The purpose of the study is to assess the efficacy and safety of ravulizumab for the treatment of adult participants with ALS.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
382
Single loading dose via intravenous infusion, followed by regular maintenance dosing, based on weight.
Single loading dose via intravenous infusion, followed by regular maintenance dosing, based on weight.
Change From Baseline In Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Total Score
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.
Time frame: Baseline, Week 50
Time To Ventilator Assistance-free Survival
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.
Time frame: Up to Week 50
Change From Baseline In Percent Predicted Slow Vital Capacity
Slow vital capacity measures slow and gradual expulsion of air from the lungs using a spirometer.
Time frame: Baseline, Week 50
Number of Participants With Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events, and TEAEs Leading To Study Drug Discontinuation
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.
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Barrow Neurological Institute
Phoenix, Arizona, United States
Neuromuscular Research Center and Clinic
Phoenix, Arizona, United States
HonorHealth Research Institute
Scottsdale, Arizona, United States
Loma Linda University Medical Center
Loma Linda, California, United States
University of Southern California
Los Angeles, California, United States
University of California-Irvine
Orange, California, United States
Stanford University Medical Center
Palo Alto, California, United States
University of California San Diego Medical Center
San Diego, California, United States
Norris MDA/ALS Center
San Francisco, California, United States
University of California San Francisco Medical Center
San Francisco, California, United States
...and 85 more locations
Time frame: Baseline up to Week 156
Change From Baseline In Muscle Strength As Assessed By Handheld Dynamometry
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.
Time frame: Baseline, Week 50
Change From Baseline In Serum Neurofilament Light Chain
Time frame: Baseline, Week 50
Change From Baseline in Serum Ravulizumab Concentration Over the Study Duration
Time frame: Baseline, Predose at Week 50
Change From Baseline in Serum Free Complement Component 5 (C5) Concentration Over the Study Duration
Time frame: Baseline, Predose at Week 50
Number of Participants With Positive Antidrug Antibodies (ADAs) to ALXN1210
Blood samples were collected to evaluate antibody response through development of ADAs.
Time frame: Week 50