Your session is about to expire
← Back to Search
ION363 for ALS
Study Summary
This trial is testing a new drug for people with FUS-ALS mutations to see if it improves clinical function and survival.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowTimeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Trial Design
Find a Location
Who is running the clinical trial?
Media Library
- I have never had gene therapy, cell transplantation, or experimental brain surgery.I haven't taken any experimental drugs or used new medical devices recently.My blood pressure is not higher than 160/100 mm Hg.I have not been treated with oligonucleotide or siRNA, except for COVID-19 vaccines.I have a condition where fluid builds up in my brain.I need a machine to help me breathe almost all day, every day.I am 12-65 years old with ALS symptoms, and if I'm 30-65, my ALS is progressing quickly.I do not have brain or spinal conditions that would affect a spinal tap.I am over 30, show ALS symptoms, and my ALS condition is progressing slowly.I haven't had cancer, except for certain skin cancers or cervical cancer in situ, in the last year.I have HIV, or untreated Hepatitis C, or Hepatitis B not managed with specific treatment.I have been on a stable dose of edaravone and/or riluzole for at least 28 days and can continue it during the study.I have no major health issues found in my medical exams.My test shows a FUS gene mutation from a certified lab.I have an ALS mutation, but it's not FUS.My lung function, when sitting, is at least half of what's expected for my age, sex, and height.
- Group 1: ION363
- Group 2: Placebo
- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
To whom does this clinical trial apply?
"To qualify for this Amyotrophic Lateral Sclerosis study, patients must be aged 12-65. There is a total of 77 open slots for participants."
Is this clinical trial prominent in Canadian hospitals?
"There are 10 medical centres currently running this trial. Some notable locations include University of Utah in Salt Lake City, The Ohio State University Wexner Medical Center in Columbus, and Stanford University Medical Center in Palo Alto."
Are there still opportunities for people to sign up for this experiment?
"The clinicaltrials.gov website verifies that this study is currently recruiting patients. This research was originally posted on June 14th, 2021 and has been updated as recently as October 23rd, 2022. There are 77 positions open for participants at 10 different locations."
Does this study have an age limit? If so, what is it?
"The age bracket for potential participants in this study are those that are 12 years or older, and younger than 65 years old."
What are the FDA's thoughts on ION363?
"ION363 received a score of 3 for safety. This is due to the fact that it is a Phase 3 trial, signifying that efficacy has been supported by some data and multiple rounds have provided evidence for its safety."
Share this study with friends
Copy Link
Messenger