Neuromyelitis Optica Spectrum DisorderPhase 2/3NCT05346354
Efficacy and Safety Study of Ravulizumab IV in Pediatric Participants With NMOSD
A Phase 2/3 study testing Ravulizumab in people with Neuromyelitis Optica Spectrum Disorder, run by Alexion Pharmaceuticals, Inc. at 16 sites in 7 countries.
Trial summary
The primary purpose of this study is to evaluate the safety and efficacy of ravulizumab in pediatric participants with Neuromyelitis Optica Spectrum Disorder (NMOSD). Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- 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
- 12 participants
- Where
- 16 sitesin 7 countries
- Study dates
- 23 Jun 2022to 30 Mar 2029
- Phase
- Phase 2/3
- Are there results?
- Not yet: the study is under way
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Participants must be anti-AQP4 Ab-positive and have a diagnosis of NMOSD as defined by the 2015 international consensus diagnostic criteria.
- Complement inhibitor treatment-naïve participants must have had at least 1 attack or relapse in the last 12 months prior to the Screening Period.
- Expanded Disability Status Scale (EDSS) score ≤ 7.
- Eculizumab-experienced participants must be clinically stable per Investigator for 30 days and have been treated with eculizumab in Study ECU-NMO-303 for at least 90 days prior to screening with no missed doses within 2 months prior to Day 1.
- Participants who enter the study receiving supportive IST(s) (eg, corticosteroid, azathioprine [AZA], mycophenolate mofetil [MMF], methotrexate [MTX], tacrolimus [TAC], cyclosporin [CsA], or cyclophosphamide [CYC]) for the prevention of relapse, either in combination or monotherapy, must be on a stable dosing regimen of adequate duration prior to Screening and remain on a stable dosing regimen during the Screening Period.
- To reduce the risk of meningococcal infection (Neisseria meningitidis), all participants must be vaccinated against meningococcal infection.
- Documented vaccination for Hib and S pneumoniae at least 14 days prior to Day 1 according to national/local guidelines for the applicable age group.
Who cannot join
- Use of rituximab within 3 months prior to screening.
- Currently treated with a biologic medications (other than eculizumab) that may affect immune system functioning, or has stopped treatment with a biologic medication that may affect immune system functioning, and 5 half lives of the medication have not elapsed by the time of the Screening Visit.
- Use of intravenous immunoglobulin (IVIg) or plasma exchange (PE) within 3 weeks prior to Screening.
- Participation in another investigational drug or investigational device study (other than Study ECU-NMO-303) within 5 half lives of that investigational product (if known) or 30 days before initiation of the first dose of study drug, whichever is longer.
- Use of immunomodulatory therapies for multiple sclerosis within 3 months prior to Screening.
Trial locations and contacts
16 sites in 7 countries.
16 sites in 7 countries
United States5 sites
Research Site
Boston, Massachusetts, 02114
Research Site
Durham, North Carolina, 27705
Research Site
Miami, Florida, 33136
Research Site
Philadelphia, Pennsylvania, 19104
Research Site
Washington D.C., District of Columbia, 20010
Canada3 sites
Research Site
Edmonton, Alberta, T6G 1C9
Research Site
Montreal, Quebec, H3T1C5
Research Site
Toronto, Ontario, M5G 1X8
Italy3 sites
Research Site
Chieti, 66013
Research Site
Gallarate, 21013
Research Site
Roma, 00165
France2 sites
Research Site
Le Kremlin-Bicêtre, 94270
Research Site
Marseille, 13005
About this study
Official title: A Phase 2/3, Open-label, Historical-controlled, Single-arm, Multicenter Study to Evaluate the Efficacy, Pharmacokinetics, Pharmacodynamics, and Safety of Ravulizumab in Children and Adolescents With Aquaporin-4 Antibody Positive (AQP4-Ab [+]) Neuromyelitis Optica Spectrum Disorder (NMOSD)
The primary purpose of this study is to evaluate the safety and efficacy of ravulizumab in pediatric participants with Neuromyelitis Optica Spectrum Disorder (NMOSD).
