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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT04201262
Registration number
NCT04201262
Ethics application status
Date submitted
11/12/2019
Date registered
17/12/2019
Titles & IDs
Public title
An Efficacy and Safety Study of Ravulizumab in Adult Participants With NMOSD
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Scientific title
A Phase 3, External Placebo-Controlled, Open-Label, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Patients With Neuromyelitis Optica Spectrum Disorder (NMOSD)
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Secondary ID [1]
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CHAMPION-NMO-307
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Secondary ID [2]
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ALXN1210-NMO-307
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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
Treatment: Other - Ravulizumab
Experimental: Ravulizumab - During the Primary Treatment Period, all participants will receive open-label ravulizumab via intravenous (IV) infusion starting on Day 1. The end of the Primary Treatment Period will be triggered when the last enrolled participant completes between 26 and 50 weeks in the study (depending on the number of adjudicated On-Trial Relapse observed).
After completion of the Primary Treatment Period, all participants will have the opportunity to continue receiving ravulizumab in the Long-Term Extension Period of the study. For each participant, the Long-Term Extension Period continues for up to 2 years, or until ravulizumab is approved and/or available (in accordance with country-specific regulations), whichever occurs first.
Treatment: Other: Ravulizumab
Participants will receive a weight-based loading dose of ravulizumab via IV infusion on Day 1, followed by weight-based maintenance doses on Day 15, then once every 8 weeks until end of the Long-Term Extension Period.
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Intervention code [1]
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Treatment: Other
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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 an Adjudicated On-trial Relapse in the Primary Treatment Period
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Assessment method [1]
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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. An adjudicated On-trial Relapse was defined by the protocol and positively adjudicated by the independent relapse adjudication committee.
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Timepoint [1]
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Baseline up to 2.25 years (end of the Primary Treatment Period)
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Secondary outcome [1]
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Adjudicated On-trial Annualized Relapse Rate (ARR) in the Primary Treatment Period
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Assessment method [1]
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The adjudicated On-trial ARR was computed as the total number of relapses divided by the total number of participant years in the study period. A central independent committee was used to adjudicate all On-trial Relapses as determined by the treating physician. Results reported as adjusted adjudicated On-trial ARR based on a Poisson regression centered on the mean historical ARR in the 24 months prior to screening.
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Timepoint [1]
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Baseline up to 2.25 years (end of the Primary Treatment Period)
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Secondary outcome [2]
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Number of Participants With Clinically Important Change From Baseline in Hauser Ambulation Index (HAI) Score at the End of Primary Treatment Period
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Assessment method [2]
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The HAI is a rating scale developed to assess mobility by evaluating the time and degree of assistance required to walk 25 feet. The scale ranges from 0 to 9, with 0 being the best score (asymptomatic; fully ambulatory with no assistance) and 9 being the worst (restricted to wheelchair; unable to transfer self independently). Clinically important change is conditional on the baseline value: worsening if the baseline HAI is 0 and at least 2 points increase or if the baseline HAI is \>0 and at least 1 point increase; improvement if the baseline value is at least 2 and at least 1 point decrease; and stable if baseline is 0 or 1 and a 0- or 1-point increase or decrease or baseline is at least 2 and not change.
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Timepoint [2]
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Baseline up to 2.25 years (end of the Primary Treatment Period)
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Secondary outcome [3]
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Change From Baseline in European Quality of Life Health 5-dimension Questionnaire (EQ-5D) Index Score at the End of Primary Treatment Period
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Assessment method [3]
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The EQ-5D is a generic, standardized, self-administered instrument that provides a simple, descriptive profile and a single index value for health status. It consists of 2 parts; the EQ-5D descriptive system and the EQ-5D visual analogue scale (VAS). The EQ-5D descriptive system includes 5 dimensions; mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Each dimension measured on 3 levels: "no problem" (level 1), "some problems" (level 2), "extreme problems" (level 3). The digits for 5 dimensions can be combined in a 5-digit number describing the respondent's health state. The 5 dimensional 3-level systems was converted into single index utility score that ranges from less than 0 to 1, with higher scores representing a better health status.
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Timepoint [3]
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Baseline, up to 2.25 years (end of the Primary Treatment Period)
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Secondary outcome [4]
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Change From Baseline in EQ-5D Visual Analog Scale (VAS) Score at the End of Primary Treatment Period
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Assessment method [4]
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The EQ-5D is a generic, standardized, self-administered instrument that provides a simple, descriptive profile and a single index value for health status. It consists of 2 parts; the EQ-5D descriptive system and the EQ-5D visual analogue scale (VAS). The EQ-5D VAS is an overall health state scale where the participant selects a number between 0 to 100 to describe the condition of their health, with 100 being 'The best health state you can imagine' and 0 being 'The worst health state you can imagine'. An increase in score indicates improvement.
