In the reALiSe study the safety and efficacy of the drug ARGX-119 for the treatment of ALS is
investigated. Furthermore, the study investigates how the body and immune system reacts
to ARGX-119. The study is sponsored by argenx BV.
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 signaling 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. Watch this video 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. In Europe, TRICALS centers based in Belgium, Denmark,
France, The Netherlands and Sweden participate.
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 participating TRICALS centres (contact details can
be found on https://www.tricals.org/en/trials/realise-study-2 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: