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New drug hopes to stop relapses in kids with rare nerve disease

NCT ID NCT05346354

Disease control Sponsor: Alexion Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests ravulizumab, a drug that calms a part of the immune system, in 12 children with NMOSD – a rare disease where the immune system attacks the nerves in the eyes and spine. The goal is to see if the drug can reduce the number of relapses and prevent disability over about a year. All children receive the drug; there is no placebo group.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Ravulizumab (Ultomiris) – a drug that blocks part of the immune system to prevent attacks
What this could lead to
If it works, this could offer a treatment option for children with NMOSD to reduce relapses and slow disability.
What could go wrong
This is a small, early-phase trial with only 12 participants and no placebo group, so results may not apply broadly. The drug also increases risk of serious infections.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2/3

Runs two stages together: whether the treatment works, then large-scale confirmation.

Participants

12 people

The number who actually took part.

Started

Jun 2022

Expected to finish

Mar 2029

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * 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. Exclusion Criteria: * 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.

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Conditions

The condition(s) this trial relates to.

neuromyelitis optica

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Neuromyelitis optica spectrum disorder

Contacts and locations

Locations

  • Research Site

    Washington D.C., District of Columbia, 20010, United States

  • Research Site

    Miami, Florida, 33136, United States

  • Research Site

    Boston, Massachusetts, 02114, United States

  • Research Site

    Durham, North Carolina, 27705, United States

  • Research Site

    Philadelphia, Pennsylvania, 19104, United States

  • Research Site

    Edmonton, Alberta, T6G 1C9, Canada

  • Research Site

    Toronto, Ontario, M5G 1X8, Canada

  • Research Site

    Montreal, Quebec, H3T1C5, Canada

  • Research Site

    Le Kremlin-Bicêtre, 94270, France

  • Research Site

    Marseille, 13005, France

  • Research Site

    Chieti, 66013, Italy

  • Research Site

    Gallarate, 21013, Italy

  • Research Site

    Roma, 00165, Italy

  • Research Site

    Yokohama, 232-0024, Japan

  • Research Site

    Goyang-si, 10408, South Korea

  • Research Site

    Esplugues de Llobregat, 8950, Spain

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