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Small Molecule
Zilganersen for Alexander Disease
Phase 3
Waitlist Available
Research Sponsored by Ionis Pharmaceuticals, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline to week 61
Awards & highlights
Study Summary
This trial will study if ION373 is safe and effective in treating patients with AxD by improving or stabilizing their gross motor function.
Who is the study for?
This trial is for people aged 2 to 65 with Alexander Disease (AxD), confirmed by specific brain imaging and a genetic mutation in the GFAP gene. Children under 18 need a caregiver to participate. Participants must be able to travel for study requirements but can't join if they've had recent major surgery, other experimental brain treatments, or are on another clinical trial.Check my eligibility
What is being tested?
The study tests zilganersen (ION373) against a placebo to see if it improves or stabilizes gross motor function in AxD patients. It's designed to compare the effects of this potential new treatment with no active treatment over time.See study design
What are the potential side effects?
While specific side effects of ION373 aren't listed here, common ones from similar treatments may include reactions at injection sites, flu-like symptoms, headache, fatigue, and potential liver toxicity. Each person's experience could vary.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ baseline to week 61
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline to week 61
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Percent Change from Baseline in the 10-Meter Walk Test (10MWT)
Secondary outcome measures
Change From Baseline in 9-Hole Peg Test (9HPT) Score
Change From Baseline in Alexander Disease Patient Domain Impression of Change (AxD-PDIC) Score
Change From Baseline in Alexander Disease Patient Domain Impression of Severity (AxD-PDIS) Score
+12 moreOther outcome measures
Change From Baseline in Pediatrics Quality of Life Inventory (PedsQL) Generic Core Scales Score
Trial Design
2Treatment groups
Experimental Treatment
Placebo Group
Group I: zilganersenExperimental Treatment1 Intervention
Zilganersen will be administered by intrathecal bolus (ITB) injection once every 12 weeks through Week 49. The 60-week double-blind treatment period will be followed by the open-label and long-term extension periods, where participants will receive zilganersen by ITB injection from Week 61 to Week 229.
Group II: PlaceboPlacebo Group1 Intervention
Matching placebo will be administered by ITB injection once every 12 weeks through Week 49. It will be followed by the open-label and long-term extension periods, where participants will receive zilganersen by ITB injection from Week 61 to Week 229.
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Who is running the clinical trial?
Ionis Pharmaceuticals, Inc.Lead Sponsor
146 Previous Clinical Trials
15,209 Total Patients Enrolled
1 Trials studying Alexander Disease
100 Patients Enrolled for Alexander Disease
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- You have a brain or spinal condition that could make it unsafe for you to have a lumbar puncture.You have a blockage in the flow of fluid in your brain.You have important health issues in your past or during the physical check-up.You have a shunt or catheter in your brain to drain fluid.You have any major abnormal lab results that would make you unsuitable for the study.You have received a specific type of medication called an oligonucleotide within the last 4 months (if you received one dose) or 12 months (if you received multiple doses), unless it was a vaccine.Your symptoms and brain scan show signs of having Alexander disease.You have a confirmed genetic mutation in the GFAP gene.You are between 2 and 65 years old when you agree to take part in the study.
Research Study Groups:
This trial has the following groups:- Group 1: zilganersen
- 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.
Does this research include participants that are over 20 years old?
"Only patients aged 2 to 65 years old can qualify for this particular clinical trial. There is one study available for patients younger than 18 and one for patients who are 65 years or older."
Answered by AI
What is the geographical scope of this experiment?
"This clinical trial is based out of IONIS Investigative Site in Chicago, Illinois (which will only be recruiting children at first), McGill University Health Centre in Houston, Texas, and Mass General Hospital in Atlanta, Georgia. There are also 11 other locations."
Answered by AI
Could you please quantify the risks associated with ION373?
"There is both pre-clinical and clinical data supporting ION373's safety, thus it was given a score of 3."
Answered by AI
Does this study have any specific inclusion or exclusion criteria?
"This research is looking for 58 individuals that have Alexander disease and are between the ages of 2 and 65. The key requirements for potential participants are as follows: must have a caretaker if they are under 18 years old, have a documented genetic mutation of the GFAP gene, and be willing and able to commit to all aspects of the study including visiting the study center for procedures and measurements."
Answered by AI
Who else is applying?
What state do they live in?
Georgia
What site did they apply to?
IONIS Investigative Site
IONIS Investigative Site (Will be initially recruiting children only)
What portion of applicants met pre-screening criteria?
Did not meet criteria
Met criteria
How many prior treatments have patients received?
1
How responsive is this trial?
Average response time
- < 2 Days
Typically responds via
Email
Most responsive sites:
- IONIS Investigative Site (Will be initially recruiting children only): < 48 hours
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