JRCT ID: jRCT2031220259
Registered date:09/08/2022
A Phase 2/3, Open-label, Historical-controlled, Single-arm, Multicenter Studyto Evaluate the Efficacy, Pharmacokinetics, Pharmacodynamics, and Safety ofRavulizumab in Children and Adolescents With Aquaporin-4 Antibody Positive(AQP4-Ab [+]) Neuromyelitis Optica Spectrum Disorder (NMOSD)
Basic Information
Recruitment status | Recruiting |
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Health condition(s) or Problem(s) studied | Neuromyelitis Optica Spectrum Disorder |
Date of first enrollment | 06/07/2021 |
Target sample size | 12 |
Countries of recruitment | US,Japan,Spain,Japan |
Study type | Interventional |
Intervention(s) | 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. |
Outcome(s)
Primary Outcome | - Change From Baseline in the Annualized Relapse Rate at Week 50 Time Frame Baseline, Week 50 - Time to First Adjudicated On-trial Relapse through Week 50 Time Frame Baseline through Week 0 |
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Secondary Outcome | - Change From Baseline in Expanded Disability Status Scale Score At Week 50 Time Frame: Baseline, Week 50 - Change From Baseline in Hauser Ambulation Index at Week 50 Time Frame: Baseline, Week 50 - Change From Baseline in Visual Acuity at Week 50 Time Frame: Baseline, Week 50 - Change From Baseline in Confrontational Visual Fields at Week 50 Time Frame: Baseline, Week 50 - Change From Baseline in Color Vision at Week 50 Time Frame: Baseline, Week 50 - Serum Ravulizumab Concentration Time Frame: Predose and postdose (at end of infusion) on Day 1, Weeks 2, 10, 18, 26, and 42, and predose on Week50 - Change from Baseline in Free Serum Complement Component 5 (C5) Concentration Over Time Through Week 50 Time Frame: Baseline through Week 50 |
Key inclusion & exclusion criteria
Age minimum | >= 2age old |
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Age maximum | < 18age old |
Gender | Both |
Include criteria | - Participants must be anti-AQP4 Ab-positive and have a diagnosis of NMOSD as defined by the 2015 international consensus diagnostic criteria. - Complement inhibitor treatment-naive participants must have had at least1 attack or relapse in the last 12 months prior to the Screening Period. - Expanded Disability Status Scale (EDSS) score 7 or less. - Eculizumab-experienced participants must be clinically stable per Investigator for 30 days and have been treated with eculizumab in StudyECU-NMO-303 for at least 90 days prior to screening with no missed doses within 2 months prior to Day 1 - Participants who enter the study receiving supportive IST(s) (eg, corticosteroid, azathioprine [AZA], mycophenolate mofetil [MMF], methotrexate [MTX], tacrolimus [TAC], cyclosporin [CsA], or cyclophosphamide [CYC]) for the prevention of relapse, either in combination or monotherapy, must be on a stable dosing regimen of adequate duration prior to Screening and remain on a stable dosing regimen during the Screening Period. - To reduce the risk of meningococcal infection (Neisseria meningitidis), all participants must be vaccinated against meningococcal infection. - Documented vaccination for Hib and S pneumoniae at least 14 days prior to Day 1 according to national/local guidelines for the applicable age group. |
Exclude criteria | - Use of rituximab within 6 months prior to Day 1. - 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. - Use of intravenous immunoglobulin (IVIg) or plasma exchange (PE) within 3 weeks prior to Screening. - 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. - Use of immunomodulatory therapies for multiple sclerosis within 3 months prior to Screening. |
Related Information
Primary Sponsor | Wuebbenhorst Hiroaki |
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Secondary Sponsor | |
Source(s) of Monetary Support | |
Secondary ID(s) | NCT05346354 |
Contact
Public contact | |
Name | Hiroaki Wuebbenhorst |
Address | Tamachi Station Tower N, 3-1-1 Shibaura, Minato-ku, Tokyo 1080023, Japan Tokyo Japan 108-0023 |
Telephone | +81-3-3457-9559 |
hiroaki.wuebbenhorst@alexion.com | |
Affiliation | Alexion Pharma Godo Kaisha |
Scientific contact | |
Name | Hiroaki Wuebbenhorst |
Address | Tamachi Station Tower N, 3-1-1 Shibaura, Minato-ku, Tokyo 1080023, Japan Tokyo Japan 108-0023 |
Telephone | +81-3-3457-9559 |
hiroaki.wuebbenhorst@alexion.com | |
Affiliation | Alexion Pharma Godo Kaisha |