An Efficacy and Safety Study of Ravulizumab in ALS Participants

• Status: Active, not recruiting
• Trial end date: 2024-03-01
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Alexion Pharmaceuticals
• Treatments: Ravulizumab

Criteria

Key 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.

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.