STUDY OF ECULIZUMAB IN REFRACTORY GENERALIZED MYASTHENIA GRAVIS (GMG)SUBJECTS

Conditions: Generalized Myasthenia Gravis
MedDRA version: 18.0Level: HLTClassification code 10071942Term: Myasthenia gravis and related conditionsSystem Organ Class: 100000004859
Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Registration Number: EUCTR2013-003589-15-FR

Lead Sponsor: Alexion Pharmaceuticals, Inc.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 92

Inclusion Criteria

- Male or female subjects =18 years old
- Diagnosis of MG
- MGFA Clinical Classification Class II to IV at screening
- MG-ADL total score must be =6 at screening
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

- History of thymoma or other neoplasms of thymus
- History of thymectomy within 12 months prior to screening
- Weakness only affecting ocular or peri-ocular muscles (MGFA Class I)
- MG crisis at screening (MGFA Class V)
- Pregnancy or lactation
- Unresolved meningococcal infection

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: Assess the efficacy of eculizumab as compared with placebo in the treatment of refractory gMG based on the improvement in the MG-specific Activities of Daily Living profile (MG-ADL).;Secondary Objective: - Safety and tolerability of eculizumab as compared with placebo in gMG subjects<br>- Efficacy of eculizumab compared with placebo<br>- Describe the pharmacokinetics (PK) and pharmacodynamics (PD) of eculizumab<br>- Characterize the effect of eculizumab as compared with placebo on Quality of Life measures;Primary end point(s): - change from baseline in the MG-ADL total score for eculizumab as compared with placebo;Timepoint(s) of evaluation of this end point: Week 26

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): - Change from baseline in QMG total score <br>- Proportion of subjects with at least a 3-point reduction in the MG-ADL total score and with no rescue therapy<br>- Proportion of subjects with at least a 5 point reduction in the QMG total score<br>- Change from baseline in the Myasthenia Gravis Composite (MGC) scale total score<br>- Change from baseline in MG-QOL-15;Timepoint(s) of evaluation of this end point: Week 26