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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT02003144
Registration number
NCT02003144
Ethics application status
Date submitted
18/11/2013
Date registered
6/12/2013
Date last updated
23/08/2022
Titles & IDs
Public title
An Open Label Extension Trial of Eculizumab in Relapsing NMO Patients
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Scientific title
A Phase III, Open-label, Extension Trial of ECU-NMO-301 to Evaluate the Safety and Efficacy of Eculizumab in Patients With Relapsing Neuromyelitis Optica (NMO)
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Secondary ID [1]
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2013-001151-12
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Secondary ID [2]
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ECU-NMO-302
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Neuromyelitis Optica
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Neuromyelitis Optica Spectrum Disorder
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Condition category
Condition code
Inflammatory and Immune System
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Autoimmune diseases
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Neurological
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Other neurological disorders
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Eye
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Diseases / disorders of the eye
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Other interventions - eculizumab
Experimental: Eculizumab - Eculizumab intravenous infusion every two weeks.
Other interventions: eculizumab
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Intervention code [1]
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Other interventions
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events
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Assessment method [1]
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An adverse event (AE) was defined as any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. Treatment-emergent adverse events (TEAEs) were defined as an AE with onset on or after the first study drug dose in Study ECU-NMO-302. A serious adverse event (SAE) was defined as an untoward medical occurrence that at any dose either results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, or is an important medical event. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module.
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Timepoint [1]
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Baseline up to end of study (up to 6.5 years)
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Primary outcome [2]
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Number of Participants With At Least 1 Post Baseline Columbia-Suicide Severity Rating Scale (C-SSRS) Assessment (Suicide-Related Thoughts or Behaviours) Abnormality
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Assessment method [2]
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The C-SSRS is a validated questionnaire to capture occurrence, severity, and frequency of suicide-related thoughts and behaviours, and has a binary response (yes/no). Suicidal Ideation: a "yes" answer to any one of 5 suicidal ideation questions: Wish to be Dead; Non-specific Active Suicidal Thoughts; Active Suicidal Ideation with Any Methods (Not Planned) without Intent to Act; Active Suicidal Ideation with Some Intent to Act, without Specific Plan; and Active Suicidal Ideation with Specific Plan and Intent. Suicidal Behaviour: a "yes" answer to any of 5 suicidal behaviour questions: Preparatory Acts or Behaviour, Aborted Attempt, Interrupted Attempt, Actual Attempt (non-fatal), and Completed Suicide. Suicidal Ideation or Behaviour: a "yes" answer to the following question: Self-injurious behaviour without suicidal intent.
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Timepoint [2]
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Baseline up to end of study (up to 6.5 years)
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Primary outcome [3]
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Number of Participants With An On-trial Relapse as Determined by The Treating Physician
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Assessment method [3]
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An On-trial Relapse was defined as a new onset of neurologic symptoms or worsening of existing neurologic symptoms with an objective change (clinical sign) on neurologic examination that persisted for more than 24 hours as confirmed by the treating physician.
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Timepoint [3]
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Baseline up to end of study (up to 6.5 years)
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Primary outcome [4]
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On-Trial Annualized Relapse Rate (ARR) as Determined by The Treating Physician
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Assessment method [4]
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The On-trial ARR was computed as the total number of relapses divided by the total number of participant years in the study period.
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Timepoint [4]
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Baseline up to end of study (up to 6.5 years)
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Secondary outcome [1]
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Change From Baseline in Expanded Disability Status Scale (EDSS) Score
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Assessment method [1]
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Disease-related disability was measured by the EDSS. The EDSS quantifies disability in 8 Functional Systems (FS) and allows neurologists to assign a Functional System Score (FSS) in each of these. The Functional Systems are pyramidal, cerebellar, brainstem, sensory, bowel and bladder, visual, cerebral, and other. The EDSS is an ordinal clinical rating scale that ranges from 0 (normal neurologic examination) to 10 (death) in half-point increments. A decrease in score indicates improvement. Baseline was defined as the last available assessment prior to the first study drug infusion in Study ECU-NMO-302.
