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Exon Skipping Agent
Viltolarsen for Duchenne Muscular Dystrophy
Phase 3
Waitlist Available
Research Sponsored by NS Pharma, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline to 48 weeks of treatment
Awards & highlights
Study Summary
This trial is testing a new drug for Duchenne muscular dystrophy patients who are eligible for exon 53 skipping.
Eligible Conditions
- Duchenne Muscular Dystrophy
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ baseline to 48 weeks of treatment
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline to 48 weeks of treatment
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
TTSTAND
Secondary outcome measures
6MWT
Hand-held dynamometer
NSAA
+2 moreTrial Design
2Treatment groups
Experimental Treatment
Placebo Group
Group I: ViltolarsenExperimental Treatment1 Intervention
Patients amenable to exon 53 skipping will receive viltolarsen intravenous (IV) infusions, weekly, at 80 mg/kg for up to 48 weeks.
Group II: PlaceboPlacebo Group1 Intervention
Patients amenable to exon 53 skipping will receive placebo intravenous (IV) infusions, weekly, for up to 48 weeks.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Viltolarsen
2021
Completed Phase 3
~100
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Who is running the clinical trial?
NS Pharma, Inc.Lead Sponsor
13 Previous Clinical Trials
497 Total Patients Enrolled
Nippon Shinyaku Co., Ltd.Industry Sponsor
12 Previous Clinical Trials
524 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- There may be additional requirements for participation in the study.There may be additional reasons why you might not be able to participate in the study.You have an allergy or strong reaction to the study drug or any of its ingredients.You have symptoms of heart muscle problems.You have received any type of gene therapy in the past.
Research Study Groups:
This trial has the following groups:- Group 1: Viltolarsen
- 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.
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