Ravulizumab for Neuromyelitis Optica Spectrum Disorders

No longer recruiting at 109 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Alexion

Must not be taking: Rituximab, Mitoxantrone, IV immunoglobulin

Disqualifiers: Neisseria meningitidis, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the effectiveness and safety of ravulizumab (Ultomiris) for individuals with Neuromyelitis Optica Spectrum Disorders (NMOSD), a disease affecting the nerves in the eyes and spinal cord. Participants will receive ravulizumab through an IV to determine its impact on managing their condition. It suits those who test positive for anti-aquaporin-4 antibodies, have experienced at least one attack or relapse in the past year, and are managing NMOSD with other stable treatments. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants the opportunity to contribute to a potentially groundbreaking treatment.

Do I have to stop taking my current medications for the trial?

The trial requires that if you are on supportive immunosuppressive therapy, you must be on a stable dose before joining. You cannot have used rituximab or mitoxantrone in the last 3 months or IV immunoglobulin in the last 3 weeks before the trial.

Is there any evidence suggesting that ravulizumab is likely to be safe for humans?

Research shows that ravulizumab is generally well-tolerated by patients. Studies have found that people taking ravulizumab for neuromyelitis optica spectrum disorders (NMOSD) did not experience any relapses during clinical trials. This suggests that the treatment effectively prevents flare-ups of the disease. However, ravulizumab can increase the risk of serious infections, particularly from Neisseria meningitidis bacteria. Patients may need to take steps to avoid these infections. While ravulizumab has shown promising results in reducing relapses, potential participants should be aware of the infection risks and discuss these with their healthcare provider. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for NMOSD?

Ravulizumab is unique because it offers a new approach to treating Neuromyelitis Optica Spectrum Disorders (NMOSD). Unlike standard treatments, which often include immunosuppressive therapies like azathioprine or rituximab, ravulizumab works by specifically targeting and inhibiting the complement system, a part of the immune system involved in inflammation and nerve damage in NMOSD. This targeted action could potentially reduce relapses more effectively and with fewer side effects. Researchers are excited about ravulizumab's potential for longer-lasting benefits due to its extended dosing intervals, which means fewer infusions and more convenience for patients.

What evidence suggests that ravulizumab might be an effective treatment for NMOSD?

Research has shown that ravulizumab, the treatment under study in this trial, greatly reduces the chance of relapse in people with Neuromyelitis Optica Spectrum Disorders (NMOSD). One study found it lowered the risk of relapse by 98.6%. This is crucial because relapses in NMOSD can be very serious. Ravulizumab works by preventing a part of the immune system from attacking the body, which is particularly beneficial for patients with AQP4-Ab+ NMOSD. Both real-world experiences and clinical trials have demonstrated its effectiveness, with no relapses in patients over a long period. Overall, the evidence suggests that ravulizumab is a strong option for managing NMOSD. ^{4 6 7 8 9}

Are You a Good Fit for This Trial?

Adults with NMOSD who weigh at least 40 kg, have had an attack in the past year, and are on stable immunosuppressive therapy can join. They must use contraception and have a disability score ≤7. People with HIV, recent rituximab or mitoxantrone treatment, IV immunoglobulin use within 3 weeks, or history of meningitis infection cannot participate.

Inclusion Criteria

I have had at least one relapse in the past year.

Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies

I weigh at least 40 kilograms.

Exclusion Criteria

I am HIV positive.

I have been treated with a complement inhibitor before.

I have not used IV immunoglobulin in the last 3 weeks.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Primary Treatment

Participants receive open-label ravulizumab via intravenous infusion

26-50 weeks

Long-Term Extension

Participants may continue receiving ravulizumab for up to 2 years or until it is approved/available

Up to 2 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Ravulizumab

Trial Overview The trial is testing Ravulizumab's effectiveness and safety for treating adults with Neuromyelitis Optica Spectrum Disorders (NMOSD). Participants will receive this medication to see if it helps prevent relapses of NMOSD symptoms.

How Is the Trial Designed?

Group I: Ravulizumab Experimental Treatment 1 Intervention

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.

