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

Study identifier:ALXN1210-NMO-317

ClinicalTrials.gov identifier:NCT05346354

EudraCT identifier:N/A

CTIS identifier:N/A

Recruiting

Official 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)

Medical condition

Neuromyelitis Optica Spectrum Disorder

Phase

Phase 2/3

Healthy volunteers

Study drug

Ravulizumab

Sex

All

Estimated Enrollment

Study type

Interventional

Age

n/a - n/a

Date

Study Start Date: 23 Jun 2022

Estimated Primary Completion Date: 31 Mar 2026

Estimated Study Completion Date: 31 Mar 2028

Study design

Allocation: Non-randomized
Endpoint Classification: -
Intervention Model: Single Group Assignment
Masking: -
Primary Purpose: Treatment

Verification:

Verified 01 Aug 2024 by Alexion Pharmaceuticals, Inc.

Collaborators

Inclusion and 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 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.

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Arms	Assigned Interventions
Experimental: Ravulizumab During the Primary Treatment Period, all participants will receive weight-based dosing of ravulizumab IV for a total of 50 weeks of treatment. During the Extension Period, participants will continue to receive weight-based dosing of ravulizumab IV for up to 104 weeks.	-

Contacts

Contact

Alexion Pharmaceuticals, Inc. (Sponsor)

1-855-752-2356

[email protected]