- Conditions
- Neuromyelitis Optica Spectrum Disorder
- Being tested
- Ravulizumab
- Keywords
- Neuromyelitis Optica Spectrum Disorder, Ravulizumab, ALXN1210, CNS Autoimmune Disorders, NMO, NMOSD
- Medical subject headings
- Neuromyelitis Optica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RavulizumabDrug
Also called ALXN1210, Ultomiris
Participants will receive a weight-based loading dose of ravulizumab on Day 1, followed by weight-based maintenance treatment with ravulizumab on Day 15 and every 8 weeks (q8w) after or once every 4 weeks (q4w) depending on weight.
During the Extension Period, participants will continue to receive weight-based maintenance doses of ravulizumab IV on Day 351 and q8w or q4w, depending on weight.
| Study group | Role | What they receive |
|---|---|---|
| Ravulizumab | Experimental | During the Primary Treatment Period, all participants will receive weight-based dosing of ravulizumab IV for a total of 50 weeks of treatment.
During the Extension Period, participants will continue to receive weight-based dosing of ravulizumab IV for up to 104 weeks. Receives: Drug: Ravulizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in the Annualized Relapse Rate at Week 50
Measured: Baseline, Week 50
Time to First Adjudicated On-trial Relapse through Week 50
Measured: Baseline through Week 50
Secondary outcomes (7)
Change From Baseline in Expanded Disability Status Scale Score At Week 50
The score ranges are 0 to 10, higher score indicates worse outcome.
Measured: Baseline, Week 50
Change From Baseline in Hauser Ambulation Index at Week 50
The score ranges are 0 to 9, higher score indicates worse outcome.
Measured: Baseline, Week 50
Change From Baseline in Visual Acuity at Week 50
Measured: Baseline, Week 50
Change From Baseline in Confrontational Visual Fields at Week 50
Measured: Baseline, Week 50
Change From Baseline in Color Vision at Week 50
Measured: Baseline, Week 50
Serum Ravulizumab Concentration
Measured: Predose and postdose (at end of infusion) on Day 1, Weeks 2, 10, 18, 26, and 42, and predose on Week 50
Change from Baseline in Free Serum Complement Component 5 (C5) Concentration Over Time Through Week 50
Measured: Baseline through Week 50
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Apr 2022
- Study start23 Jun 2022
- Primary completion22 Jun 2026 (estimated)
- Study completion30 Mar 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuromyelitis Optica Spectrum DisorderStudies 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 2/3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 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
- NCT05346354
- Sponsor's protocol ID
- ALXN1210-NMO-317
- Status last verified
- Jun 2026
- First submitted
- 20 Apr 2022
- Last updated
- 24 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.
Study at a glance
From the registry record, last updated by the sponsor on 24 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2/3, Open-label, Historical-controlled, Single-arm, Multicenter Study to Evaluate the Efficacy, Pharmacokinetics, Pharmacodynamics, and Safety of Ravulizumab in Children and Adolescents With Aquaporin-4 Antibody Positive (AQP4-Ab [+]) Neuromyelitis Optica Spectrum Disorder (NMOSD)
- ClinicalTrials.gov ID
- NCT05346354
- Acronym / sponsor ID
- ALXN1210-NMO-317
- Conditions
- Neuromyelitis Optica Spectrum Disorder
- Keywords
- Neuromyelitis Optica Spectrum Disorder, Ravulizumab, ALXN1210, CNS Autoimmune Disorders, NMO, NMOSD
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 23 Jun 2022 (actual)
- Primary completion
- 22 Jun 2026 (estimated)
- Study completion
- 30 Mar 2029 (estimated)
- First posted
- 26 Apr 2022 (actual)
- Results
- Not posted in the source record
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 safety and efficacy of ravulizumab in pediatric participants with Neuromyelitis Optica Spectrum Disorder (NMOSD).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Ravulizumab | Experimental | Drug: RavulizumabDuring the Primary Treatment Period, all participants will receive weight-based dosing of ravulizumab IV for a total of 50 weeks of treatment. During the Extension Period, participants will continue to receive weight-based dosing of ravulizumab IV for up to 104 weeks. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Participants must be anti-AQP4 Ab-positive and have a diagnosis of NMOSD as defined by the 2015 international consensus diagnostic criteria.
