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Antisense Oligonucleotide
ION363 for ALS
Phase 3
Recruiting
Research Sponsored by Ionis Pharmaceuticals, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Participants in Cohort A must be 12 - 65 years of age with signs or symptoms consistent with an ALS disease. If 30 to 65 years of age, have an ALSFRS-R pre-study slope ≥ 0.4 points per month
Cohort B must be > 30 years of age, with signs or symptoms consistent with an ALS disease process and have an ALSFRS-R pre-study slope < 0.4 points per month
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to day 505 in part 1
Awards & highlights
Study Summary
This trial is testing a new drug for people with FUS-ALS mutations to see if it improves clinical function and survival.
Who is the study for?
This trial is for ALS patients with FUS mutations. Adults over 30 and children aged 12-65 can join if they meet certain disease progression rates and breathing capacity criteria. Participants must have stable medication use, a caregiver to report on their condition, and no recent participation in other trials or treatments like gene therapy.Check my eligibility
What is being tested?
The study tests ION363's effect on ALS symptoms and survival compared to a placebo. It aims to understand how the drug works in the body (pharmacokinetics) and its impact on the disease process (pharmacodynamics).See study design
What are the potential side effects?
While specific side effects of ION363 are not listed here, similar drugs may cause injection site reactions, flu-like symptoms, potential liver issues, or changes in blood counts.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am 12-65 years old with ALS symptoms, and if I'm 30-65, my ALS is progressing quickly.
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I am over 30, show ALS symptoms, and my ALS condition is progressing slowly.
Select...
My test shows a FUS gene mutation from a certified lab.
Select...
My lung function, when sitting, is at least half of what's expected for my age, sex, and height.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to day 505 in part 1
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to day 505 in part 1
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Change from Baseline (Day 1) through Study Day 505 in Part 1 in functional impairment
Secondary outcome measures
Change from Baseline in Amyotrophic Lateral Sclerosis Specific Quality of Life - Revised (ALSSQOL-R) Score to Day 505 in Part 1
Change from Baseline in FUS Concentration in Cerebrospinal Fluid (CSF) to Day 505
Change from Baseline in Handheld Dynamometry (HHD) to Day 505 in Part 1
+4 moreTrial Design
2Treatment groups
Experimental Treatment
Placebo Group
Group I: ION363Experimental Treatment1 Intervention
ION363 will be administered by lumbar intrathecal (IT) bolus injection every 12 weeks, with an additional loading dose at 4 weeks, over a 60-week double-blind treatment period in Part 1; every 12 weeks for 84 weeks in the open-label extension treatment period (Part 2), with an additional loading dose administered 4 weeks after the first dose. Patients may continue to receive open-label ION363 every 12 weeks in Part 3 for up to 3 additional years or until ION363 becomes commercially available in the patient's country or until the Sponsor discontinues the development program, whichever occurs earlier.
Group II: PlaceboPlacebo Group1 Intervention
Placebo will be administered by lumbar IT bolus injection every12 weeks, with an additional loading dose at 4 weeks, over a 60-week double-blind treatment period (Part 1).
Find a Location
Who is running the clinical trial?
Ionis Pharmaceuticals, Inc.Lead Sponsor
146 Previous Clinical Trials
15,187 Total Patients Enrolled
2 Trials studying Amyotrophic Lateral Sclerosis
209 Patients Enrolled for Amyotrophic Lateral Sclerosis
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- 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.
Research Study Groups:
This trial has the following groups:- Group 1: ION363
- Group 2: Placebo
Awards:
This trial has 1 awards, including:- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Timeline:
This trial has the following timeline:- 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
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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."
Answered by AI
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."
Answered by AI
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."
Answered by AI
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."
Answered by AI
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."
Answered by AI
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