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
Conditions
Sponsors
Alexion Pharmaceuticals, Inc.
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 | — |
Secondary
| Measure | Time frame |
|---|---|
| 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;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. | — |
Countries
Canada, France, Germany, Italy, Japan, Korea, Republic of, Spain, United States
Contacts
Public ContactEuropean Clinical Trial Information
Alexion Europe SAS
Outcome results
None listed