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Timepoint [4]
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Baseline, up to 2.25 years (end of the Primary Treatment Period)
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Secondary outcome [5]
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Number of Participants With Clinically Important Worsening From Baseline in Expanded Disability Status Scale (EDSS) Score at the End of Primary Treatment Period
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Assessment method [5]
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Disease-related disability was measured by the EDSS. The EDSS is an ordinal clinical rating scale that ranges from 0 (normal neurologic examination) to 10 (death) in half-point increments. Clinically important worsening was defined as an increase in EDSS score conditional on the baseline value: If the baseline EDSS was 0 and at least 2-point increase; if the baseline is 1 to 5, and at least 1-point increase; if the baseline is \> 5 and at least 0.5 increase.
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Timepoint [5]
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Baseline up to 2.25 years (end of the Primary Treatment Period)
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Secondary outcome [6]
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Number of Participants With Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and TEAEs Leading to Study Drug Discontinuation in the Primary Treatment Period
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Assessment method [6]
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An AE was as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), important medical event or reaction. TEAEs were AEs with a start date on or after the date of the first dose of study drug. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
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Timepoint [6]
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Baseline up to 2.25 years (end of the Primary Treatment Period)
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Secondary outcome [7]
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Serum Ravulizumab Concentration
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Assessment method [7]
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Timepoint [7]
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Predose and end of infusion (EOI) at Week 26
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Secondary outcome [8]
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Change From Baseline in Serum Free C5 Concentration at Week 26 and 50
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Assessment method [8]
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Timepoint [8]
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Baseline, Week 26 (Predose and EOI)
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Secondary outcome [9]
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Number of Participants With Anti-drug Antibodies (ADAs) During the Primary Treatment Period
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Assessment method [9]
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Timepoint [9]
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Baseline, Weeks 26, 50, 82, and 106
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Eligibility
Key inclusion criteria
1. Anti-aquaporin-4 antibody-positive and a diagnosis of NMOSD as defined by the 2015 international consensus diagnostic criteria.
2. At least 1 attack or relapse in the last 12 months prior to the Screening Period.
3. Expanded Disability Status Scale score =7.
4. Participants who enter the study receiving supportive immunosuppressive therapy must be on a stable dosing regimen of adequate duration prior to Screening.
5. Body weight =40 kilograms.
6. Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
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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. History of neisseria meningitidis infection.
2. Human immunodeficiency virus (HIV) infection (evidenced by HIV-1 or HIV-2 antibody titer).
3. Previously or currently treated with a complement inhibitor.
4. Use of rituximab or mitoxantrone within 3 months prior to Screening.
5. Use of IV immunoglobulin within 3 weeks prior to Screening.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
NA
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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
Active, not recruiting
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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
13/12/2019
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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
31/07/2024
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Actual
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Sample size
Target
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Accrual to date
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Final
58
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Recruitment in Australia
Recruitment state(s)
NSW,VIC
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Recruitment hospital [1]
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Clinical Study Site - Camperdown
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Recruitment hospital [2]
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Clinical Study Site - New Lambton Heights
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Recruitment hospital [3]
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Clinical Study Site - Parkville
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Recruitment hospital [4]
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Clinical Study Site - Fitzroy
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Recruitment postcode(s) [1]
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2050 - Camperdown
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Recruitment postcode(s) [2]
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2305 - New Lambton Heights
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Recruitment postcode(s) [3]
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3050 - Parkville
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Recruitment postcode(s) [4]
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3065 - Fitzroy
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Recruitment outside Australia
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United States of America
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State/province [1]
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Alabama
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United States of America
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California
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United States of America
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Colorado
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District of Columbia
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United States of America
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Florida
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United States of America
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Georgia
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Illinois
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Kansas
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Mississippi
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Missouri
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North Carolina
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Ohio
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Austria
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Wien
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British Columbia
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Canada
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Edmonton
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Denmark
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Aarhus
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Bron Cedex
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France
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Nîmes
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Berlin
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Dresden
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Germany
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Leipzig
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Germany
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Muenchen
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Italy
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Gallarate
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Italy
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Napoli
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Italy
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Orbassano
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Italy
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Chiba
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Japan
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Sendai
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Korea, Republic of
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Goyang-si
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Korea, Republic of
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Seoul
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Poland
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Katowice
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Poland
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Lodz
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Barakaldo
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Spain
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Barcelona
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Spain
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Madrid
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Spain
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Malaga
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Spain
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Valencia
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United Kingdom
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Liverpool
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United Kingdom
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Oxford
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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 primary purpose of this study is to evaluate the efficacy and safety of ravulizumab for the treatment of adult participants with NMOSD.
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Trial website
https://clinicaltrials.gov/study/NCT04201262
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Address
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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What data in particular will be shared?
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When will data be available (start and end dates)?
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Available to whom?
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Available for what types of analyses?
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How or where can data be obtained?
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What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/62/NCT04201262/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/62/NCT04201262/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT04201262