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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
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)
Secondary ID [1] 0 0
2019-004619-30
Secondary ID [2] 0 0
ALXN1210-ALS-308
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Amyotrophic Lateral Sclerosis 0 0
ALS 0 0
Condition category
Condition code
Neurological 0 0 0 0
Neurodegenerative diseases
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
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.
Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change From Baseline In Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Total Score
Timepoint [1] 0 0
Baseline, Week 50
Secondary outcome [1] 0 0
Time To Ventilator Assistance-free Survival
Timepoint [1] 0 0
Up to Week 50
Secondary outcome [2] 0 0
Change From Baseline In Percent Predicted Slow Vital Capacity
Timepoint [2] 0 0
Baseline, Week 50
Secondary outcome [3] 0 0
Number of Participants With Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events, and TEAEs Leading To Study Drug Discontinuation
Timepoint [3] 0 0
Baseline up to Week 156
Secondary outcome [4] 0 0
Change From Baseline In Muscle Strength As Assessed By Handheld Dynamometry
Timepoint [4] 0 0
Baseline, Week 50
Secondary outcome [5] 0 0
Change From Baseline In Serum Neurofilament Light Chain
Timepoint [5] 0 0
Baseline, Week 50
Secondary outcome [6] 0 0
Change From Baseline in Serum Ravulizumab Concentration Over the Study Duration
Timepoint [6] 0 0
Baseline, Predose at Week 50
Secondary outcome [7] 0 0
Change From Baseline in Serum Free Complement Component 5 (C5) Concentration Over the Study Duration
Timepoint [7] 0 0
Baseline, Predose at Week 50
Secondary outcome [8] 0 0
Number of Participants With Positive Antidrug Antibodies (ADAs) to ALXN1210
Timepoint [8] 0 0
Week 50

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.

Key
Minimum age
18 Years
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. 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
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
Blinded (masking used)
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
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
30/03/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
17/10/2021
Sample size
Target
Accrual to date
Final
382
Recruitment in Australia
Recruitment state(s)
NSW,QLD,WA
Recruitment hospital [1] 0 0
Brain and Mind Centre - Camperdown
Recruitment hospital [2] 0 0
Westmead Hospital - Westmead
Recruitment hospital [3] 0 0
Royal Brisbane and Women's Hospital - Herston
Recruitment hospital [4] 0 0
Perron Institute for Neurological and Translational Science - Nedlands
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
2145 - Westmead
Recruitment postcode(s) [3] 0 0
4029 - Herston
Recruitment postcode(s) [4] 0 0
6009 - Nedlands
Recruitment outside Australia
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United States of America
State/province [1] 0 0
Arizona
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California
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Colorado
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Illinois
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Kansas
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Kentucky
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Maryland
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Massachusetts
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Ohio
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Tennessee
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Vermont
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Leuven
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Bayern
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Dublin
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Haifa
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Israel
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Jerusalem
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Israel
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Milano
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Pisa
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Torino
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Utrecht
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Greater London
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United Kingdom
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West Midlands

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

Ethics approval
Ethics application status

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.
Trial website
https://clinicaltrials.gov/study/NCT04248465
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/NCT04248465