Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity Study of Ravulizumab in Chinese Adults With Neuromyelitis Optica Spectrum Disorder (NMOSD)

Официальное название

A Phase 3b, Open-label, Single-arm, Multicenter Study to Evaluate the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adult Participants With Neuromyelitis Optica Spectrum Disorder (NMOSD)

Обзор

The primary objective of this study is to confirm the efficacy, safety, pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of ravulizumab in the treatment of Chinese adults with anti-aquaporin-4 (AQP4) antibody (Ab) + neuromyelitis optica spectrum disorder (NMOSD).

Вмешательства

• Препарат Ravulizumab
  Participants will receive ravulizumab via intravenous (IV) infusion.

Первичные конечные точки

• Adjudicated On-Trial Annualized Relapse Rate (ARR) [Срок оценки: Baseline up to Week 50]

Вторичные конечные точки (9)

• Number of Participants With Clinically Important Change From Baseline in Hauser Ambulation Index (HAI) Score [Срок оценки: Baseline up to Week 50]
• Number of Participants With Clinically Important Worsening From Baseline in Expanded Disability Status Scale (EDSS) Score [Срок оценки: Baseline up to Week 50]
• Change From Baseline in European Quality of Life Health 5-item Questionnaire (EQ-5D) Index Score [Срок оценки: Baseline, Week 50]
• Change From Baseline in EQ-5D Visual Analog Scale (VAS) Score [Срок оценки: Baseline, Week 50]
• Number of Participants With Treatment Emergent Adverse Events (TEAEs), Treatment Emergent Serious Adverse Events (TESAEs) and Adverse Events of Special Interests (AESIs) [Срок оценки: Baseline up to Week 50]
• Serum Ravulizumab Concentration [Срок оценки: Day 1 to Day 351]
• Change From Baseline in Serum Free Complement Component 5 (C5) Concentration [Срок оценки: Baseline Up to Day 351]
• Number of Participants With Anti-Drug Antibodies (ADAs) [Срок оценки: Day 1 up to Day 351]
• Number of Participants With Neutralizing Antibodies (NAb) [Срок оценки: Day 1 up to Day 351]

Критерии участия

Key Inclusion (essential)

• Diagnosis: NMOSD per 2015 international consensus criteria, and anti AQP4 antibody positive at Screening.
• Disease activity: ≥1 attack/relapse in the past 12 months.
• Disability: EDSS ≤7.
• Background therapy: If on IST and/or oral corticosteroids, participant should be on a stable maintenance regimen prior to Screening and plan to remain stable during the study unless relapse occurs. (Detailed agent specific duration/dose rules to be confirmed at screening.)
• Body weight: ≥40 kg.
• Vaccinated against meningococcal infections from serogroups A, C, W, Y (and B where available) within the 3 years prior to study intervention administration on Day 1.

Key Exclusion (essential)

• Pregnancy/lactation: Pregnant, breastfeeding, or intending to conceive during the study.
• Infection risk: History of meningococcal disease or unresolved meningococcal disease, active systemic infection within 14 days, or fever ≥38°C within 7 days before Day 1.
• Hypersensitivity: To murine proteins or ravulizumab excipients.
• Serious comorbidities: Any condition that in the Investigator's judgment adds risk or interferes with participation/assessment.
• Viral infections: Known HIV, active HBV, or active HCV.
• Prior/concomitant immunomodulatory treatments:

• B cell-depleting therapy (e.g., rituximab, inebilizumab) within 3 months before Screening.
• Mitoxantrone or satralizumab within 3 months before Screening.
• IVIg within 3 weeks before Screening.
• Any prior or current complement inhibitor.

Note: Other protocol-defined criteria may apply and should be verified during full eligibility review.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Китай · 5 центров

• Research Site — Чэнду
• Research Site — Дунгуань
• Research Site — Шанхай
• Research Site — Wenzhou
• Research Site — Ухань

Идентификаторы

NCT: NCT07557420 · D9282C00006 · ALXN1210-NMO-324

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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