJ. Safety and efficacy of ravulizumab in patients with NMOSD previously treated with rituximab: A post hoc analysis of the CHAMPION-NMOSD trial. Mult Scler. 2026 Apr;32(4):396-408. doi: 10.1177/13524585261425076. Epub 2026 Mar 4. PubMed 41782198 ↗
  • Ortiz S, Pittock SJ, Berthele A, Levy M, Nakashima I, Oreja-Guevara C, Allen K, Mashhoon Y, Parks B, Kim HJ. Immediate and sustained terminal complement inhibition with ravulizumab in patients with anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder. Front Neurol. 2024 Jan 31;15:1332890. doi: 10.3389/fneur.2024.1332890. eCollection 2024. PubMed 38356884 ↗

Related links

Study documents

  • Study protocol · Sep 1, 2021
  • Statistical analysis plan · Jul 25, 2022

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: Yes

09

Registry details

Key details

Study ID
NCT04201262
Lead sponsor
Alexion Pharmaceuticals, Inc.
Responsible party
Sponsor
First posted
Dec 17, 2019
Start date
Dec 9, 2019
Primary completion
Mar 15, 2022
Completion
Oct 31, 2024
Results posted
Aug 9, 2023
Last update
Oct 14, 2025

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
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