Generalized Myasthenia Gravis (gMG)Phase 3ECU-MG-304NCT06764160
Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Eculizumab in Chinese Adults With gMG
A Phase 3 study testing Eculizumab in people with Generalized Myasthenia Gravis (gMG) and Refractory gMG, run by Alexion Pharmaceuticals, Inc. at 6 sites in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Generalized Myasthenia Gravis (gMG), Refractory gMG
- What is being tested?
- Eculizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 6 sitesChina
- Study dates
- 6 Jan 2025to 29 Dec 2025
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Show all 11Show fewer
- 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
Who cannot join
- 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
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
China6 sites
Research Site
Changchun, 130021
Research Site
Fuzhou, 350001
Research Site
Guangzhou, 510080
Research Site
Guangzhou, 510620
Research Site
Qingdao, 266035
Research Site
Shanghai, 200040
About this study
Official title: An Open-label, Single-arm, Multi-center Study to Evaluate the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Eculizumab in Chinese Participants With Refractory Generalized Myasthenia Gravis (gMG)
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.
Read the detailed description
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.
- Being tested
- Eculizumab
- Keywords
- Generalized Myasthenia Gravis (gMG), Refractory gMG, Eculizumab
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EculizumabDrug
Participants will receive Eculizumab via intravenous (IV) infusion.
| Study group | Role | What they receive |
|---|---|---|
| Eculizumab | Experimental | Eculizumab Receives: Drug: Eculizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: from baseline at Week 26
Secondary outcomes (9)
To assess the efficacy of eculizumab in the treatment of refractory gMG by QMG
The mean change of quantitative myasthenia gravis (QMG) total score from baseline at Week 26 along with 2-sided 95% CI
Measured: from baseline at Week 26
To assess the efficacy of eculizumab in the treatment of refractory gMG by MGC
The mean change of myasthenia gravis composite (MGC) total score from baseline at Week 26 along with 2-sided 95% CI
Measured: from baseline at Week 26
To assess the efficacy of eculizumab in the treatment of refractory gMG by MG-QoL 15r
The mean change of revised Myasthenia Gravis Quality of Life 15-Item Scale (MG-QoL 15r scale) from baseline at Week 26 along with 2-sided 95% CI
Measured: from baseline at Week 26
To assess the efficacy of eculizumab in the treatment of refractory gMG by MG-ADL responder status
The proportion of participants who achieve the Myasthenia Gravis Activities of Daily Living Profile (MG-ADL) response at Week 26, along with the 2-sided 95% CI
Measured: from baseline at Week 26
To assess the efficacy of eculizumab in the treatment of refractory gMG by QMG responder analysis
The proportion of participants who achieve the quantitative myasthenia gravis (QMG) response at Week 26, along with the 2-sided 95% CI
Measured: from baseline at Week 26
To assess the safety and tolerability of eculizumab in the treatment of refractory gMG
Incidence of AEs, SAEs, and AEs leading to study intervention discontinuation (onset after first infusion)
Measured: from baseline at Week 26
To characterize the PK of eculizumab in participants with refractory gMG
Mean serum eculizumab concentrations at all scheduled visits
Measured: from baseline at Week 26
To characterize the PD of eculizumab in participants with refractory gMG
Mean changes in serum free complement component 5 (C5) concentrations at all scheduled visits
Measured: from baseline at Week 26
To characterize the immunogenicity of eculizumab in participants with refractory gMG
Proportion of treatment-emergent anti-drug antibody (ADA) positive participants
Measured: from baseline at Week 26
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jan 2025
- Study start6 Jan 2025
- Primary completion29 Dec 2025
- Study completion29 Dec 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Generalized Myasthenia Gravis (gMG)Studies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06764160
- Sponsor's protocol ID
- D7411C00003
- Other IDs
- ECU-MG-304
- Acronym
- ECU-MG-304
- Status last verified
- Jan 2026
- First submitted
- 11 Dec 2024
- Last updated
- 15 Jan 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries. Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal. All request will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.
Study at a glance
From the registry record, last updated by the sponsor on 15 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Single-arm, Multi-center Study to Evaluate the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Eculizumab in Chinese Participants With Refractory Generalized Myasthenia Gravis (gMG)
- ClinicalTrials.gov ID
- NCT06764160
- Acronym / sponsor ID
- ECU-MG-304 / D7411C00003
- Keywords
- Generalized Myasthenia Gravis (gMG), Refractory gMG, Eculizumab
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 6 Jan 2025 (actual)
- Primary completion
- 29 Dec 2025 (actual)
- Study completion
- 29 Dec 2025 (actual)
- First posted
- 8 Jan 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Eculizumab | Experimental | Drug: EculizumabEculizumab |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Diagnosis of MG must be made by the following tests:
- MGFA Clinical Classification Class II to IV at Screening
- MG-ADL total score must be ≥ 6 at Screening and Day 1
- Participants who have:
Key exclusion criteria (8 listed)
- 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:
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EculizumabParticipants will receive Eculizumab via intravenous (IV) infusion. | Drug | Eculizumab |
Primary outcome
To assess the efficacy of eculizumab in the treatment of refractory gMG based on the improvement in the MG-ADL
Time frame: from baseline at Week 26
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
9 secondary outcomes
To assess the efficacy of eculizumab in the treatment of refractory gMG by QMG
Time frame: from baseline at Week 26
To assess the efficacy of eculizumab in the treatment of refractory gMG by MGC
Time frame: from baseline at Week 26
To assess the efficacy of eculizumab in the treatment of refractory gMG by MG-QoL 15r
Time frame: from baseline at Week 26
To assess the efficacy of eculizumab in the treatment of refractory gMG by MG-ADL responder status
Time frame: from baseline at Week 26
To assess the efficacy of eculizumab in the treatment of refractory gMG by QMG responder analysis
Time frame: from baseline at Week 26
To assess the safety and tolerability of eculizumab in the treatment of refractory gMG
Time frame: from baseline at Week 26
To characterize the PK of eculizumab in participants with refractory gMG
Time frame: from baseline at Week 26
To characterize the PD of eculizumab in participants with refractory gMG
Time frame: from baseline at Week 26
To characterize the immunogenicity of eculizumab in participants with refractory gMG
Time frame: from baseline at Week 26
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Jan 2026.
- Individual participant dataSharing planned: Yes. Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries. Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal. All request will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Eculizumab in people with Generalized Myasthenia Gravis (gMG) and Refractory gMG, run by Alexion Pharmaceuticals, Inc. at 6 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 15 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To assess the efficacy of eculizumab in the treatment of refractory gMG based on the improvement in the MG-ADL (time frame: from baseline at Week 26). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Jan 2026.