NCT05346354

Efficacy and Safety Study of Ravulizumab IV in Pediatric Participants With NMOSD

A Phase 2/3, Open-label, Historical-controlled, Single-arm, Multicenter Study to Evaluate the Efficacy, Pharmacokinetics, Pharmacodynamics, and Safety of Ravulizumab in Children and Adolescents With Aquaporin-4 Antibody Positive (AQP4-Ab [+]) Neuromyelitis Optica Spectrum Disorder (NMOSD)

• Status: Recruiting
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: Alexion Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

The primary purpose of this study is to evaluate the safety and efficacy of ravulizumab in pediatric participants with Neuromyelitis Optica Spectrum Disorder (NMOSD).

Conditions

• Neuromyelitis Optica Spectrum Disorder

Interventions

Type	Name	Description
DRUG	Ravulizumab	Participants will receive a weight-based loading dose of ravulizumab on Day 1, followed by weight-based maintenance treatment with ravulizumab on Day 15 and every 8 weeks (q8w) after or once every 4 weeks (q4w) depending on weight. During the Extension Period, participants will continue to receive weight-based maintenance doses of ravulizumab IV on Day 351 and q8w or q4w, depending on weight.

Timeline

• Start date: 2022-06-23
• Primary completion: 2026-03-31
• Completion: 2029-04-02
• First posted: 2022-04-26
• Last updated: 2025-11-12

Locations

21 sites across 8 countries: United States, Canada, France, Germany, Italy, Japan, South Korea, Spain

Regulatory

• FDA-regulated drug study