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

Study Purpose

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.

Recruitment Criteria

Accepts Healthy Volunteers Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms	No
Study Type An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes. Searching Both is inclusive of interventional and observational studies.	Interventional
Eligible Ages	18 Years and Over
Gender	All

More Inclusion & Exclusion Criteria

Inclusion Criteria:

- Diagnosis of MG must be made by the following tests: 1.

Positive serologic test for anti-AChR Abs as confirmed at Screening, and. 2. One of the following: 1. Abnormal neuromuscular transmission demonstrated by repetitive nerve stimulation, or. 2. History of positive anticholinesterase test, eg, neostigmine test, or. 3. 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: 1.

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, 2. 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.

Exclusion Criteria:

- 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: 1. Treatment completed > 5 years prior to the Screening Visit. 2. No recurrence within the 5 years prior to the Screening Visit. 3. 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

Trial Details

Trial ID: This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.	NCT06764160
Phase Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans. Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.	Phase 3
Lead Sponsor The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.	Alexion Pharmaceuticals, Inc.
Principal Investigator The person who is responsible for the scientific and technical direction of the entire clinical study.	N/A
Principal Investigator Affiliation	N/A
Agency Class Category of organization(s) involved as sponsor (and collaborator) supporting the trial.	Industry
Overall Status	Recruiting
Countries	China
Conditions The disease, disorder, syndrome, illness, or injury that is being studied.	Generalized Myasthenia Gravis (gMG), Refractory gMG

Additional Details

This post-approval study 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. There will be 3 periods in this study: Screening Period (up to 4 weeks), Treatment Period (26 weeks, including an Induction Phase and a Maintenance Phase), and Safety Follow-up Period (8 weeks). The overall study duration for an individual participant is estimated to be up to 38 weeks. Approximately 15 participants will be enrolled in the study.

Arms & Interventions

Arms

Experimental: Eculizumab

Eculizumab

Interventions

Drug: - Eculizumab

Participants will receive Eculizumab via intravenous (IV) infusion.

Contact a Trial Team

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