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

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ClinicalTrials.gov Identifier: NCT02301624
Recruitment Status : Completed
First Posted : November 26, 2014
Results First Posted : February 5, 2020
Last Update Posted : February 5, 2020
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Brief 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).

Condition or disease Intervention/treatment Phase
Refractory Generalized Myasthenia Gravis Biological: Eculizumab Drug: Placebo Phase 3

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 117 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Following the Blind Induction Phase, all participants received open-label eculizumab.
Masking: None (Open Label)
Masking Description: The study consisted of a 4-week Blind Induction Phase to preserve the blinded nature of Study ECU-MG-301, followed by an Open-Label Maintenance Phase.
Primary Purpose: Treatment
Official Title: 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)
Actual Study Start Date : November 12, 2014
Actual Primary Completion Date : January 15, 2019
Actual Study Completion Date : January 15, 2019

Resource links provided by the National Library of Medicine

Drug Information available for: Eculizumab

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.




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02301624


Locations
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Sponsors and Collaborators
Alexion Pharmaceuticals
Investigators
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Study Director: Marcus Yountz, MD Alexion Pharmaceuticals
  Study Documents (Full-Text)

Documents provided by Alexion Pharmaceuticals:
Study Protocol  [PDF] December 19, 2016
Statistical Analysis Plan  [PDF] February 6, 2019

Publications of Results:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02301624    
Other Study ID Numbers: ECU-MG-302
2013-002191-41 ( EudraCT Number )
First Posted: November 26, 2014    Key Record Dates
Results First Posted: February 5, 2020
Last Update Posted: February 5, 2020
Last Verified: January 2020
Keywords provided by Alexion Pharmaceuticals:
gMG
Myasthenia Gravis
safety
efficacy
Additional relevant MeSH terms:
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Muscle Weakness
Myasthenia Gravis
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Pathologic Processes
Autoimmune Diseases of the Nervous System
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases