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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
Phases
Phase 2Phase 3
Study type
Interventional
Source
EU CTIS
Registry ID
CTIS2023-508534-33-00
Acronym
ALXN1210-NMO-317
Enrollment
6
Registered
2024-03-27
Start date
2022-06-16
Completion date
Unknown
Last updated
2025-08-18

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Neuromyelitis Optica Spectrum Disorder (NMOSD)

Brief summary

- The change from baseline in the annualized relapse rate (ARR) - Time to First Adjudicated On-trial Relapse (TFR)

Detailed description

Efficacy: - Change from baseline in expanded disability status scale (EDSS) score - Change from baseline in Hauser Ambulation Index (HAI) - The change from baseline in visual acuity at the end of the Primary Treatment Period, Efficacy: - The change from baseline in confrontational visual fields at the end of the Primary Treatment Period - The change from baseline in color vision at the end of the Primary Treatment Period, PK/PD: - Serum ravulizumab concentrations through the end of the Primary Treatment Period - Absolute values, change from baseline, and percentage change from baseline for free serum C5 concentrations over time through the end of the Primary Treatment Period, Health-related QoL: - Change from baseline in PedsQL Scales at the end of the Primary Treatment Period, Safety: - Incidence of AEs and SAEs - Change from baseline in vital signs, physical growth (weight, height, and head circumference [participants ≤ 3 years of age only]), and laboratory parameters at scheduled visits Extension treatment period: The endpoints of the Primary Treatment Period will be evaluated during the Extension Period.

Interventions

Sponsors

Alexion Pharmaceuticals Inc.
Lead SponsorINDUSTRY

Eligibility

Sex/Gender
All
Age
0 Years to 17 Years

Design outcomes

Primary

MeasureTime frame
- The change from baseline in the annualized relapse rate (ARR) - Time to First Adjudicated On-trial Relapse (TFR)

Secondary

MeasureTime frame
Efficacy: - Change from baseline in expanded disability status scale (EDSS) score - Change from baseline in Hauser Ambulation Index (HAI) - The change from baseline in visual acuity at the end of the Primary Treatment Period, Efficacy: - The change from baseline in confrontational visual fields at the end of the Primary Treatment Period - The change from baseline in color vision at the end of the Primary Treatment Period, PK/PD: - Serum ravulizumab concentrations through the end of the Primary Treatment Period - Absolute values, change from baseline, and percentage change from baseline for free serum C5 concentrations over time through the end of the Primary Treatment Period, Health-related QoL: - Change from baseline in PedsQL Scales at the end of the Primary Treatment Period, Safety: - Incidence of AEs and SAEs - Change from baseline in vital signs, physical growth (weight, height, and head circumference [participants ≤ 3 years of age only]), and laboratory parameters at scheduled vi

Countries

France, Italy, Spain

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026