Ravulizumab is already approved in United States, European Union for the following indications:

Approved in United States as Ultomiris for:

• Paroxysmal nocturnal hemoglobinuria (PNH)
• Atypical hemolytic uremic syndrome (aHUS)

Approved in European Union as Ultomiris for:

• Paroxysmal nocturnal haemoglobinuria (PNH)

Who Is Running the Clinical Trial?

Alexion

Lead Sponsor

Trials

247

Recruited

38,600+

Marc Dunoyer

Alexion

Chief Executive Officer since 2021

PhD in Molecular Biology, University of Brussels

Christophe Hotermans

Alexion

Chief Medical Officer since 2021

MD, University of Leuven

Alexion Pharmaceuticals, Inc.

Lead Sponsor

Trials

267

Recruited

141,000+

Dr. Alberto R. Martinez

Alexion Pharmaceuticals, Inc.

Chief Medical Officer since 2010

MD from University of Sao Paulo

Marc Dunoyer

Alexion Pharmaceuticals, Inc.

Chief Executive Officer since 2021

PhD in Molecular Biology

Alexion Pharmaceuticals

Lead Sponsor

Trials

231

Recruited

36,700+

Marc Dunoyer

Alexion Pharmaceuticals

Chief Executive Officer since 2021

B.A. in Psychology from the University of New Hampshire

Christophe Hotermans

Alexion Pharmaceuticals

Chief Medical Officer since 2023

MD

Citations

1. ultomirishcp.com ultomirishcp.com/nmosd/efficacy

Efficacy | ULTOMIRIS® (ravulizumab-cwvz) | NMOSD ULTOMIRIS® reduced the risk of relapse by 98.6% (HR=0.014; 95% CI: 0.000‑0.103)2.

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11136926/

Network Meta-analysis of Ravulizumab and Alternative ... NMA results suggest ravulizumab, a C5 inhibitor, is likely to be more effective in preventing NMOSD relapse in patients with AQP4-Ab+ NMOSD.

3. neurology.org neurology.org/doi/10.1212/WNL.0000000000208776

NMO SPOTLIGHT Registry: Real-World Clinical Outcomes ... Conclusions: Consistent with clinical trial and real-world data previously reported on eculizumab and/or ravulizumab among patients with AQP4- ...

4. astrazeneca.com astrazeneca.com/content/astraz/media-centre/press-releases/2025/alexion-data-presented-at-ectrims-2025-reinforces-ultomiris-leadership-in-transforming-care-for-patients-with-nmosd.html

Alexion data presented at ECTRIMS 2025 reinforces ... Final results of CHAMPION-NMOSD Phase III trial long-term extension will show zero relapses in patients on Ultomiris through a median ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT04201262

NCT04201262 | An Efficacy and Safety Study of ... The primary purpose of this study is to evaluate the efficacy and safety of ravulizumab for the treatment of adult participants with NMOSD.

6. ultomirishcp.com ultomirishcp.com/nmosd/safety

Safety | ULTOMIRIS® (ravulizumab-cwvz) | NMOSD ULTOMIRIS, a complement inhibitor, increases the risk of serious infections caused by Neisseria meningitidis [see Warnings and Precautions (5.1)]

7. ultomiris.com ultomiris.com/nmosd

Official NMOSD Patient Site People taking ULTOMIRIS® had ZERO relapses during the clinical trial. ULTOMIRIS is an FDA-approved treatment that reduced the risk of relapse by 98.6% ...

8. va.gov va.gov/formularyadvisor/DOC_PDF/MON_Ravulizumab_ULTOMIRIS_for_NMOSD_Monograph_Addendum_Nov_2024.pdf

Ravulizumab-cwvz (ULTOMIRIS) for the Treatment of ... • Safety data from CHAMPION-NMOSD is summarized in Table 3 ... • Neuromyelitis Optica Spectrum Disorder (NMOSD) is a rare CNS inflammatory demyelinating.

9. ultomirishcp.com ultomirishcp.com/nmosd

Neuromyelitis optica spectrum disorder (NMOSD) - Ultomiris Zero relapses were seen in the CHAMPION-NMOSD study1*. ULTOMIRIS® significantly reduced the risk of relapse by 98.6% vs the placebo arm (HR 0.014; ...