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Timepoint [1]
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Baseline, Weeks 52, 104 and 156
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Secondary outcome [2]
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Change From Baseline in Modified Rankin Scale (mRS) Score
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Assessment method [2]
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Disease-related disability was measured by the mRS score. The mRS is a commonly used scale for measuring the degree of disability or dependence in the daily activities of people who have suffered from a neurological disability. The scale ranges from 0 (no symptoms at all) to 6 (death) in whole-point increments. A decrease in score indicates improvement. Baseline was defined as the last available assessment prior to the first study drug infusion in Study ECU-NMO-302.
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Timepoint [2]
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Baseline, Weeks 52, 104 and 156
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Secondary outcome [3]
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Change From Baseline in Hauser Ambulation Index (HAI) in Participants With Abnormal Baseline Ambulatory Function
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Assessment method [3]
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The HAI evaluates gait and was used to assess the time and effort used by the participant to walk 25 feet (8 meters). The scale ranges from 0 to 9, with 0 being the best score (asymptomatic; fully active) and 9 being the worst (restricted to wheelchair; unable to transfer self independently). A decrease in score indicates improvement. Baseline is defined as the last available assessment prior to the first study drug infusion in Study ECU-NMO-302.
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Timepoint [3]
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Baseline, Weeks 52, 104 and 156
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Secondary outcome [4]
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Change From Baseline in European Quality of Life (EuroQoL) 5-Dimension Visual Analog Scale (EQ-5D VAS) Health Status Score
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Assessment method [4]
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The EQ-5D is a generic, standardized participant self-administered health status instrument. EQ-5D general health status can also be measured by a visual analog scale (EQ-5D VAS). EQ-5D-VAS recorded the participant's self-rated health on a vertical visual analog scale (VAS) that allowed the participants to indicate their health state that ranged from 0 (worst imaginable) to 100 (best imaginable). Baseline is defined as the last available assessment prior to the first study drug infusion in Study ECU-NMO-302.
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Timepoint [4]
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Baseline, Weeks 52, 104 and 156
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Secondary outcome [5]
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Change From Baseline in Kurtzke Visual Functional System Scores (FSS) in Participants With Abnormal Baseline Visual Function
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Assessment method [5]
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The EDSS assesses multiple Kurtzke functional systems in the context of a standard neurological exam, including visual function. The visual score ranges from 0 to 6. A score of 0 implies the participant has normal visual function. Higher scores represent worse disability. Baseline is defined as the last available assessment prior to the first study drug infusion in Study EC-NMO-302.
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Timepoint [5]
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Baseline, Weeks 52, 104 and 156
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Eligibility
Key inclusion criteria
Key
1. Patient completed the ECU-NMO-301 trial
2. Patient has given written informed consent
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
1. Patients who have withdrawn from the ECU-NMO-301 trial as a result of an AE related to
trial drug
2. Female patients who are pregnant, breastfeeding, or intend to conceive during the
course of the trial
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Study design
Purpose of the study
Treatment
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Allocation to intervention
N/A
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
12/01/2015
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
12/07/2021
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Sample size
Target
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Accrual to date
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Final
119
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Recruitment in Australia
Recruitment state(s)
NSW,VIC
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Recruitment hospital [1]
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Brain and Mind Research Institute - Camperdown
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Recruitment hospital [2]
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St. Vincent's Hospital - Fitzroy
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Recruitment postcode(s) [1]
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- Camperdown
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Recruitment postcode(s) [2]
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3065 - Fitzroy
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Recruitment outside Australia
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Arizona
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Florida
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Santa Fe
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Argentina
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Ciudad Autonoma, Buenos Aires,
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Ciudad Autonoma, Buenos Aires
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Samsun
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Liverpool
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Alexion Pharmaceuticals, Inc.
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Address
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Ethics approval
Ethics application status
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Summary
Brief summary
The purpose of this study is to determine whether eculizumab long-term use is safe and
effective in patients with relapsing NMO.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT02003144
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Trial related presentations / publications
Pittock SJ, Lennon VA, McKeon A, Mandrekar J, Weinshenker BG, Lucchinetti CF, O'Toole O, Wingerchuk DM. Eculizumab in AQP4-IgG-positive relapsing neuromyelitis optica spectrum disorders: an open-label pilot study. Lancet Neurol. 2013 Jun;12(6):554-62. doi: 10.1016/S1474-4422(13)70076-0. Epub 2013 Apr 26.
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Public notes
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Contacts
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT02003144
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