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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)


Brief Summary:

The purpose of the study is to assess the efficacy and safety of ravulizumab for the
treatment of adult participants with ALS.


Inclusion Criteria:

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.

Exclusion Criteria:

1. History of Neisseria meningitidis infection.

2. Human immunodeficiency virus (HIV) infection (evidenced by HIV 1 or HIV 2 antibody

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.


  • Loma Linda University Medical Center, Loma Linda, California, United States, 92354
  • University of Southern California, Los Angeles, California, United States, 90033
  • University of California-Irvine, Orange, California, United States, 92868
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