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

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)

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).

Study Overview

• Status: Not yet recruiting
• Conditions: Generalized Myasthenia Gravis; gMG
• Intervention / Treatment: Drug: Ravulizumab

• Study Type: Interventional
• Enrollment (Estimated): 20
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Alexion Pharmaceuticals, Inc. (Sponsor); Phone Number: 1-855-752-2356; Email: clinicaltrials@alexion.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Ravulizumab; Participants will receive ravulizumab for up to 26 weeks.	Drug: Ravulizumab; Participants will receive ravulizumab via intravenous (IV) infusion.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Change From Baseline in Myasthenia Gravis Activities of Daily Living Profile (MG-ADL) Total Score at Week 26	Baseline, Week 26

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Alexion Pharmaceuticals, Inc.

Study record dates

Study Major Dates

• Study Start (Estimated): July 30, 2026
• Primary Completion (Estimated): August 27, 2027
• Study Completion (Estimated): August 27, 2027

Study Registration Dates

• First Submitted: May 13, 2026
• First Submitted That Met QC Criteria: May 13, 2026
• First Posted (Actual): May 19, 2026

Study Record Updates

• Last Update Posted (Actual): May 19, 2026
• Last Update Submitted That Met QC Criteria: May 13, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: gMG; generalized myasthenia gravis; ravulizumab
• Additional Relevant MeSH Terms: Nervous System Diseases; Neoplasms by Site; Neoplasms; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases; Autoimmune Diseases of the Nervous System; Neurodegenerative Diseases; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Paraneoplastic Syndromes; Neuromuscular Junction Diseases; Myasthenia Gravis; ravulizumab
• Other Study ID Numbers: D9281C00003; ALXN1210-MG-326 (Other Identifier: Alexion)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No