An Efficacy and Safety Study of Ravulizumab in ALS Participants

Alexion Pharmaceuticals

Status and phase: Terminated; Phase 3

Conditions: ALS; Amyotrophic Lateral Sclerosis

Treatments: Biological: Ravulizumab; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT04248465

2019-004619-30 (EudraCT Number)

ALXN1210-ALS-308

Details and patient eligibility

About

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

Enrollment: 382 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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382 participants in 2 patient groups, including a placebo group

Ravulizumab

Experimental group

Description:

Participants will receive ravulizumab for the duration of the study.

Treatment:

Biological: Ravulizumab

Placebo

Placebo Comparator group

Description:

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:

Drug: Placebo

Biological: Ravulizumab