Phase 3

ALXN1210-ALS-308 trial

Industry trial

This phase 3 trial aims to study the safety and efficacy of the drug ‘ravulizumab’ in people living with ALS. To register for this study, you should contact your local ALS centre or treating physician.

Study design

The ALXN1210-ALS-308 trial is sponsored by Alexion Pharmaceuticals and is a randomised, double-blind, placebo-controlled trial. This means that the effect of ravulizumab is tested by comparing a group of patients that receive ravulizumab to a group of patients receiving placebo. Participants will be randomly assigned to receive ravulizumab or placebo. A placebo is something that looks exactly the same as the study medicine but does not have any active medicine in it. Participants are twice as likely to be assigned to ravulizumab treatment versus placebo.The study has a randomized controlled period of 50 weeks and during this time, the participant and the research team do not know who is receiving placebo or ravulizumab.  After the randomized controlled period, participants can enter the Open-label Extension Period of the study and receive ravulizumab.

Ravulizumab and placebo will be administered via an injection into the bloodstream (intravenously).

Key Inclusion Criteria

The study intends to recruit 354 adults with either sporadic or familial ALS. Key eligibility criteria for people with ALS to participate, include:

  • Disease onset within the prior 36 months
  • Documented meningococcal vaccination not more than 3 years prior to, or at the time of, initiating study treatment
  • Slow vital capacity (SVC), a measure of respiratory function, of at least 65% predicted at screening
  • If the participant is taking riluzole, he/she must be on a stable dose for 30 days
  • If the participant is taking edaravone, he/she must have been on a stable dose for 60 days
  • Body weight at least 40 kilograms at screening.
  • Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies

The ALXN1210-ALS-308 trial runs in ALS centres around the globe, including several TRICALS centres. For further information, please refer to:  https://clinicaltrials.gov/ct2/show/NCT04248465.