Last reviewed · How we verify

NCT02301624

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

Completed Phase 3 Results posted Last updated 5 February 2020
What this trial tests

Phase 3 trial testing Eculizumab in Refractory Generalized Myasthenia Gravis in 117 participants. Completed in 15 January 2019.

Timeline
12 November 2014
Primary endpoint
15 January 2019
15 January 2019

Quick facts

Lead sponsorAlexion Pharmaceuticals, Inc.
PhasePhase 3
StatusCompleted
Study typeINTERVENTIONAL
Allocationnon randomized
Designparallel
Maskingnone
Primary purposetreatment
Enrollment117
Start date12 November 2014
Primary completion15 January 2019
Estimated completion15 January 2019
Sites60 locations across Denmark, Italy, Finland, Japan, Netherlands, Belgium, Sweden, United Kingdom

Drugs / interventions tested

Conditions studied

Sponsor

Alexion Pharmaceuticals, Inc. — full company profile →

Who can join

18 and older, any sex, with Refractory Generalized Myasthenia Gravis. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Count Of Participants With Treatment-Emergent Adverse Events Primary · 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.

TEAEs
GroupValue95% CI
Eculizumab/Eculizumab55
Placebo/Eculizumab59
TEAEs leading to withdrawal
GroupValue95% CI
Eculizumab/Eculizumab3
Placebo/Eculizumab5
TESAEs
GroupValue95% CI
Eculizumab/Eculizumab30
Placebo/Eculizumab30
TESAEs leading to withdrawal
GroupValue95% CI
Eculizumab/Eculizumab3
Placebo/Eculizumab4
Deaths
GroupValue95% CI
Eculizumab/Eculizumab2
Placebo/Eculizumab1
Change From Baseline In Myasthenia Gravis Activities Of Daily Living Profile (MG-ADL) Total Score At Week 4 And Week 130 Secondary · 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 f

Change from Baseline At Week 4 (Blind Induction)
GroupValue95% CI
Eculizumab/Eculizumab-0.2± 1.77
Placebo/Eculizumab-2.4± 3.04
Change from Baseline at Week 130 (Open-label)
GroupValue95% CI
Eculizumab/Eculizumab-0.7± 4.19
Placebo/Eculizumab-3.9± 3.68

Adverse events — posted to ClinicalTrials.gov

Time frame: Day 1 after dosing of study drug through Week 208.. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Eculizumab/Eculizumab
Serious: 30/56 (54%)
Deaths: 2/56
Placebo/Eculizumab
Serious: 30/61 (49%)
Deaths: 1/61
Eculizumab (Combined Total)
Serious: 60/117 (51%)
Deaths: 3/117

Serious adverse events (105 terms)

ReactionSystemEculizumab/EculizumabPlacebo/EculizumabEculizumab (Combined Total)
Myasthenia gravisNervous system disorders
PneumoniaInfections and infestations
Myasthenia gravis crisisNervous system disorders
AnaemiaBlood and lymphatic system disorders
PyrexiaGeneral disorders
GastroenteritisInfections and infestations
SepsisInfections and infestations
HeadacheNervous system disorders
Acute respiratory failureRespiratory, thoracic and mediastinal disorders
Pulmonary embolismRespiratory, thoracic and mediastinal disorders
BronchitisInfections and infestations
InfluenzaInfections and infestations
Localised infectionInfections and infestations
Upper respiratory tract infectionInfections and infestations
Urinary tract infectionInfections and infestations
Humerus fractureInjury, poisoning and procedural complications
Colon cancerNeoplasms benign, malignant and unspecified (incl cysts and polyps)
Acute kidney injuryRenal and urinary disorders
Pneumonia aspirationRespiratory, thoracic and mediastinal disorders
Disseminated intravascular coagulationBlood and lymphatic system disorders
Histiocytosis haematophagicBlood and lymphatic system disorders
Acute myocardial infarctionCardiac disorders
Aortic valve incompetenceCardiac disorders
Atrial fibrillationCardiac disorders
Cardiogenic shockCardiac disorders
Other adverse events (79 terms — click to expand)

