EXTENSION STUDY OF ECU-MG-301 TO EVALUATE ECULIZUMAB IN REFRACTORY GENERALIZED MYASTHENIA GRAVIS (GMG) SUBJECTS

A PHASE III, OPEN-LABEL, EXTENSION TRIAL OF ECU-MG-301 TO EVALUATE THE SAFETY AND EFFICACY OF ECULIZUMAB IN SUBJECTS WITH REFRACTORY GENERALIZED MYASTHENIA GRAVIS (gMG)

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2013-002191-41-GB

Enrollment

Registered

2014-07-14

Start date

2014-10-27

Completion date

Unknown

Last updated

2020-07-27

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Generalized Myasthenia Gravis MedDRA version: 19.1 Level: HLT Classification code 10071942 Term: Myasthenia gravis and related conditions System Organ Class: 100000004859

Interventions

Trade Name: SOLIRIS Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: ECULIZUMAB CAS Number: 219685-50-4 Current Sponsor code: h5G1.1-mAb Concentration unit: mg/ml millig

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Subject has completed the ECU-MG-301 trial. 2. Subject has given written informed consent. 3. Subject is willing and able to comply with the protocol requirements for the duration of the trial. 4. Female subjects of child-bearing potential must have a negative pregnancy test (serum human chorionic gonadotropin [HCG]). All subjects must practice an effective, reliable and medically approved contraceptive regimen during the trial and for up to 5 months following discontinuation of treatment. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 83 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 9

Exclusion criteria

Exclusion criteria: 1. Subjects who withdrew from the ECU-MG-301 trial as a result of an AE related to trial drug. 2. Female subjects who are pregnant, breastfeeding or intend to conceive during the course of the trial. 3. Any medical condition or circumstances that, in the opinion of the Investigator, might interfere with the subject’s participation in the trial, pose any added risk for the subject, or confound the assessment of the subjects. 4. Unresolved meningococcal infection 5. Hypersensitivity to murine proteins or one of the excipients of eculizumab

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate the long-term safety of eculizumab in subjects with refractory gMG.;Secondary Objective: - To evaluate the long-term efficacy of eculizumab in subjects with refractory gMG as measured by the improvement or maintenance of the MG-specific Activities of Daily Living profile (MG-ADL). - To evaluate the long-term efficacy of eculizumab by additional efficacy measures including: Quantitative Myasthenia Gravis (QMG) score, Myasthenia Gravis Composite (MGC) score, and Improvement or maintenance in primary symptoms that are most clinically meaningful to the subjects. - To characterize the effect of eculizumab on quality of life measures. - To describe the pharmacokinetics (PK) and pharmacodynamics (PD) of eculizumab in subjects with gMG.;Primary end point(s): Change from baseline in the MG-ADL total score.;Timepoint(s) of evaluation of this end point: From week 1 to 182	—

Measure

Time frame

Main Objective: To evaluate the long-term safety of eculizumab in subjects with refractory gMG.;Secondary Objective: - To evaluate the long-term efficacy of eculizumab in subjects with refractory gMG as measured by the improvement or maintenance of the MG-specific Activities of Daily Living profile (MG-ADL). - To evaluate the long-term efficacy of eculizumab by additional efficacy measures including: Quantitative Myasthenia Gravis (QMG) score, Myasthenia Gravis Composite (MGC) score, and Improvement or maintenance in primary symptoms that are most clinically meaningful to the subjects. - To characterize the effect of eculizumab on quality of life measures. - To describe the pharmacokinetics (PK) and pharmacodynamics (PD) of eculizumab in subjects with gMG.;Primary end point(s): Change from baseline in the MG-ADL total score.;Timepoint(s) of evaluation of this end point: From week 1 to 182

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Secondary

Measure	Time frame
Secondary end point(s): 1. Change from baseline in QMG total score. 2. Proportion of subjects with at least a 3-point reduction in the MG-ADL total score from baseline and with no rescue therapy. 3. Proportion of subjects with at least a 5-point reduction in the QMG total score from baseline and with no rescue therapy. 4. Change from baseline in the MGC scale total score. 5. Change from baseline in MG-QOL-15.;Timepoint(s) of evaluation of this end point: From week 1 to 182	—

Measure

Time frame

Secondary end point(s): 1. Change from baseline in QMG total score. 2. Proportion of subjects with at least a 3-point reduction in the MG-ADL total score from baseline and with no rescue therapy. 3. Proportion of subjects with at least a 5-point reduction in the QMG total score from baseline and with no rescue therapy. 4. Change from baseline in the MGC scale total score. 5. Change from baseline in MG-QOL-15.;Timepoint(s) of evaluation of this end point: From week 1 to 182

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Countries

Argentina, Australia, Austria, Belgium, Brazil, Canada, Czech Republic, Denmark, Finland, France, Germany, Greece, Hungary, Ireland, Italy, Japan, Korea, Republic of, Netherlands, Norway, Russian Federation, Spain, Sweden, Switzerland, Turkey, United Kingdom, United States

Contacts

Public ContactEuropean Clinical Trial Information

ALEXION EUROPE SAS

clinicaltrials.eu@alexion.com+33147100606

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026