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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
Date last updated
23/10/2024

Titles & IDs
Public title
An Efficacy and Safety Study of Ravulizumab in Adult Participants With NMOSD
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)
Secondary ID [1] 0 0
CHAMPION-NMO-307
Secondary ID [2] 0 0
ALXN1210-NMO-307
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Neuromyelitis Optica 0 0
Neuromyelitis Optica Spectrum Disorder 0 0
Condition category
Condition code
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases
Neurological 0 0 0 0
Other neurological disorders
Eye 0 0 0 0
Diseases / disorders of the eye

Intervention/exposure
Study type
Interventional
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.
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With an Adjudicated On-trial Relapse in the Primary Treatment Period
Timepoint [1] 0 0
Baseline up to 2.25 years (end of the Primary Treatment Period)
Secondary outcome [1] 0 0
Adjudicated On-trial Annualized Relapse Rate (ARR) in the Primary Treatment Period
Timepoint [1] 0 0
Baseline up to 2.25 years (end of the Primary Treatment Period)
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
Timepoint [2] 0 0
Baseline up to 2.25 years (end of the Primary Treatment Period)
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
Timepoint [3] 0 0
Baseline, up to 2.25 years (end of the Primary Treatment Period)
Secondary outcome [4] 0 0
Change From Baseline in EQ-5D Visual Analog Scale (VAS) Score at the End of Primary Treatment Period
Timepoint [4] 0 0
Baseline, up to 2.25 years (end of the Primary Treatment Period)
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
Timepoint [5] 0 0
Baseline up to 2.25 years (end of the Primary Treatment Period)
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
Timepoint [6] 0 0
Baseline up to 2.25 years (end of the Primary Treatment Period)
Secondary outcome [7] 0 0
Serum Ravulizumab Concentration
Timepoint [7] 0 0
Predose and end of infusion (EOI) at Week 26
Secondary outcome [8] 0 0
Change From Baseline in Serum Free C5 Concentration at Week 26 and 50
Timepoint [8] 0 0
Baseline, Week 26 (Predose and EOI)
Secondary outcome [9] 0 0
Number of Participants With Anti-drug Antibodies (ADAs) During the Primary Treatment Period
Timepoint [9] 0 0
Baseline, Weeks 26, 50, 82, and 106

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.
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
10/12/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
14/02/2025
Actual
Sample size
Target
Accrual to date
Final
58
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Research Site - Camperdown
Recruitment hospital [2] 0 0
Research Site - Fitzroy
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
3065 - Fitzroy
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Colorado
Country [2] 0 0
United States of America
State/province [2] 0 0
District of Columbia
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Massachusetts
Country [5] 0 0
United States of America
State/province [5] 0 0
Minnesota
Country [6] 0 0
United States of America
State/province [6] 0 0
Mississippi
Country [7] 0 0
United States of America
State/province [7] 0 0
Missouri
Country [8] 0 0
United States of America
State/province [8] 0 0
Ohio
Country [9] 0 0
United States of America
State/province [9] 0 0
Texas
Country [10] 0 0
Austria
State/province [10] 0 0
Vienna
Country [11] 0 0
Canada
State/province [11] 0 0
British Columbia
Country [12] 0 0
Denmark
State/province [12] 0 0
Aarhus
Country [13] 0 0
France
State/province [13] 0 0
Strasbourg
Country [14] 0 0
Germany
State/province [14] 0 0
Berlin
Country [15] 0 0
Germany
State/province [15] 0 0
Leipzig
Country [16] 0 0
Germany
State/province [16] 0 0
Muenchen
Country [17] 0 0
Italy
State/province [17] 0 0
Cefalu
Country [18] 0 0
Italy
State/province [18] 0 0
Gallarate
Country [19] 0 0
Italy
State/province [19] 0 0
Napoli
Country [20] 0 0
Italy
State/province [20] 0 0
Roma
Country [21] 0 0
Italy
State/province [21] 0 0
Rome
Country [22] 0 0
Japan
State/province [22] 0 0
Chiba-shi
Country [23] 0 0
Japan
State/province [23] 0 0
Fukuoka City
Country [24] 0 0
Japan
State/province [24] 0 0
Kawagoe-shi
Country [25] 0 0
Japan
State/province [25] 0 0
Niigata-shi
Country [26] 0 0
Japan
State/province [26] 0 0
Sendai-shi
Country [27] 0 0
Korea, Republic of
State/province [27] 0 0
Goyang-si
Country [28] 0 0
Korea, Republic of
State/province [28] 0 0
Seoul
Country [29] 0 0
Poland
State/province [29] 0 0
Katowice
Country [30] 0 0
Poland
State/province [30] 0 0
Lódz
Country [31] 0 0
Spain
State/province [31] 0 0
Barcelona
Country [32] 0 0
Spain
State/province [32] 0 0
Madrid
Country [33] 0 0
Spain
State/province [33] 0 0
Malaga
Country [34] 0 0
United Kingdom
State/province [34] 0 0
Oxford

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Alexion Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

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.
Trial website
https://clinicaltrials.gov/study/NCT04201262
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT04201262