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

NCT ID NCT05346354

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

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.

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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

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Washington D.C., District of Columbia, 20010, United States
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Miami, Florida, 33136, United States
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Boston, Massachusetts, 02114, United States
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Durham, North Carolina, 27705, United States
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Philadelphia, Pennsylvania, 19104, United States
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Edmonton, Alberta, T6G 1C9, Canada
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Toronto, Ontario, M5G 1X8, Canada
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Montreal, Quebec, H3T1C5, Canada
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Le Kremlin-Bicêtre, 94270, France
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Marseille, 13005, France
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Chieti, 66013, Italy
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Gallarate, 21013, Italy
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Roma, 00165, Italy
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Yokohama, 232-0024, Japan
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Goyang-si, 10408, South Korea
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Esplugues de Llobregat, 8950, Spain

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