ARGX-119
ARGX-119 is an antibody (protein) that binds to another protein (referred to as MuSK) on muscle cells that helps receive signals from nerve cells and maintain the muscle-nerve connection. In some muscle control diseases, such as ALS, the links between nerve cells and muscle cells are disrupted. When this happens, the muscle cells do not receive all the signals from the nerve cells. This can cause weakness and tiredness. ARGX-119 may help maintain the links between nerves and muscles and improve the signalling between them. This has the potential to improve muscle strength and reduce tiredness.
ARGX-119 will be administered through an intravenous infusion.
Study design
The study is double-blinded, randomized and placebo-controlled.
In this study, there will be two groups of participants. One group will receive the medicine being studied and the other group will receive a placebo. The placebo is a substance that looks like ARGX-119, but is not active. In the study, three different concentrations of the drug will be tested, to determine what dose of the drug is best in terms of effectiveness and tolerability.
Each participant will be assigned by chance through a computer system to receive ARGX-119 or the placebo. There is a 1 in 4 chance you will receive the placebo and a 3 in 4 chance you will receive ARGX-119. If you will receive ARGX-119, you will receive 1 of 3 possible doses.
Double-blinded means that neither you nor the study doctor or the study staff will know which treatment you are receiving.
In total, participation in the study will last up to approximately 2 years. The study is split into different periods:
Screening: During the screening period, you will be checked to see if you are eligible to join the study.
Double-blinded treatment period: After the screening, and if you are found eligible to participate in the study, you’ll enter the double-blinded treatment period. During this period, you will receive either a placebo or one of the three concentration ARGX-119, as explained above. During the first 4 weeks, you will receive the study drug once every other week. After that and for the rest of the “double-blinded treatment period”, you will receive the study drug about once every 4 weeks.
Active treatment extension period: After the double-blinded treatment period, all participants will enter the active treatment extension period. During the active treatment extension period, all participants will receive a fixed dose of ARGX-119. Participants who already received ARGX-119 during the double-blinded treatment period will be given a short 4-week period where they are given the placebo, before continuing with the study drug.
Safety-follow-up period: After the extension period, your health will be monitored during the safety-follow-up period. Participants will not receive any study drug during this period.
MScan – electrophysiological assessment
During the study, participants undergo an electrophysiological muscle scan (MScan). The MScan is an electrophysiological method to measure your muscles response after being stimulated. During the assessment different muscles are activated with small electric shocks. This procedure is non-invasive, meaning that it does not require inserting an instrument through or under the skin or into a body opening. The assessment is always performed by a licensed professional. Access the video below for a demonstration of the assessment.
Can I participate in the study?
The main criteria to participate in this study are:
- Age between 18-80 years old
- Diagnosis of ALS
Note: This list is not exhaustive, but these are the main criteria.
Registration and more information
The study aims to enroll 60 patients across some European countries and Canada. Currently, the study is active in the Netherlands.
If you are interested in participating in the reALiSe study and would like to see if you fit the main study criteria, please contact one of the participating TRICALS centres below (contact details can be found by clicking on ‘read more’). When contacting the centre of your choice by email, please provide your full name, your date of birth and the date of onset of symptoms. The centre can provide you with more information and answer any questions you may have. If you experience difficulty contacting a centre, you may reach out directly to the TRICALS operational team at info@tricals.org for support in this process. More information about the study can be found at: Study Details | A Safety and Efficacy Study of ARGX-119 in Adult Patients With Amyotrophic Lateral Sclerosis (ALS) | ClinicalTrials.gov
