What side effects are associated with this type of intervention?

Common treatments for Neuromyelitis Optica Spectrum Disorders (NMOSD) that inhibit complement protein C5, like Ravulizumab, include eculizumab. Known side effects of these treatments can include headache, upper respiratory tract infections, nasopharyngitis, nausea, diarrhea, back pain, and infusion-related reactions. Additionally, there is an increased risk of serious infections, particularly meningococcal infections, due to the inhibition of the complement system.

What prior FDA approvals exist?

The FDA has approved several treatments for Neuromyelitis Optica Spectrum Disorders (NMOSD). One notable approval is eculizumab (Soliris), which, like ravulizumab, is a complement inhibitor targeting the C5 protein to reduce inflammation and prevent relapses. Other FDA-approved treatments for NMOSD include inebilizumab (Uplizna), which targets CD19 on B cells, and satralizumab (Enspryng), which inhibits the interleukin-6 receptor. These treatments aim to reduce the frequency of attacks and manage symptoms in patients with NMOSD.

What other types of research exist?

Promising novel alternatives to Ravulizumab for NMOSD patients include Eculizumab, which also inhibits complement protein C5 and has shown efficacy in clinical trials. Additionally, Satralizumab, an interleukin-6 receptor inhibitor, and Inebilizumab, an anti-CD19 monoclonal antibody, are emerging treatments that have demonstrated positive results in reducing relapses and managing NMOSD.

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Monoclonal Antibodies

Ravulizumab for Neuromyelitis Optica Spectrum Disorders

Phase 2 & 3

Recruiting

Research Sponsored by Alexion Pharmaceuticals

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Complement inhibitor treatment-naïve participants must have had at least 1 attack or relapse in the last 12 months prior to the Screening Period

All participants must be vaccinated against meningococcal infection

Must not have

Use of rituximab within 6 months prior to Day 1

Use of intravenous immunoglobulin (IVIg) or plasma exchange (PE) within 3 weeks prior to Screening

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, week 50

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a medication called ravulizumab in children with Neuromyelitis Optica Spectrum Disorder (NMOSD). NMOSD is a serious condition where the immune system attacks the nervous system. Ravulizumab works by blocking certain proteins that cause inflammation and damage. The goal is to see if this treatment is safe and effective for these children.

Who is the study for?

This trial is for pediatric patients with Neuromyelitis Optica Spectrum Disorder (NMOSD) who are positive for anti-AQP4 antibodies. They should have an EDSS score ≤ 7 and be stable on current treatments, including eculizumab if applicable. Participants must be vaccinated against meningococcal infections and other specific diseases, and not have used certain immune therapies or participated in conflicting studies recently.

What is being tested?

The study tests the safety and effectiveness of a medication called Ravulizumab in children with NMOSD. It aims to see how well this drug works in preventing relapses of the disease compared to previous treatments they may have received.

What are the potential side effects?

While specific side effects for Ravulizumab in this trial aren't listed, similar drugs can cause reactions like headache, nausea, potential risk of infection due to immune system suppression, infusion-related reactions, and rarely meningitis.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have had at least one attack or relapse of my condition in the past year and have not received complement inhibitor treatment.

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I am vaccinated against meningococcal infection.

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My disability score is 7 or less.

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I have been stable on eculizumab for 30 days and took it for at least 90 days without missing doses in the last 2 months.

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I am positive for anti-AQP4 antibodies and have been diagnosed with NMOSD.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have not used rituximab in the last 6 months.

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I haven't had IVIg or plasma exchange in the last 3 weeks.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, week 50

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, week 50

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, week 50 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Change From Baseline in Expanded Disability Status Scale Score At Week 50

Change From Baseline in Hauser Ambulation Index at Week 50

Side effects data

From 2022 Phase 4 trial • 18 Patients • NCT04320602

33%

Fatigue

17%

Abdominal pain

17%

COVID-19

17%

SARS-CoV-2 test positive

17%

Cough

11%

Back pain

11%

Nasopharyngitis

11%

Non-cardiac chest pain

11%

Dyspnoea

Pain

Pain in extremity

Chest discomfort

Body temperature abnormal

Rash

Herpes zoster

Upper respiratory tract congestion

Arthralgia

Peripheral swelling

Influenza like illness

Pneumonia

Asthenia

Retinal haemorrhage

Headache

Upper respiratory tract infection

Chromaturia

Haemoglobinuria

Neurodermatitis

Pyrexia

Skin lesion

Nephrolithiasis

Urinary tract infection

Constipation

Extravascular haemolysis

Haemoglobin decreased

Oropharyngeal pain

Nail ridging

100%

80%

60%

40%

20%

Study treatment Arm

Ravulizumab

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: RavulizumabExperimental Treatment1 Intervention

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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Ravulizumab

2016

Completed Phase 4

~1090

0 / 7Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Ravulizumab, a treatment for Neuromyelitis Optica Spectrum Disorders (NMOSD), works by inhibiting complement protein C5, thereby reducing inflammation and preventing damage to the central nervous system. This mechanism is crucial for NMOSD patients as it helps to mitigate the autoimmune attack on the optic nerves and spinal cord, which are characteristic of the disorder. Other treatments, such as eculizumab, also target the complement system to achieve similar protective effects. By reducing the inflammatory response, these treatments aim to prevent relapses and long-term disability, significantly improving the quality of life for NMOSD patients.