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

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 117

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

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

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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).<br> - 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.<br> - To characterize the effect of eculizumab on quality of life measures.<br> - 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

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): 1. Change from baseline in QMG total score.<br>2. Proportion of subjects with at least a 3-point reduction in the MG-ADL total score from baseline and with no rescue therapy.<br>3. Proportion of subjects with at least a 5-point reduction in the QMG total score from baseline and with no rescue therapy.<br>4. Change from baseline in the MGC scale total score.<br>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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