An Efficacy and Safety Study of Ravulizumab in Adult Participants With NMOSD

Alexion Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: Neuromyelitis Optica; Neuromyelitis Optica Spectrum Disorder

Treatments: Biological: Ravulizumab

Study type: Interventional

Funder types: Industry

Identifiers

NCT04201262

2019-003352-37 (EudraCT Number)

CHAMPION-NMO-307 (Other Identifier)

ALXN1210-NMO-307

Details and patient eligibility

About

The primary purpose of this study is to evaluate the efficacy and safety of ravulizumab for the treatment of adult participants with NMOSD.

Enrollment: 58 patients

Sex: All

Ages: 18 to 100 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Anti-aquaporin-4 antibody-positive and a diagnosis of NMOSD as defined by the 2015 international consensus diagnostic criteria.
2. At least 1 attack or relapse in the last 12 months prior to the Screening Period.
3. Expanded Disability Status Scale score ≤7.
4. Participants who enter the study receiving supportive immunosuppressive therapy must be on a stable dosing regimen of adequate duration prior to Screening.
5. Body weight ≥40 kilograms.
6. Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.

Exclusion criteria

1. History of Neisseria meningitidis infection.
2. Human immunodeficiency virus (HIV) infection (evidenced by HIV-1 or HIV-2 antibody titer).
3. Previously or currently treated with a complement inhibitor.
4. Use of rituximab or mitoxantrone within 3 months prior to Screening.
5. Use of IV immunoglobulin within 3 weeks prior to Screening.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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58 participants in 1 patient group

Ravulizumab

Experimental group

Description:

During the Primary Treatment Period, all participants will receive open-label ravulizumab via intravenous (IV) infusion starting on Day 1. The end of the Primary Treatment Period will be triggered when the last enrolled participant completes between 26 and 50 weeks in the study (depending on the number of adjudicated On-Trial Relapse observed). After completion of the Primary Treatment Period, all participants will have the opportunity to continue receiving ravulizumab in the Long-Term Extension Period of the study. For each participant, the Long-Term Extension Period continues for up to 3 years, or until ravulizumab is approved and/or available (in accordance with country-specific regulations), whichever occurs first.

Treatment:

Biological: Ravulizumab