Purpose

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

Conditions

Eligibility

Eligible Ages
Over 18 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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 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

Phase
Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Ravulizumab
Participants will receive ravulizumab for the duration of the study.
  • Biological: Ravulizumab
    Single loading dose via intravenous infusion, followed by regular maintenance dosing, based on weight.
    Other names:
    • ALXN1210
    • Ultomiris
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.
  • Drug: Placebo
    Single loading dose via intravenous infusion, followed by regular maintenance dosing, based on weight.
  • Biological: Ravulizumab
    Single loading dose via intravenous infusion, followed by regular maintenance dosing, based on weight.
    Other names:
    • ALXN1210
    • Ultomiris

More Details

Status
Terminated
Sponsor
Alexion Pharmaceuticals, Inc.

Study Contact

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.