Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Eculizumab in Chinese Adults With gMG

NCT06764160 | Completed Phase 3

• 2 other identifiers: D7411C00003ECU-MG-304
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 6

Brief Summary

This is an open-label, single-arm, multi-center study to evaluate the efficacy, safety, PK, PD, and immunogenicity of eculizumab in Chinese participants with refractory gMG. Approximately 15 participants will be enrolled in the study.

Conditions

Refractory gMG; Generalized Myasthenia Gravis (gMG)

Keywords

Generalized Myasthenia Gravis (gMG); Refractory gMG; Eculizumab

Outcome Measures

Primary Outcomes (1)

• To assess the efficacy of eculizumab in the treatment of refractory gMG based on the improvement in the MG-ADL

  The mean change of Myasthenia Gravis Activities of Daily Living Profile (MG-ADL) total score from baseline at Week 26 along with 2-sided 95% CI

  from baseline at Week 26

Secondary Outcomes (9)

• To assess the efficacy of eculizumab in the treatment of refractory gMG by QMG

  from baseline at Week 26

• To assess the efficacy of eculizumab in the treatment of refractory gMG by MGC

  from baseline at Week 26

• To assess the efficacy of eculizumab in the treatment of refractory gMG by MG-QoL 15r

  from baseline at Week 26

• To assess the efficacy of eculizumab in the treatment of refractory gMG by MG-ADL responder status

  from baseline at Week 26

• To assess the efficacy of eculizumab in the treatment of refractory gMG by QMG responder analysis

  from baseline at Week 26

Study Arms (1)

Eculizumab

EXPERIMENTAL

Eculizumab

Drug: Eculizumab

Interventions

Eculizumab DRUG

Participants will receive Eculizumab via intravenous (IV) infusion.

Eculizumab

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of MG must be made by the following tests:
• Positive serologic test for anti-AChR Abs as confirmed at Screening, and
• One of the following:
• Abnormal neuromuscular transmission demonstrated by repetitive nerve stimulation, or
• History of positive anticholinesterase test, eg, neostigmine test, or
• Participant has demonstrated improvement in MG signs on oral cholinesterase inhibitors as assessed by the treating physician
• MGFA Clinical Classification Class II to IV at Screening
• MG-ADL total score must be ≥ 6 at Screening and Day 1
• Participants who have:
• Failed treatment with 2 or more ISTs over one year (either in combination or as monotherapy), ie, continue to have impairment ADLs (persistent weakness, experience crisis, or unable to tolerate IST) despite ISTs or,
• Failed at least one IST and require chronic PE or IVIg to control symptoms, ie, participants who require PE or IVIg on a regular basis for the management of muscle weakness at least 2 cycles over last 12 months

You may not qualify if:

• Any untreated thymic malignancy, carcinoma, or thymoma
• History of thymectomy or any other thymic surgery within 6 months prior to Screening. Participants with a history of treated thymic malignancy or carcinoma are eligible if they meet all of the following conditions:
• Treatment completed \> 5 years prior to the Screening Visit
• No recurrence within the 5 years prior to the Screening Visit
• No radiological indication of recurrence in a computed tomography (CT) or magnetic resonance imaging (MRI) scan, including administration of intravenous (IV) contrast, performed within 6 months of first dose on Day 1
• Weakness only affecting ocular or peri-ocular muscles (MGFA Class I)
• MG crisis at Screening (MGFA Class V). However, such participants may be rescreened with Alexion approval once they are treated and medically stable, in the opinion of the Investigator
• History of N meningitidis infection or unresolved meningococcal disease

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Study Sites (6)

Research Site

Changchun, 130021, China

Location

Research Site

Fuzhou, 350001, China

Location

Research Site

Guangzhou, 510080, China

Location

Research Site

Guangzhou, 510620, China

Location

Research Site

Qingdao, 266035, China

Location

Research Site

Shanghai, 200040, China

Location

MeSH Terms

Conditions

Myasthenia Gravis

Interventions

eculizumab

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 11, 2024
• First Posted: January 8, 2025
• Study Start: January 6, 2025
• Primary Completion: December 29, 2025
• Study Completion: December 29, 2025
• Last Updated: January 15, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR
• Time Frame: AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA Pharma Data Sharing Principles. For details of our timelines, please refer to our disclosure commitment at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
• Access Criteria: When a request has been approved AstraZeneca will provide access to the deidentified individual patient-level data in an approved sponsored tool. Signed Data Sharing Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information. Additionally, all users will need to accept the terms and conditions of the SAS MSE to gain access. For additional details, please review the Disclosure Statements at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.

Locations

CN (6)