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Phase 3 N=117 Treatment

Extension Study of ECU-MG-301 to Evaluate Safety and Efficacy of Eculizumab in Refractory Generalized Myasthenia Gravis

Refractory Generalized Myasthenia Gravis

Enrolled (actual)
117
Serious AEs
51.3%
Results posted
Feb 2020
Primary outcome: Primary: Count Of Participants With Treatment-Emergent Adverse Events — 55; 59; 3; 5 Participants

Study Design & Population

Study type
Interventional
Phase
Phase 3
Interventions
Eculizumab (Biological); Placebo (Drug)
Age
Adult, Older Adult · 18+ yrs
Sex
All
Sponsor
Alexion Pharmaceuticals, Inc.
Primary completion
Jan 2019

Outcome Measures

OutcomeResultp-value
PRIMARY
Count Of Participants With Treatment-Emergent Adverse Events
55; 59; 3; 5; 30; 30
SECONDARY
Change From Baseline In Myasthenia Gravis Activities Of Daily Living Profile (MG-ADL) Total Score At Week 4 And Week 130
-0.2; -2.4; -0.7; -3.9

Summary

To evaluate the safety and efficacy of eculizumab in the treatment of refractory generalized myasthenia gravis (gMG) as an extension study for the participants who previously completed Study ECU-MG-301(NCT01997229).

Eligibility Criteria

Inclusion Criteria

  • Participant has completed Study ECU-MG-301.
  • Participant has given written informed consent.
  • Participant was willing and able to comply with the protocol requirements for the duration of the study.
  • Female participant of childbearing potential must have had a negative pregnancy test (serum human chorionic gonadotropin). All participants were required to practice an effective, reliable, and medically approved contraceptive regimen during the study and for up to 5 months following discontinuation of treatment.

Exclusion Criteria

  • Participants who withdrew from Study ECU-MG-301 as a result of an adverse event related to study drug.
  • Female participants who were pregnant, breastfeeding, or intended to conceive during the course of the study.
  • Unresolved meningococcal infection
  • Hypersensitivity to murine proteins or to one of the excipients of eculizumab
  • Any medical condition or circumstances that, in the opinion of the investigator, might have interfered with the participant's participation in the study, posed any added risk for the participant, or confounded the assessment of the participants.
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02301624). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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