ReactionSystemEculizumab/EculizumabPlacebo/EculizumabEculizumab (Combined Total)
HeadacheNervous system disorders
NasopharyngitisInfections and infestations
DiarrhoeaGastrointestinal disorders
Upper respiratory tract infectionInfections and infestations
ArthralgiaMusculoskeletal and connective tissue disorders
NauseaGastrointestinal disorders
CoughRespiratory, thoracic and mediastinal disorders
InfluenzaInfections and infestations
Urinary tract infectionInfections and infestations
Pain in extremityMusculoskeletal and connective tissue disorders
Myasthenia gravisNervous system disorders
FatigueGeneral disorders
FallInjury, poisoning and procedural complications
PyrexiaGeneral disorders
BronchitisInfections and infestations
ContusionInjury, poisoning and procedural complications
Back painMusculoskeletal and connective tissue disorders
MyalgiaMusculoskeletal and connective tissue disorders
Oropharyngeal painRespiratory, thoracic and mediastinal disorders
GastroenteritisInfections and infestations
SinusitisInfections and infestations
Infusion related reactionInjury, poisoning and procedural complications
Muscle spasmsMusculoskeletal and connective tissue disorders
DepressionPsychiatric disorders
Abdominal discomfortGastrointestinal disorders
VomitingGastrointestinal disorders
Seasonal allergyImmune system disorders
Gastroenteritis viralInfections and infestations
PneumoniaInfections and infestations
CataractEye disorders
Neck painMusculoskeletal and connective tissue disorders
DizzinessNervous system disorders
InsomniaPsychiatric disorders
AnaemiaBlood and lymphatic system disorders
Abdominal pain upperGastrointestinal disorders
ToothacheGastrointestinal disorders
CellulitisInfections and infestations
AnxietyPsychiatric disorders
PruritusSkin and subcutaneous tissue disorders
VertigoEar and labyrinth disorders

Most-reported serious reactions: Myasthenia gravis, Pneumonia, Myasthenia gravis crisis, Anaemia, Pyrexia, Gastroenteritis, Sepsis, Headache.

Data from ClinicalTrials.gov NCT02301624 adverse events section.

Sponsor's own description

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).

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. The renaissance of complement therapeutics.
    Ricklin D, Mastellos DC, Reis ES, Lambris JD. · · 2018 · cited 305× · PMID 29199277 · DOI 10.1038/nrneph.2017.156
  2. Long-term safety and efficacy of eculizumab in generalized myasthenia gravis.
    Muppidi S, Utsugisawa K, Benatar M, Murai H, et al · · 2019 · cited 178× · PMID 30767274 · DOI 10.1002/mus.26447
  3. When myasthenia gravis is deemed refractory: clinical signposts and treatment strategies.
    Mantegazza R, Antozzi C. · · 2018 · cited 150× · PMID 29403543 · DOI 10.1177/1756285617749134
  4. Utilization of MG-ADL in myasthenia gravis clinical research and care.
    Muppidi S, Silvestri NJ, Tan R, Riggs K, et al · · 2022 · cited 66× · PMID 34989427 · DOI 10.1002/mus.27476
  5. 'Minimal symptom expression' in patients with acetylcholine receptor antibody-positive refractory generalized myasthenia gravis treated with eculizumab.
    Vissing J, Jacob S, Fujita KP, O'Brien F, et al · · 2020 · cited 66× · PMID 32189108 · DOI 10.1007/s00415-020-09770-y
  6. Post-intervention Status in Patients With Refractory Myasthenia Gravis Treated With Eculizumab During REGAIN and Its Open-Label Extension.
    Mantegazza R, Wolfe GI, Muppidi S, Wiendl H, et al · · 2021 · cited 54× · PMID 33229455 · DOI 10.1212/wnl.0000000000011207
  7. Complement Inhibitor Therapy for Myasthenia Gravis.
    Albazli K, Kaminski HJ, Howard JF. · · 2020 · cited 53× · PMID 32582144 · DOI 10.3389/fimmu.2020.00917
  8. Long-term efficacy of eculizumab in refractory generalized myasthenia gravis: responder analyses.
    Howard JF, Karam C, Yountz M, O'Brien FL, et al · · 2021 · cited 45× · PMID 34043280 · DOI 10.1002/acn3.51376

Verify or expand the search:

Other trials of Eculizumab

Trials testing the same drug.

Other Alexion Pharmaceuticals, Inc. trials

Trials by the same sponsor.

Verify against primary sources

Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02301624.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing