Набор скоро начнётся NCT07596784

Efficacy and Safety of Ravulizumab in Chinese Adults Participants With Generalized Myasthenia Gravis (gMG)

Фаза III С лечением Generalized Myasthenia Gravis gMG

Ориентир для пациента и семьи

Простыми словами

Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.

Что изучают: В протоколе указаны: Ravulizumab.
Кому может быть актуально: Состояния в реестре: Generalized Myasthenia Gravis, gMG. Базовые параметры: 18 лет — 130 лет · Все.
Что важно проверить: Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
Где проводится: Китай
Следующий шаг: Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →

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Официальное название

An Open-label, Single-arm, Multi-center, Interventional Study to Evaluate the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adult Patients With Generalized Myasthenia Gravis (gMG)

Обзор

The primary purpose of this study is to evaluate the efficacy, safety, pharmacokinetics, pharmacodynamics, and immunogenicity of ravulizumab in Chinese adult participants with Acetylcholine receptor (AChR) + Generalized Myasthenia Gravis (gMG).

Вмешательства

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

Первичные конечные точки

Change From Baseline in Myasthenia Gravis Activities of Daily Living Profile (MG-ADL) Total Score at Week 26 [Срок оценки: Baseline, Week 26]

Вторичные конечные точки (9)

Change From Baseline in Quantitative Myasthenia gravis (QMG) Total Score at Week 26 [Срок оценки: Baseline, Week 26]
Number of Participants With Reduction by >=5 Points From Baseline in QMG Total Score at Week 26 [Срок оценки: Baseline up to Week 26]
Number of Participants With Reduction by >=3 Points From Baseline in MG-ADL Total Score at Week 26 [Срок оценки: Baseline up to Week 26]
Change From Baseline in Revised Myasthenia Gravis Quality of Life 15-Item Scale (MG-QoL15r) Total Score at Week 26 [Срок оценки: Baseline, Week 26]
Change From Baseline in Neurological Quality of Life (Neuro-QoL) Fatigue Score at Week 26 [Срок оценки: Baseline, Week 26]
Serum Ravulizumab Concentration [Срок оценки: Day 1 up to Week 26]
Change From Baseline in Serum Free C5 Concentration [Срок оценки: Baseline, Week 34]
Number of Participants With Anti-Drug Antibodies (ADAs) [Срок оценки: Baseline up to Week 34]
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 34]

Критерии участия

Inclusion (key)

Confirmed generalized MG: Diagnosis ≥6 months before screening, anti-AChR antibody positive, and supportive diagnostic evidence (e.g., abnormal SFEMG/RNS or response to anticholinesterase therapy).
Disease severity: MGFA Class II-IV at screening.
Symptoms threshold: MG-ADL ≥6 at screening and on Day 1.
Meningococcal vaccination: Up to date within 3 years or vaccinated before first dose to mitigate risk with complement inhibition.
Body weight: ≥40 kg.
Vaccinated against meningococcal infections within the 3 years prior to, or at the time of, initiating study drug.

Exclusion (key)

Thymic disease:

Untreated thymic malignancy/carcinoma/thymoma excluded.
Prior thymic malignancy allowed only if treatment completed >5 years, no recurrence in last 5 years, and clear CT/MRI within 6 months.
Prior benign thymoma allowed if confirmed benign, treatment >12 months ago, no recurrence in last 12 months, and clear CT/MRI within 6 months; otherwise follow malignancy rules.
Thymectomy within the last 12 months
Infection risk:

History of meningococcal disease or unresolved infection, or active systemic infection within 14 days of Day 1 excluded.
Persistent/recurrent infections in past 12 months that add risk
HIV, active HBV (HBsAg+ or anti-HBc+ with anti-HBs-), or active HCV (unless documented successful treatment/SVR)
Safety/medical status:

Hypersensitivity to study drug components (including murine proteins)
Recent hospitalization ≥24 hours within 28 days of screening
Substance use disorder per DSM within 12 months.
Recent/other malignancy within 5 years (except as above for thymic).
Prior/Concomitant Therapy

Complement inhibitor within < 5 half-lives before Day 1.
Human neonatal Fc receptor (FcRn) inhibitor within < 5 half-lives before Day 1.
Rituximab, ocrelizumab or other B cell-depleting therapy within ≤ 6 months (180 days) before Day 1.
Periodic (chronic) administration of PP/PE, or IVIg as maintenance therapy received or scheduled within ≤ 6 months before Day 1
Key labs: