Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Trial Review
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT04201262
Registration number
NCT04201262
Ethics application status
Date submitted
11/12/2019
Date registered
17/12/2019
Date last updated
9/08/2023
Titles & IDs
Public title
An Efficacy and Safety Study of Ravulizumab in Adult Participants With NMOSD
Query!
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)
Query!
Secondary ID [1]
0
0
CHAMPION-NMO-307
Query!
Secondary ID [2]
0
0
ALXN1210-NMO-307
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Neuromyelitis Optica
0
0
Query!
Neuromyelitis Optica Spectrum Disorder
0
0
Query!
Condition category
Condition code
Inflammatory and Immune System
0
0
0
0
Query!
Autoimmune diseases
Query!
Neurological
0
0
0
0
Query!
Other neurological disorders
Query!
Eye
0
0
0
0
Query!
Diseases / disorders of the eye
Query!
Intervention/exposure
Study type
Interventional
Query!
Description of intervention(s) / exposure
Other interventions - 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.
Other interventions: 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.
Query!
Intervention code [1]
0
0
Other interventions
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Number of Participants With an Adjudicated On-trial Relapse in the Primary Treatment Period
Query!
Assessment method [1]
0
0
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.
Query!
Timepoint [1]
0
0
Baseline up to 2.25 years (end of the Primary Treatment Period)
Query!
Secondary outcome [1]
0
0
Adjudicated On-trial Annualized Relapse Rate (ARR) in the Primary Treatment Period
Query!
Assessment method [1]
0
0
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.
Query!
Timepoint [1]
0
0
Baseline up to 2.25 years (end of the Primary Treatment Period)
Query!
Secondary outcome [2]
0
0
Number of Participants With Clinically Important Change From Baseline in Hauser Ambulation Index (HAI) Score at the End of Primary Treatment Period
Query!
Assessment method [2]
0
0
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.
Query!
Timepoint [2]
0
0
Baseline up to 2.25 years (end of the Primary Treatment Period)
Query!
Secondary outcome [3]
0
0
Change From Baseline in European Quality of Life Health 5-dimension Questionnaire (EQ-5D) Index Score at the End of Primary Treatment Period
Query!
Assessment method [3]
0
0
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.
Query!
Timepoint [3]
0
0
Baseline, up to 2.25 years (end of the Primary Treatment Period)
Query!
Secondary outcome [4]
0
0
Change From Baseline in EQ-5D Visual Analog Scale (VAS) Score at the End of Primary Treatment Period
Query!
Assessment method [4]
0
0
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.
Query!
Timepoint [4]
0
0
Baseline, up to 2.25 years (end of the Primary Treatment Period)
Query!
Secondary outcome [5]
0
0
Number of Participants With Clinically Important Worsening From Baseline in Expanded Disability Status Scale (EDSS) Score at the End of Primary Treatment Period
Query!
Assessment method [5]
0
0
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.
Query!
Timepoint [5]
0
0
Baseline up to 2.25 years (end of the Primary Treatment Period)
Query!
Secondary outcome [6]
0
0
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
Query!
Assessment method [6]
0
0
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.
Query!
Timepoint [6]
0
0
Baseline up to 2.25 years (end of the Primary Treatment Period)
Query!
Secondary outcome [7]
0
0
Serum Ravulizumab Concentration
Query!
Assessment method [7]
0
0
Query!
Timepoint [7]
0
0
Predose and end of infusion (EOI) at Week 26
Query!
Secondary outcome [8]
0
0
Change From Baseline in Serum Free C5 Concentration at Week 26 and 50
Query!
Assessment method [8]
0
0
Query!
Timepoint [8]
0
0
Baseline, Week 26 (Predose and EOI)
Query!
Secondary outcome [9]
0
0
Number of Participants With Anti-drug Antibodies (ADAs) During the Primary Treatment Period
Query!
Assessment method [9]
0
0
Query!
Timepoint [9]
0
0
Baseline, Weeks 26, 50, 82, and 106
Query!
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.
Query!
Minimum age
18
Years
Query!
Query!
Maximum age
No limit
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
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.
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
N/A
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Open (masking not used)
Query!
Who is / are masked / blinded?
Query!
Query!
Query!
Query!
Intervention assignment
Single group
Query!
Other design features
Query!
Phase
Phase 3
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Active, not recruiting
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
13/12/2019
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
31/07/2024
Query!
Actual
Query!
Sample size
Target
Query!
Accrual to date
Query!
Final
58
Query!
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Query!
Recruitment hospital [1]
0
0
Clinical Study Site - Camperdown
Query!
Recruitment hospital [2]
0
0
Clinical Study Site - New Lambton Heights
Query!
Recruitment hospital [3]
0
0
Clinical Study Site - Parkville
Query!
Recruitment hospital [4]
0
0
Clinical Study Site - Fitzroy
Query!
Recruitment postcode(s) [1]
0
0
2050 - Camperdown
Query!
Recruitment postcode(s) [2]
0
0
2305 - New Lambton Heights
Query!
