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)

Phase 3

Completed

Conditions: Refractory generalized myasthenia gravis
alter
Antibodies block
or destroy the receptors for acetylcholine at the NMJ (neuromuscular junction)
Neuromuscular Disease

Registration Number: NL-OMON41966

Lead Sponsor: Alexion Pharmaceuticals

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 3

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.

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

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>- Safety and tolerability of eculizumab.<br /><br>Primary efficacy endpoint:<br /><br>- Change from baseline in the MG-ADL total score.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>1. Change from baseline in QMG total score.<br /><br>2. Proportion of subjects with at least a 3-point reduction in the MG-ADL total<br /><br>score from baseline and with no rescue therapy.<br /><br>3. Proportion of subjects with at least a 5-point reduction in the QMG total<br /><br>score from baseline and with no rescue therapy.<br /><br>4. Change from baseline in the MGC scale total score.<br /><br>5. Change from baseline in MG-QOL-15.</p><br>