What is the drug thought to do in the body?
ION363 (also known as Jacifusen) is an investigational antisense medicine that is designed to reduce the production of the Fused in Sarcoma (FUS) protein which can lead to rapid, progressive loss of motor neurons. People with a change in the FUS gene can develop a rare form of ALS, referred to as FUS-ALS.
Phase 3
Current status: Open for recruitment
Primary outcome: To investigate if ION363 changes the rate of disease progression, from day 1 to day 225, measured by the ALS functional rating scale (ALSFRS-R).
The Phase 3 trial is investigating the safety and efficacy of ION363 by comparing the treatment to placebo over 61-weeks. The trial is also testing different dosages to confirm the best dose to use. An open-label extension is available at the end of the trial, where all participants in the trial will receive ION363 for 85 weeks. Find out more on the MND Clinical Studies Group website or on clinicaltrials.gov.
How can I take part in the trial?
Recruiting Sites
The trial is recruiting in the UK at the following sites:
Please contact the individual trial site, by clicking each site above, for more information on how to take part in the trial.
Trial Information
Phase: 1-3
Placebo controlled: Yes - this means that people joining the trial could be randomly assigned to receive the placebo (dummy drug).
Open-Label Extension: Yes - this means that all participants on the trial, regardless of whether they received the treatment or placebo, will have an opportunity to access the treatment once the randomised part of the trial is complete.
Key eligibility criteria: This trial is only recruiting people with MND who have a FUS gene mutation. More criteria here.
Latest News
2022
May 2022 - The protocol of the trial was changed to increase the length of the double-blind phase (from 29 weeks to 61 weeks). This is to increase the likelihood of detecting a clinically meaningful signal.
2021
Dec 2021 - King's College London was the first trial centre in Europe to recruit participants for ION363
Resources
Last updated: 16/08/2024