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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT04248465
Registration number
NCT04248465
Ethics application status
Date submitted
27/01/2020
Date registered
30/01/2020
Date last updated
10/01/2023
Titles & IDs
Public title
An Efficacy and Safety Study of Ravulizumab in ALS Participants
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Scientific title
A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Ravulizumab in Patients With Amyotrophic Lateral Sclerosis (ALS)
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Secondary ID [1]
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2019-004619-30
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Secondary ID [2]
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ALXN1210-ALS-308
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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:
Amyotrophic Lateral Sclerosis
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ALS
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Condition category
Condition code
Neurological
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Neurodegenerative diseases
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Placebo
Treatment: Other - Ravulizumab
Experimental: Ravulizumab - Participants will receive ravulizumab for the duration of the study.
Placebo comparator: Placebo - Participants will receive placebo during the 50-week Randomized Controlled Period of the study, after which they will enter the Open-label Extension Period of the study and switch to receive ravulizumab.
Treatment: Drugs: Placebo
Single loading dose via intravenous infusion, followed by regular maintenance dosing, based on weight.
Treatment: Other: Ravulizumab
Single loading dose via intravenous infusion, followed by regular maintenance dosing, based on weight.
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Intervention code [1]
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Treatment: Drugs
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Intervention code [2]
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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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Change From Baseline In Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Total Score
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Assessment method [1]
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The ALSFRS-Revised is a validated instrument for evaluating the levels of the functional status of participants with amyotrophic lateral sclerosis (ALS) in 4 areas, including bulbar, gross motor activity, fine motor activity, and respiratory functions. The scale included 12 functional items and each item is rated on a 0 to 4 scale, with a maximum total score of 48. A higher score indicated greater retention of function. Baseline was defined as last non-missing value on or before first study drug administration.
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Timepoint [1]
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Baseline, Week 50
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Secondary outcome [1]
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Time To Ventilator Assistance-free Survival
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Assessment method [1]
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Ventilation Assistance-Free Survival (VAFS) is a composite endpoint of survival and severe and irreversible respiratory decline. The use of VAFS allowed for the collection of survival data that was not impacted by survival prolongation from noninvasive or permanent ventilatory interventions which could prolong life without impacting underlying disease progression.
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Timepoint [1]
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Up to Week 50
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Secondary outcome [2]
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Change From Baseline In Percent Predicted Slow Vital Capacity
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Assessment method [2]
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Slow vital capacity measures slow and gradual expulsion of air from the lungs using a spirometer.
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Timepoint [2]
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Baseline, Week 50
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Secondary outcome [3]
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Number of Participants With Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events, and TEAEs Leading To Study Drug Discontinuation
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Assessment method [3]
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An adverse event (AE) was defined as any unfavorable and unintended sign (for example, including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal product or procedure, whether or not considered related to the medicinal product or procedure, which occurred during the course of the clinical study. TEAEs were defined as AEs that occurred on or after the date and time of study drug administration, or those that first occurred before dosing but worsened in frequency or severity after study drug administration. 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 [3]
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Baseline up to Week 156
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Secondary outcome [4]
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Change From Baseline In Muscle Strength As Assessed By Handheld Dynamometry
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Assessment method [4]
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Handheld dynamometry (HHD) is a procedure for quantitative strength testing. Muscle strength testing was performed on prespecified muscles in the upper and lower extremities bilaterally and the force measurements were recorded. Force of measurement is reported in megascores (lower, upper, total). The total megascore is defined as the average of the non-missing ratios over baseline for all the muscles involved. The megascore at baseline is always 100. The range of a potential megascore can not be determined in advance. A megascore \>100 indicates more strength compared to baseline.
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Timepoint [4]
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Baseline, Week 50
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Secondary outcome [5]
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Change From Baseline In Serum Neurofilament Light Chain
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Assessment method [5]
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Timepoint [5]
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Baseline, Week 50
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Secondary outcome [6]
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Change From Baseline in Serum Ravulizumab Concentration Over the Study Duration
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Assessment method [6]
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Timepoint [6]
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Baseline, Predose at Week 50
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Secondary outcome [7]
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Change From Baseline in Serum Free Complement Component 5 (C5) Concentration Over the Study Duration
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Assessment method [7]
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Baseline, Predose at Week 50
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Secondary outcome [8]
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Number of Participants With Positive Antidrug Antibodies (ADAs) to ALXN1210
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Assessment method [8]
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Blood samples were collected to evaluate antibody response through development of ADAs.
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Timepoint [8]
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Week 50
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Eligibility
Key inclusion criteria
Key
1. A diagnosis of sporadic or familial ALS, defined by the El Escorial criteria (possible, laboratory-supported probable, probable, or definite ALS).
2. ALS onset = 36 months from Screening.
3. Documented meningococcal vaccination not more than 3 years prior to, or at the time of, initiating study treatment.
4. Upright slow vital capacity = 65% predicted at Screening.
5. If on riluzole, participant must be on a stable dose for 30 days; if on edaravone, participant must be on a stable dose for 60 days (2 cycles).
6. Body weight = 40 kilograms at Screening.
7. 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
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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. Dependence on invasive or non-invasive mechanical ventilation.
4. Previously or currently treated with a complement inhibitor.
5. Exposure to an investigational drug or device within 30 days of Screening or 5 half lives of the study drug, whichever is greater.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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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
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Parallel
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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
Stopped early
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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
30/03/2020
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Date of last participant enrolment
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Actual
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Date of last data collection
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Actual
17/10/2021
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Sample size
Target
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Accrual to date
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Final
382
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Recruitment in Australia
Recruitment state(s)
NSW,QLD,WA
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Recruitment hospital [1]
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Brain and Mind Centre - Camperdown
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Recruitment hospital [2]
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Westmead Hospital - Westmead
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Recruitment hospital [3]
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Royal Brisbane and Women's Hospital - Herston
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Recruitment hospital [4]
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Perron Institute for Neurological and Translational Science - Nedlands
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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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2145 - Westmead
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Recruitment postcode(s) [3]
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4029 - Herston
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Recruitment postcode(s) [4]
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6009 - Nedlands
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Recruitment outside Australia
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West Midlands
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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 the study is to assess the efficacy and safety of ravulizumab for the treatment of adult participants with ALS.
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Trial website
https://clinicaltrials.gov/study/NCT04248465
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
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No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/65/NCT04248465/Prot_002.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/65/NCT04248465/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT04248465
Download to PDF