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 |
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Recruitment Status :
Completed
First Posted : November 26, 2014
Results First Posted : February 5, 2020
Last Update Posted : February 5, 2020
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| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Refractory Generalized Myasthenia Gravis | Biological: Eculizumab Drug: Placebo | Phase 3 |
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 (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 |
| Arm | Intervention/treatment |
|---|---|
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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. |
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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. |
- 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.
- 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.
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| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
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.
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
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| Study Director: | Marcus Yountz, MD | Alexion |
Documents provided by Alexion:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | Alexion |
| 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 |
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gMG Myasthenia Gravis safety efficacy |
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Myasthenia Gravis Muscle Weakness Muscular Diseases Musculoskeletal Diseases Neuromuscular Manifestations Neurologic Manifestations Nervous System Diseases Pathologic Processes Paraneoplastic Syndromes, Nervous System Nervous System Neoplasms Neoplasms by Site Neoplasms |
Paraneoplastic Syndromes Autoimmune Diseases of the Nervous System Neurodegenerative Diseases Neuromuscular Junction Diseases Neuromuscular Diseases Autoimmune Diseases Immune System Diseases Eculizumab Complement Inactivating Agents Immunosuppressive Agents Immunologic Factors Physiological Effects of Drugs |