Clinical study testing the efficacy and safety of Ravulizumab in pediatric patients 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) - A Phase 2/3 Efficacy and Safety Study of Ravulizumab in Pediatric Patients with NMOSD

Status

Active, not recruiting

Phases

Phase 2Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2021-006075-42-FR

Enrollment

Registered

2022-06-09

Start date

2022-08-01

Completion date

Unknown

Last updated

2024-10-14

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) MedDRA version: 21.1 Level: PT Classification code 10077875 Term: Neuromyelitis optica spectrum disorder System Organ Class: 10029205 - Nervous system disorders

Interventions

Trade Name: ULTOMIRIS® Product Name: Ravulizumab Product Code: ALXN1210 Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: Ravulizumab CAS Number: 1803171-55-2 Current Spo

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Participant must be = 2 to =65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: 1. Known to be human immunodeficiency virus (HIV) positive 2. History of N meningitidis infection 3. Active systemic bacterial, viral, or fungal infection within 14 days prior to Day 1. 4. Hypersensitivity to ravulizumab, murine proteins or to one of the excipients of ravulizumab. 5. Use of rituximab within 6 months prior to Day 1. 6. 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 7. Use of intravenous immunoglobulin (IVIg) or plasma exchange (PE) within 3 weeks prior to Screening. 8. 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. 9. Use of immunomodulatory therapies for multiple sclerosis within 3 months prior to Screening. Please see the detailed list of exclusion criteria in the protocol.

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate the efficacy of ravulizumab in pediatric participants with NMOSD;Secondary Objective: - To evaluate the effect of ravulizumab on disease-related disability in pediatric participants with NMOSD - To evaluate the effect of ravulizumab on neurologic function in pediatric participants with NMOSD - To characterize the PK of treatment with ravulizumab in pediatric participants with NMOSD - To characterize the PD of treatment with ravulizumab in pediatric participants with NMOSD - To assess quality of life based on patient-reported outcomes in pediatric participants with NMOSD based on treatment with Ravulizumab - To evaluate safety of ravulizumab in pediatric participants with NMOSD - To assess immunogenicity to ravulizumab in pediatric participants with NMOSD - Descriptive comparison of ravulizumab data to historical data from a NMOSD observational study (NCT 03766437) to contextualize efficacy data from this study. - To characterize the long-term effect of ravulizumab on efficacy, safety, PK, PD, and immunogenicity;Primary end point(s): - The change from baseline in the annualized relapse rate (ARR) - Time to First Adjudicated On-trial Relapse (TFR);Timepoint(s) of evaluation of this end point: Primary treatment period: On-Trial Relapses will be monitored throughout the study and at 50 weeks	—

Measure

Time frame

Main Objective: To evaluate the efficacy of ravulizumab in pediatric participants with NMOSD;Secondary Objective: - To evaluate the effect of ravulizumab on disease-related disability in pediatric participants with NMOSD - To evaluate the effect of ravulizumab on neurologic function in pediatric participants with NMOSD - To characterize the PK of treatment with ravulizumab in pediatric participants with NMOSD - To characterize the PD of treatment with ravulizumab in pediatric participants with NMOSD - To assess quality of life based on patient-reported outcomes in pediatric participants with NMOSD based on treatment with Ravulizumab - To evaluate safety of ravulizumab in pediatric participants with NMOSD - To assess immunogenicity to ravulizumab in pediatric participants with NMOSD - Descriptive comparison of ravulizumab data to historical data from a NMOSD observational study (NCT 03766437) to contextualize efficacy data from this study. - To characterize the long-term effect of ravulizumab on efficacy, safety, PK, PD, and immunogenicity;Primary end point(s): - The change from baseline in the annualized relapse rate (ARR) - Time to First Adjudicated On-trial Relapse (TFR);Timepoint(s) of evaluation of this end point: Primary treatment period: On-Trial Relapses will be monitored throughout the study and at 50 weeks

—

Secondary

Measure	Time frame
Secondary end point(s): Primary treatment period: 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 - 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.;Timepoint(s) of evaluation of this end point: Primary treatment period: throughout the primary treatment period and at 50 weeks Extension treatment period: 104 weeks	—

Measure

Time frame

Secondary end point(s): Primary treatment period: 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 - 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.;Timepoint(s) of evaluation of this end point: Primary treatment period: throughout the primary treatment period and at 50 weeks Extension treatment period: 104 weeks

—

Countries

Canada, France, Germany, Italy, Japan, Korea, Republic of, Spain, United States

Contacts

Public ContactEuropean Clinical Trial Information

Alexion Europe SAS

clinicaltrials.eu@alexion.com+33147 10 06 15

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026