Neuromyelitis OpticaPhase 3NCT04201262
An Efficacy and Safety Study of Ravulizumab in Adult Participants With NMOSD
A Phase 3 study testing Ravulizumab in people with Neuromyelitis Optica and Neuromyelitis Optica Spectrum Disorder, run by Alexion Pharmaceuticals, Inc. at 41 sites in 13 countries.
Trial summary
The primary purpose of this study is to evaluate the efficacy and safety of ravulizumab for the treatment of adult participants with NMOSD. Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Neuromyelitis Optica, Neuromyelitis Optica Spectrum Disorder
- What is being tested?
- Ravulizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 58 participants
- Where
- 41 sitesin 13 countries
- Study dates
- 9 Dec 2019to 31 Oct 2024
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Anti-aquaporin-4 antibody-positive and a diagnosis of NMOSD as defined by the 2015 international consensus diagnostic criteria.
- At least 1 attack or relapse in the last 12 months prior to the Screening Period.
- Expanded Disability Status Scale score ≤7.
- Participants who enter the study receiving supportive immunosuppressive therapy must be on a stable dosing regimen of adequate duration prior to Screening.
- Body weight ≥40 kilograms.
- Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
Who cannot join
- History of Neisseria meningitidis infection.
- Human immunodeficiency virus (HIV) infection (evidenced by HIV-1 or HIV-2 antibody titer).
- Previously or currently treated with a complement inhibitor.
- Use of rituximab or mitoxantrone within 3 months prior to Screening.
- Use of IV immunoglobulin within 3 weeks prior to Screening.
Trial locations and contacts
41 sites in 13 countries.
41 sites in 13 countries
United States10 sites
Research Site
Aurora, Colorado, 80045
Research Site
Boston, Massachusetts, 02114
Research Site
Cincinnati, Ohio, 45219
Research Site
Fort Collins, Colorado, 80528
Research Site
Houston, Texas, 77030
Research Site
Jackson, Mississippi, 39216
Research Site
Miami, Florida, 33136
Research Site
Rochester, Minnesota, 55905
Research Site
St Louis, Missouri, 63110
Research Site
Washington D.C., District of Columbia, 20007
Japan6 sites
Research Site
Chiba, 260-0877
Research Site
Fukuoka, 812-8582
Research Site
Kawagoe-shi, 350-8550
Research Site
Niigata, 951-8585
Research Site
Sendai, 980-8574
Research Site
Sendai, 983-8512
Italy5 sites
Research Site
Cefalù, 90015
Research Site
Gallarate, 21013
Research Site
Naples, 80131
Research Site
Roma, 00133
Research Site
Rome, 00178
South Korea5 sites
Research Site
Goyang-si, 10408
Research Site
Seoul, 02841
Research Site
Seoul, 03722
Research Site
Seoul, 06351
Research Site
Seoul, 143-729
Germany3 sites
Research Site
Berlin, 10117
Research Site
Leipzig, 04103
Research Site
München, 81675
Spain3 sites
Research Site
Barcelona, 08036
Research Site
Madrid, 28040
Research Site
Málaga, 29010
About this study
Official title: A Phase 3, External Placebo-Controlled, Open-Label, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Patients With Neuromyelitis Optica Spectrum Disorder (NMOSD)
The primary purpose of this study is to evaluate the efficacy and safety of ravulizumab for the treatment of adult participants with NMOSD.
- Being tested
- Ravulizumab
- 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
- Medical subject headings
- Neuromyelitis Optica, Myelitis, Transverse, Optic Neuritis, Recurrence, Demyelinating Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RavulizumabBiological
Also called ALXN1210, Ultomiris
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.
| Study group | Role | What they receive |
|---|---|---|
| Ravulizumab | Experimental | 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. Receives: Biological: Ravulizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline up to 2.25 years (end of the Primary Treatment Period)
Secondary outcomes (9)
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.
Measured: Baseline up to 2.25 years (end of the Primary Treatment Period)
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.
Measured: Baseline up to 2.25 years (end of the Primary Treatment Period)
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.
Measured: Baseline, up to 2.25 years (end of the Primary Treatment Period)
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.
Measured: Baseline, up to 2.25 years (end of the Primary Treatment Period)
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.
Measured: Baseline up to 2.25 years (end of the Primary Treatment Period)
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.