- Complement inhibitor treatment-naïve participants must have had at least 1 attack or relapse in the last 12 months prior to the Screening Period.
- Expanded Disability Status Scale (EDSS) score ≤ 7.
- Eculizumab-experienced participants must be clinically stable per Investigator for 30 days and have been treated with eculizumab in Study ECU-NMO-303 for at least 90 days prior to screening with no missed doses within 2 months prior to Day 1.
- Participants who enter the study receiving supportive IST(s) (eg, corticosteroid, azathioprine [AZA], mycophenolate mofetil [MMF], methotrexate [MTX], tacrolimus [TAC], cyclosporin [CsA], or cyclophosphamide [CYC]) for the prevention of relapse, either in combination or monotherapy, must be on a stable dosing regimen of adequate duration prior to Screening and remain on a stable dosing regimen during the Screening Period.
Key exclusion criteria (5 listed)
- Use of rituximab within 3 months prior to screening.
- Currently treated with a biologic medications (other than eculizumab) that may affect immune system functioning, or has stopped treatment with a biologic medication that may affect immune system functioning, and 5 half lives of the medication have not elapsed by the time of the Screening Visit.
- Use of intravenous immunoglobulin (IVIg) or plasma exchange (PE) within 3 weeks prior to Screening.
- Participation in another investigational drug or investigational device study (other than Study ECU-NMO-303) within 5 half lives of that investigational product (if known) or 30 days before initiation of the first dose of study drug, whichever is longer.
- Use of immunomodulatory therapies for multiple sclerosis within 3 months 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 on Day 1, followed by weight-based maintenance treatment with ravulizumab on Day 15 and every 8 weeks (q8w) after or once every 4 weeks (q4w) depending on weight. During the Extension Period, participants will continue to receive weight-based maintenance doses of ravulizumab IV on Day 351 and q8w or q4w, depending on weight. | Drug | Ravulizumab |
Primary outcome
Change From Baseline in the Annualized Relapse Rate at Week 50
Time frame: Baseline, Week 50
Time to First Adjudicated On-trial Relapse through Week 50
Time frame: Baseline through Week 50
7 secondary outcomes
Change From Baseline in Expanded Disability Status Scale Score At Week 50
Time frame: Baseline, Week 50
Change From Baseline in Hauser Ambulation Index at Week 50
Time frame: Baseline, Week 50
Change From Baseline in Visual Acuity at Week 50
Time frame: Baseline, Week 50
Change From Baseline in Confrontational Visual Fields at Week 50
Time frame: Baseline, Week 50
Change From Baseline in Color Vision at Week 50
Time frame: Baseline, Week 50
Serum Ravulizumab Concentration
Time frame: Predose and postdose (at end of infusion) on Day 1, Weeks 2, 10, 18, 26, and 42, and predose on Week 50
Change from Baseline in Free Serum Complement Component 5 (C5) Concentration Over Time Through Week 50
Time frame: Baseline through Week 50
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jun 2026.
- Individual participant dataSharing planned: Yes. Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.
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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.
16 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Ravulizumab in people with Neuromyelitis Optica Spectrum Disorder, run by Alexion Pharmaceuticals, Inc. at 16 sites in 7 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 7 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: under way, not looking for participants, as of the sponsor's last update on 24 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in United States, Canada, Italy, France, Japan and 2 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 combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
This study is still under way. Results are not available in the source record yet.
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?
Change From Baseline in the Annualized Relapse Rate at Week 50 (time frame: Baseline, Week 50). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 24 Jun 2026.