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

January 27, 2020 updated by: Alexion Pharmaceuticals

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)

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).

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

ECU-MG-302 was an extension study designed to provide the participants who completed Study ECU-MG-301 an opportunity to receive eculizumab and collect clinical data to provide long-term safety and efficacy information on eculizumab in participants with refractory gMG.

After receiving blinded study treatment (eculizumab or placebo) in Study ECU-MG-301 for 26 weeks, participants were eligible to enroll in the ECU-MG-302 extension study. Participants were to enter Study ECU-MG-302 within 2 weeks after completing their Week 26 visit in Study ECU-MG-301.

Study ECU-MG-302 consisted of a 4-week Blind Induction Phase to preserve the blinded nature of Study ECU-MG-301, an Open-Label Maintenance Phase (up to 4 years), and a Safety Follow-up visit 8 weeks after the last dose for participants who withdrew from the study or discontinued eculizumab treatment at any time and for any reason after receiving any amount of eculizumab.

Study Type

Interventional

Enrollment (Actual)

117

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Argentina
      • Buenos Aires, Argentina
  • Belgium
      • Edegem, Belgium
      • Gent, Belgium
      • Leuven, Belgium
  • Brazil
      • São Paulo, Brazil
  • Canada
      • Edmonton, Canada
  • Czechia
      • Ostrava - Poruba, Czechia
      • Praha 2, Czechia
  • Denmark
      • Arhus C, Denmark
      • Kobenhavn, Denmark
  • Finland
      • Turku, Finland
  • Hungary
      • Szeged, Hungary
  • Italy
      • Milano, Italy
      • Napoli, Italy
      • Roma, Italy
  • Japan
      • Chiba, Japan
      • Fukuoka, Japan
      • Hanamaki, Japan
      • Hokkaido, Japan
      • Miyagi, Japan
      • Nagasaki, Japan
      • Osaka, Japan
      • Osaka-Fu, Japan
  • Korea, Republic of
      • Seoul, Korea, Republic of
  • Netherlands
      • Amsterdam, Netherlands
  • Spain
      • Barcelona, Spain
      • Madrid, Spain
  • Sweden
      • Stockholm, Sweden
  • Turkey
      • Ankara, Turkey
      • Kocaeli, Turkey
      • İzmir, Turkey
  • United Kingdom
      • Birmingham, United Kingdom
      • Liverpool, United Kingdom
      • London, United Kingdom
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35233
    • California
      • Los Angeles, California, United States, 90033
      • Orange, California, United States, 92868
      • Palo Alto, California, United States, 94304
      • San Francisco, California, United States, 94115
    • Connecticut
      • New Haven, Connecticut, United States, 06519
    • Florida
      • Jacksonville, Florida, United States, 32209
      • Miami, Florida, United States, 33136
      • Tampa, Florida, United States, 33612
    • Illinois
      • Springfield, Illinois, United States, 62702
    • Indiana
      • Indianapolis, Indiana, United States, 46202
    • Iowa
      • Iowa City, Iowa, United States, 52242
    • Kansas
      • Kansas City, Kansas, United States, 66160
    • Maryland
      • Baltimore, Maryland, United States, 21201-1595
      • Baltimore, Maryland, United States, 21287-0876
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
      • Burlington, Massachusetts, United States, 01805
    • Nevada
      • Las Vegas, Nevada, United States, 89145
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27599
      • Charlotte, North Carolina, United States, 28207
      • Durham, North Carolina, United States, 27710
    • Ohio
      • Columbus, Ohio, United States, 43210
    • Oregon
      • Portland, Oregon, United States, 97239
    • Texas
      • San Antonio, Texas, United States, 78229
    • Vermont
      • Burlington, Vermont, United States, 05401
    • Washington
      • Seattle, Washington, United States, 98195

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  1. Participant has completed Study ECU-MG-301.
  2. Participant has given written informed consent.
  3. Participant was willing and able to comply with the protocol requirements for the duration of the study.
  4. 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:

  1. Participants who withdrew from Study ECU-MG-301 as a result of an adverse event related to study drug.
  2. Female participants who were pregnant, breastfeeding, or intended to conceive during the course of the study.
  3. Unresolved meningococcal infection
  4. Hypersensitivity to murine proteins or to one of the excipients of eculizumab
  5. 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Eculizumab/Eculizumab

Blind Induction Phase: Participants who had received blinded treatment with eculizumab in Study ECU-MG-301 were administered eculizumab (4 vials/1200 milligrams [mg]) on Day 1 and Week 2 and placebo (4 vials/0 mg) at Weeks 1 and 3.

Open-Label Maintenance Phase: Participants received open-label eculizumab (4 vials/1200 mg) every 2 weeks starting at Week 4 and continued throughout the study.

Eculizumab 1200 mg was administered for up to 4 years in this extension study.
Biological: Eculizumab
Intravenous administration of eculizumab.
Drug: Placebo
Intravenous administration of matching placebo. Participants received placebo only during the Blind Induction Phase.
Experimental: Placebo/Eculizumab

Blind Induction Phase: Participants who had received blinded treatment with placebo in Study ECU-MG-301 were administered eculizumab/placebo (3 vials/900 mg, plus 1 vial/0 mg, respectively) on Day 1 and Weeks 1 through 3.

Open-Label Maintenance Phase: Participants received open-label eculizumab (4 vials/1200 mg) every 2 weeks starting at Week 4 and continued throughout the study.

Eculizumab 1200 mg was administered for up to 4 years in this extension study.
Biological: Eculizumab
Intravenous administration of eculizumab.
Drug: Placebo
Intravenous administration of matching placebo. Participants received placebo only during the Blind Induction Phase.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Count Of Participants With Treatment-Emergent Adverse Events
Time Frame: Day 1 (after dosing) through End of Study (Week 208)
Treatment-emergent adverse events (TEAEs) are adverse events with onset on or after the first study drug dose in Study ECU-MG-302. Likewise, treatment-emergent serious adverse events (TESAEs) are serious adverse events that onset on or after the first study drug dose in Study ECU-MG-302. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module.
Day 1 (after dosing) through End of Study (Week 208)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change From Baseline In Myasthenia Gravis Activities Of Daily Living Profile (MG-ADL) Total Score At Week 4 And Week 130
Time Frame: Baseline, Week 4 and Week 130
The MG-ADL scale is a validated 8-item patient-reported outcome measure. Participants assessed their functional disability secondary to ocular (2 items), bulbar (3 items), respiratory (1 item), and gross motor or limb impairment (2 items). These 8 items were not weighted and were individually graded from 0 (normal) to 3 (most severe), providing a total MG-ADL score ranging from 0 to 24 points. A reduction in score indicates improvement in condition. Baseline was defined as the last available assessment prior to treatment (first study drug infusion) with eculizumab in Study ECU-MG-302. Change from Baseline in MG-ADL total score at Week 4 (blind induction phase) and at Week 130 (open-label eculizumab phase) are presented.
Baseline, Week 4 and Week 130

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alexion Pharmaceuticals

Investigators

  • Study Director: Marcus Yountz, MD, Alexion Pharmaceuticals

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 12, 2014

Primary Completion (Actual)

January 15, 2019

Study Completion (Actual)

January 15, 2019

Study Registration Dates

First Submitted

November 22, 2014

First Submitted That Met QC Criteria

November 25, 2014

First Posted (Estimate)

November 26, 2014

Study Record Updates

Last Update Posted (Actual)

February 5, 2020

Last Update Submitted That Met QC Criteria

January 27, 2020

Last Verified

January 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ECU-MG-302
  • 2013-002191-41 (EudraCT Number)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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