Measured: Baseline up to 2.25 years (end of the Primary Treatment Period)
Serum Ravulizumab Concentration
Measured: Predose and end of infusion (EOI) at Week 26
Change From Baseline in Serum Free C5 Concentration at Week 26
Measured: Baseline, Week 26 (Predose and EOI)
Number of Participants With Anti-drug Antibodies (ADAs) During the Primary Treatment Period
Measured: Baseline, Weeks 26, 50, 82, and 106
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Dec 2019
- Study start9 Dec 2019
- Primary completion15 Mar 2022
- Study completion31 Oct 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Neuromyelitis OpticaStudies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Model description
- External Placebo-Controlled, Open-Label Design
- Masking
- None (open label)
- Enrolment
- 58 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04201262
- Sponsor's protocol ID
- ALXN1210-NMO-307
- Other IDs
- CHAMPION-NMO-307, 2019-003352-37, 2019
- Status last verified
- Sep 2025
- First submitted
- 11 Dec 2019
- Last updated
- 14 Oct 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes
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 42441931Bennett 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 41782198Ortiz 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
Study at a glance
From the registry record, last updated by the sponsor on 14 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, External Placebo-Controlled, Open-Label, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Patients With Neuromyelitis Optica Spectrum Disorder (NMOSD)
- ClinicalTrials.gov ID
- NCT04201262
- Acronym / sponsor ID
- ALXN1210-NMO-307
- Keywords
- Neuromyelitis Optica, Neuromyelitis Optica Spectrum Disorder, Devic's Disease, Transverse Myelitis, Optic Neuritis, Relapse, Ravulizumab, Ultomiris
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 58 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 9 Dec 2019 (actual)
- Primary completion
- 15 Mar 2022 (actual)
- Study completion
- 31 Oct 2024 (actual)
- First posted
- 17 Dec 2019 (actual)
- Results
- Posted 9 Aug 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary purpose of this study is to evaluate the efficacy and safety of ravulizumab for the treatment of adult participants with NMOSD.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- External Placebo-Controlled, Open-Label Design
| Arm | Type | Receives |
|---|---|---|
| Ravulizumab | Experimental | Biological: RavulizumabDuring 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. |
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Anti-aquaporin-4 antibody-positive and a diagnosis of NMOSD as defined by the 2015 international consensus diagnostic criteria.
- At least 1 attack or relapse in the last 12 months prior to the Screening Period.
- Expanded Disability Status Scale score ≤7.
- Participants who enter the study receiving supportive immunosuppressive therapy must be on a stable dosing regimen of adequate duration prior to Screening.
- Body weight ≥40 kilograms.
Key exclusion criteria (5 listed)
- History of Neisseria meningitidis infection.
- Human immunodeficiency virus (HIV) infection (evidenced by HIV-1 or HIV-2 antibody titer).
- Previously or currently treated with a complement inhibitor.
- Use of rituximab or mitoxantrone within 3 months prior to Screening.
- Use of IV immunoglobulin within 3 weeks prior to Screening.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RavulizumabAlso: ALXN1210, UltomirisParticipants 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. | Biological | Ravulizumab |
Primary outcome
Number of Participants With an Adjudicated On-trial Relapse in the Primary Treatment Period
Time frame: 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.
9 secondary outcomes
Adjudicated On-trial Annualized Relapse Rate (ARR) in the Primary Treatment Period
Time frame: Baseline up to 2.25 years (end of the Primary Treatment Period)
Number of Participants With Clinically Important Change From Baseline in Hauser Ambulation Index (HAI) Score at the End of Primary Treatment Period
Time frame: Baseline up to 2.25 years (end of the Primary Treatment Period)
Change From Baseline in European Quality of Life Health 5-dimension Questionnaire (EQ-5D) Index Score at the End of Primary Treatment Period
Time frame: Baseline, up to 2.25 years (end of the Primary Treatment Period)
Change From Baseline in EQ-5D Visual Analog Scale (VAS) Score at the End of Primary Treatment Period
Time frame: Baseline, up to 2.25 years (end of the Primary Treatment Period)
Number of Participants With Clinically Important Worsening From Baseline in Expanded Disability Status Scale (EDSS) Score at the End of Primary Treatment Period
Time frame: Baseline up to 2.25 years (end of the Primary Treatment Period)
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
Time frame: Baseline up to 2.25 years (end of the Primary Treatment Period)
Serum Ravulizumab Concentration
Time frame: Predose and end of infusion (EOI) at Week 26
Change From Baseline in Serum Free C5 Concentration at Week 26
Time frame: Baseline, Week 26 (Predose and EOI)
Number of Participants With Anti-drug Antibodies (ADAs) During the Primary Treatment Period
Time frame: Baseline, Weeks 26, 50, 82, and 106
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Oct 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 42441931Pittock 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.
- Derived from this study: PubMed 41782198Bennett 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.
- Derived from this study: PubMed 38356884Ortiz 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.
- Related InfoLink provided by the sponsor.
- Individual participant dataSharing planned: Yes
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
41 sites in 13 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Ravulizumab in people with Neuromyelitis Optica and Neuromyelitis Optica Spectrum Disorder, run by Alexion Pharmaceuticals, Inc. at 41 sites in 13 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 41 sites in United States, Japan, Italy, South Korea, Germany and 8 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 9 Aug 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With an Adjudicated On-trial Relapse in the Primary Treatment Period (time frame: Baseline up to 2.25 years (end of the Primary Treatment Period)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04201262, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Oct 2025.