A Safety and Efficacy Study of ARGX-119 in Adult Patients With Amyotrophic Lateral Sclerosis (ALS)

To be considered for this study, individuals typically needed to be between 18 and 80 years old. They also needed to have received a clear diagnosis of ALS, whether it runs in their family or appeared without a clear family link. There were also specific requirements about their overall health and how their lungs were functioning, measured by something called slow vital capacity, which needed to be at least 60% of what is expected for their age and size.

Certain things would have prevented someone from joining the study. For instance, if they used a breathing machine (noninvasive ventilation) for more than 10 hours a day, or had a tube in their windpipe (tracheostomy) to help them breathe, they would not have been able to participate. Also, if they had ever received certain types of gene or cell treatments for ALS, or were pregnant, breastfeeding, or planning to become pregnant during the study, they would not have been eligible.

Taking part in this study would have involved several visits over approximately 100 weeks (about two years). Initially, participants would have been given either ARGX-119 at one of three different dose levels or a placebo (a dummy medicine). After a certain period, all participants would then have received the active ARGX-119 medicine at a standard dose. During these visits, doctors and nurses would have carried out various assessments to check on your general health, how your ALS was progressing, and how well the treatment was tolerated. This would have included checks on your muscle function and breathing. Even though the study has stopped, follow-up visits are still happening for those who participated to ensure their wellbeing.