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)

JRCT ID: jRCT2031220259

Registered date:09/08/2022

Basic Information

• Recruitment status: Recruiting
• 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

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
• 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
• Secondary ID(s): NCT05346354