Recruitment postcode(s) [3]
0
0
3050 - Parkville
Query!
Recruitment postcode(s) [4]
0
0
3065 - Fitzroy
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
Alabama
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
California
Query!
Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
Colorado
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
District of Columbia
Query!
Country [5]
0
0
United States of America
Query!
State/province [5]
0
0
Florida
Query!
Country [6]
0
0
United States of America
Query!
State/province [6]
0
0
Georgia
Query!
Country [7]
0
0
United States of America
Query!
State/province [7]
0
0
Illinois
Query!
Country [8]
0
0
United States of America
Query!
State/province [8]
0
0
Kansas
Query!
Country [9]
0
0
United States of America
Query!
State/province [9]
0
0
Kentucky
Query!
Country [10]
0
0
United States of America
Query!
State/province [10]
0
0
Massachusetts
Query!
Country [11]
0
0
United States of America
Query!
State/province [11]
0
0
Minnesota
Query!
Country [12]
0
0
United States of America
Query!
State/province [12]
0
0
Mississippi
Query!
Country [13]
0
0
United States of America
Query!
State/province [13]
0
0
Missouri
Query!
Country [14]
0
0
United States of America
Query!
State/province [14]
0
0
North Carolina
Query!
Country [15]
0
0
United States of America
Query!
State/province [15]
0
0
Ohio
Query!
Country [16]
0
0
United States of America
Query!
State/province [16]
0
0
Texas
Query!
Country [17]
0
0
Austria
Query!
State/province [17]
0
0
Wien
Query!
Country [18]
0
0
Canada
Query!
State/province [18]
0
0
British Columbia
Query!
Country [19]
0
0
Canada
Query!
State/province [19]
0
0
Edmonton
Query!
Country [20]
0
0
Denmark
Query!
State/province [20]
0
0
Aarhus
Query!
Country [21]
0
0
France
Query!
State/province [21]
0
0
Bron Cedex
Query!
Country [22]
0
0
France
Query!
State/province [22]
0
0
Montpellier
Query!
Country [23]
0
0
France
Query!
State/province [23]
0
0
Nîmes
Query!
Country [24]
0
0
France
Query!
State/province [24]
0
0
Strasbourg
Query!
Country [25]
0
0
Germany
Query!
State/province [25]
0
0
Berlin
Query!
Country [26]
0
0
Germany
Query!
State/province [26]
0
0
Dresden
Query!
Country [27]
0
0
Germany
Query!
State/province [27]
0
0
Leipzig
Query!
Country [28]
0
0
Germany
Query!
State/province [28]
0
0
Muenchen
Query!
Country [29]
0
0
Italy
Query!
State/province [29]
0
0
Cefalu
Query!
Country [30]
0
0
Italy
Query!
State/province [30]
0
0
Gallarate
Query!
Country [31]
0
0
Italy
Query!
State/province [31]
0
0
Napoli
Query!
Country [32]
0
0
Italy
Query!
State/province [32]
0
0
Orbassano
Query!
Country [33]
0
0
Italy
Query!
State/province [33]
0
0
Roma
Query!
Country [34]
0
0
Japan
Query!
State/province [34]
0
0
Chiba
Query!
Country [35]
0
0
Japan
Query!
State/province [35]
0
0
Fukuoka-shi
Query!
Country [36]
0
0
Japan
Query!
State/province [36]
0
0
Kawagoe-shi
Query!
Country [37]
0
0
Japan
Query!
State/province [37]
0
0
Niigata-shi
Query!
Country [38]
0
0
Japan
Query!
State/province [38]
0
0
Sendai-shi
Query!
Country [39]
0
0
Japan
Query!
State/province [39]
0
0
Sendai
Query!
Country [40]
0
0
Korea, Republic of
Query!
State/province [40]
0
0
Goyang-si
Query!
Country [41]
0
0
Korea, Republic of
Query!
State/province [41]
0
0
Seoul
Query!
Country [42]
0
0
Poland
Query!
State/province [42]
0
0
Katowice
Query!
Country [43]
0
0
Poland
Query!
State/province [43]
0
0
Lodz
Query!
Country [44]
0
0
Spain
Query!
State/province [44]
0
0
Barakaldo
Query!
Country [45]
0
0
Spain
Query!
State/province [45]
0
0
Barcelona
Query!
Country [46]
0
0
Spain
Query!
State/province [46]
0
0
Madrid
Query!
Country [47]
0
0
Spain
Query!
State/province [47]
0
0
Malaga
Query!
Country [48]
0
0
Spain
Query!
State/province [48]
0
0
Valencia
Query!
Country [49]
0
0
United Kingdom
Query!
State/province [49]
0
0
Liverpool
Query!
Country [50]
0
0
United Kingdom
Query!
State/province [50]
0
0
Oxford
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Query!
Name
Alexion Pharmaceuticals, Inc.
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
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.
Query!
Trial website
https://clinicaltrials.gov/ct2/show/NCT04201262
Query!
Trial related presentations / publications
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT04201262
Download to PDF