New drug shows promise in rare nerve disease trial

NCT ID NCT04201262

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

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This Phase 3 trial tested the drug ravulizumab (Ultomiris) in 58 adults with neuromyelitis optica spectrum disorder (NMOSD), a rare condition where the immune system attacks the optic nerves and spinal cord. The goal was to see if the drug could prevent relapses. Participants received intravenous infusions every 8 weeks. The study is complete, and results will show whether ravulizumab reduces relapse rates compared to an external 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)

What this could lead to

If successful, this could provide a new treatment option to reduce relapses in people with NMOSD, a rare autoimmune disease that attacks the nervous system.

What could go wrong

This is a completed Phase 3 trial, but results are not yet published. The study used an external placebo comparison, which may be less reliable than a randomized controlled design. Risks include infusion reactions and increased infection risk.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

acute transverse myelitis demyelinating disease neuromyelitis optica optic neuritis Recurrence

As listed by the trial registrant

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

NEUROMYELITIS OPTICA NEUROMYELITIS OPTICA SPECTRUM DISORDER

Contacts and locations

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Locations

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Aurora, Colorado, 80045, United States
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Fort Collins, Colorado, 80528, United States
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Washington D.C., District of Columbia, 20007, United States
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Miami, Florida, 33136, United States
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Boston, Massachusetts, 02114, United States
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Rochester, Minnesota, 55905, United States
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Jackson, Mississippi, 39216, United States
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St Louis, Missouri, 63110, United States
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Cincinnati, Ohio, 45219, United States
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Houston, Texas, 77030, United States
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Camperdown, 2050, Australia
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Fitzroy, 3065, Australia
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Vienna, 1090, Austria
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Burnaby, British Columbia, V5G 2X6, Canada
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Aarhus, 8200, Denmark
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Strasbourg, 67098, France
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Berlin, 10117, Germany
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Leipzig, 04103, Germany
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München, 81675, Germany
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Cefalù, 90015, Italy
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Gallarate, 21013, Italy
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Naples, 80131, Italy
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Roma, 00133, Italy
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Rome, 00178, Italy
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Chiba, 260-0877, Japan
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Fukuoka, 812-8582, Japan
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Kawagoe-shi, 350-8550, Japan
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Niigata, 951-8585, Japan
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Sendai, 980-8574, Japan
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Sendai, 983-8512, Japan
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Katowice, 40-571, Poland
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Lódz, 90-324, Poland
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Goyang-si, 10408, South Korea
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Seoul, 02841, South Korea
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Seoul, 03722, South Korea
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Seoul, 06351, South Korea
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Seoul, 143-729, South Korea
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Barcelona, 08036, Spain
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Madrid, 28040, Spain
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Málaga, 29010, Spain
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Oxford, OX3 9DU, United Kingdom