Actively Recruiting
Study of Ravulizumab IV in Children and Adolescents With Aquaporin-4 Antibody Positive Neuromyelitis Optica Spectrum Disorder
Led by Alexion Pharmaceuticals, Inc. · Updated on 2025-11-12
12
Participants Needed
21
Research Sites
156 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effectiveness of ravulizumab in children and adolescents diagnosed with Neuromyelitis Optica Spectrum Disorder (NMOSD) who test positive for aquaporin-4 antibodies. This study is a Phase 2/3, open-label, single-arm trial designed to collect data on how ravulizumab behaves in the body and its impact on this rare neurological condition. Participants will receive weight-based doses of ravulizumab through intravenous infusions. The Primary Treatment Period lasts 50 weeks, during which all participants are treated with ravulizumab. Following this, an Extension Period of up to 104 weeks allows participants to continue receiving the medication. Dosing schedules vary based on participant weight, with maintenance doses given every 8 or 4 weeks. Throughout the study, participants will undergo regular assessments including measurements of relapse rates, disability scores, walking ability, vision tests, and blood tests to monitor drug levels and immune activity. These evaluations occur at baseline and at scheduled intervals up to week 50 and beyond during the Extension Period. The study aims to carefully track safety and treatment effects over time, with total participation potentially lasting over two years.
CONDITIONS
Brief Title
Efficacy and Safety Study of Ravulizumab IV in Pediatric Participants With NMOSD
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants must be anti-AQP4 antibody positive with a diagnosis of NMOSD per 2015 international criteria.
- Complement inhibitor treatment-nafve participants must have had at least one attack or relapse in the last 12 months before screening.
- Expanded Disability Status Scale (EDSS) score must be 7 or less.
- Eculizumab-experienced participants must be clinically stable for 30 days and treated with eculizumab for at least 90 days prior to screening without missed doses in the last 2 months.
- Participants receiving supportive immunosuppressive therapy must be on a stable dosing regimen before and during screening.
- All participants must be vaccinated against meningococcal infection.
- Documented vaccination for Haemophilus influenzae type b and Streptococcus pneumoniae at least 14 days before Day 1 according to local guidelines.
You will not qualify if you...
- Use of rituximab within 3 months before screening.
- Current treatment with biologic medications other than eculizumab that affect the immune system, or insufficient time elapsed since stopping such treatments.
- Use of intravenous immunoglobulin or plasma exchange within 3 weeks before screening.
- Participation in another investigational drug or device study within 5 half-lives of that product or 30 days before starting study drug.
- Use of immunomodulatory therapies for multiple sclerosis within 3 months before screening.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - 50 weeks
Participants receive weight-based dosing of ravulizumab IV starting with a loading dose on Day 1, followed by maintenance doses on Day 15 and every 4 or 8 weeks depending on weight for a total of 50 weeks during the Primary Treatment Period.
Multiple visits for infusions including Day 1, Day 15, and every 4 or 8 weeks thereafter until Week 50
Duration - Up to 104 weeks
Participants who continue in the Extension Period will receive ongoing weight-based maintenance doses of ravulizumab IV for up to an additional 104 weeks.
Regular infusions every 4 or 8 weeks depending on weight during Extension Period
Trial Site Locations
Total: 21 locations
1
Research Site
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
2
Research Site
Miami, Florida, United States, 33136
Actively Recruiting
3
Research Site
Boston, Massachusetts, United States, 02114
Actively Recruiting
4
Research Site
St Louis, Missouri, United States, 63110
Withdrawn
5
Research Site
Durham, North Carolina, United States, 27705
Actively Recruiting
6
Research Site
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
7
Research Site
Edmonton, Alberta, Canada, T6G 1C9
Actively Recruiting
8
Research Site
Toronto, Ontario, Canada, M5G 1X8
Actively Recruiting
9
Research Site
Montreal, Quebec, Canada, H3T1C5
Actively Recruiting
10
Research Site
Le Kremlin-Bicêtre, France, 94270
Actively Recruiting
11
Research Site
Marseille, France, 13005
Actively Recruiting
12
Research Site
Montpellier, France, 34295
Actively Recruiting
13
Research Site
Bochum, Germany, 44791
Withdrawn
14
Research Site
Giessen, Germany, 35392
Withdrawn
15
Research Site
Catania, Italy, 95123
Withdrawn
16
Research Site
Chieti, Italy, 66013
Actively Recruiting
17
Research Site
Gallarate, Italy, 21013
Actively Recruiting
18
Research Site
Roma, Italy, 00165
Actively Recruiting
19
Research Site
Yokohama, Japan, 232-0024
Actively Recruiting
20
Research Site
Goyang-si, South Korea, 10408
Actively Recruiting
21
Research Site
Esplugues de Llobregat, Spain, 8950
Actively Recruiting
Research Team
A
Alexion Pharmaceuticals, Inc. (Sponsor)
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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