A Randomized Controlled Trial of Eculizumab in AQP4 Antibody-positive Participants With NMO (PREVENT Study)

Study Identifier:
ECU-NMO-301
ClinicalTrials.gov Identifier:
NCT01892345
EudraCT Identifier:
N/A
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Other

Available Documents

ProtocolStatistical Analysis Plan

Study Details

Medical Condition
  • Neuromyelitis Optica
  • Neuromyelitis Optica Spectrum Disorder
Study Drug
  • Drug: Eculizumab
  • Drug: Placebo
Date
Apr 2014 - Jul 2018
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 18+ years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Male or female participants ≥ 18 years old.
  • Diagnosis of NMO or NMOSD.
  • AQP4 antibody seropositive.
  • Historical relapse of at least 2 relapses in the last 12 months or 3 relapses in the last 24 months with at least 1 relapse in the 12 months prior to the screening.
  • Expanded Disability Status Scale score ≤ 7.
  • If a participant entered the study receiving immunosuppressive therapy (IST) for relapse prevention, the participant must have been on a stable maintenance dose of IST(s), as defined by the treating physician, prior to Screening and must have remained on that dose for the duration of the study, unless the participant experienced a relapse.
  • Female participants of childbearing potential were to have a negative pregnancy test (serum human chorionic gonadotropin). 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
  • Use of rituximab within 3 months prior to Screening.
  • Use of mitoxantrone within 3 months prior to Screening.
  • Use of intravenous immunoglobulin within 3 weeks prior to Screening.
Sex
Female & Male
Age
18+ years

Study Details

Medical Condition
  • Neuromyelitis Optica
  • Neuromyelitis Optica Spectrum Disorder
Study Drug
  • Drug: Eculizumab
  • Drug: Placebo
Date
Apr 2014 - Jul 2018
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 18+ years years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Male or female participants ≥ 18 years old.
  • Diagnosis of NMO or NMOSD.
  • AQP4 antibody seropositive.
  • Historical relapse of at least 2 relapses in the last 12 months or 3 relapses in the last 24 months with at least 1 relapse in the 12 months prior to the screening.
  • Expanded Disability Status Scale score ≤ 7.
  • If a participant entered the study receiving immunosuppressive therapy (IST) for relapse prevention, the participant must have been on a stable maintenance dose of IST(s), as defined by the treating physician, prior to Screening and must have remained on that dose for the duration of the study, unless the participant experienced a relapse.
  • Female participants of childbearing potential were to have a negative pregnancy test (serum human chorionic gonadotropin). 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
  • Use of rituximab within 3 months prior to Screening.
  • Use of mitoxantrone within 3 months prior to Screening.
  • Use of intravenous immunoglobulin within 3 weeks prior to Screening.

Protocol Summary

The objectives of this time-to-event study were to assess the efficacy and safety of eculizumab as compared with placebo in participants with neuromyelitis optica spectrum disorder (NMOSD) who were anti-aquaporin-4 (AQP4) antibody-positive.

Trial Locations

Location
Status
Location
Mayo Clinic Arizona
Scottsdale, Arizona, United States, 85259
Status
N/A
Location
The Research Center of Southern California
Carlsbad, California, United States, 92011
Status
N/A
Location
Georgetown University Hospital
Washington D.C., District of Columbia, United States, 20007
Status
N/A
Location
University of Miami McKnight Brain Institute
Miami, Florida, United States, 33136
Status
N/A
Location
Neurological Services of Orlando
Orlando, Florida, United States, 32806
Status
N/A
Location
Fort Wayne Neurological Center
Fort Wayne, Indiana, United States, 46804
Status
N/A
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