Efficacy and Safety Study of Ravulizumab IV in Pediatric Participants With NMOSD

• Register: ClinicalTrials.gov
• Last refreshed on: 28 April 2025
• Main ID: NCT05346354
• Date of registration: 20/04/2022
• Prospective Registration: Yes
• Primary sponsor: Alexion Pharmaceuticals, Inc.
• Public title: Efficacy and Safety Study of Ravulizumab IV in Pediatric Participants With NMOSD
• Scientific title: 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)
• Date of first enrolment: June 23, 2022
• Target sample size: 12
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT05346354
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2/Phase 3

Countries of recruitment

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

Contacts

• Name: Alexion Pharmaceuticals, Inc. (Sponsor)

Key inclusion & exclusion criteria

Inclusion 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-naïve participants must have had at least 1 attack or
relapse in the last 12 months prior to the Screening Period.

- Expanded Disability Status Scale (EDSS) score = 7.

- Eculizumab-experienced participants must be clinically stable per Investigator for
30 days and have been treated with eculizumab in Study ECU-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.

Exclusion Criteria:

- Use of rituximab within 3 months prior to screening.

- 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.


Age minimum: N/A
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Neuromyelitis Optica Spectrum Disorder

Intervention(s)

Drug: Ravulizumab

Primary Outcome(s)

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 50]

Secondary Outcome(s)

Change From Baseline in Expanded Disability Status Scale Score 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 Week 50]

Change From Baseline in Hauser Ambulation Index 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 Visual Acuity at Week 50 [Time Frame: Baseline, Week 50]

Change From Baseline in Color Vision at Week 50 [Time Frame: Baseline, Week 50]

Change from Baseline in Free Serum Complement Component 5 (C5) Concentration Over Time Through Week 50 [Time Frame: Baseline through Week 50]

Secondary ID(s)

ALXN1210-NMO-317

Source(s) of Monetary Support

Please refer to primary and secondary sponsors