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

Official title

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)

Overview

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).

Interventions

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

Primary outcome measures

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

Secondary outcome measures (9)

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

Eligibility criteria

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:

• ALT >2× ULN, direct bilirubin >2× ULN.
• eGFR <30 mL/min/1.73 m² or on dialysis.
• Any other clinically significant lab abnormality making participation unsafe.

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

Non-randomized

Model

Single group

Masking

Open label

Primary purpose

Treatment

Study locations

China · 10 centers

• Research Site — Beijing
• Research Site — Changchun
• Research Site — Fuzhou
• Research Site — Jinan
• Research Site — Shanghai
• Research Site — Shijiazhuang
• Research Site — Taiyuan
• Research Site — Tianjin
• … and 2 more centers

Identifiers

NCT: NCT07596784 · D9281C00003 · ALXN1210-MG-326

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