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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 weeks
Treatment ~ Varies
Follow Up ~baseline to 48 weeks of treatment
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to 48 weeks of treatment for reporting.

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 more

Trial 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

Find a Location

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

Viltolarsen (Exon Skipping Agent) Clinical Trial Eligibility Overview. Trial Name: NCT04060199 — Phase 3
Duchenne Muscular Dystrophy Research Study Groups: Viltolarsen, Placebo
Duchenne Muscular Dystrophy Clinical Trial 2023: Viltolarsen Highlights & Side Effects. Trial Name: NCT04060199 — Phase 3
Viltolarsen (Exon Skipping Agent) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04060199 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are patients currently being accepted into this trial?

"Yes, this information is accurate. The most recent update to the study was on May 23rd, 2022 and it is still recruiting patients."

Answered by AI

What are the possible dangers associated with taking Viltolarsen?

"Viltolarsen is estimated to be a safe drug, as it has reached Phase 3 in clinical trials. This suggests that not only is there supporting data for its efficacy, but also that there have been multiple rounds of data supporting its safety."

Answered by AI

In how many different places can patients participate in this trial?

"5 sites are enrolling patients, these being CHU de Quebec Research Centre, University of California Davis Medical Center, Alberta Children's Hospital, and 5 other locations."

Answered by AI

Does this study enroll patients that are senior citizens?

"This particular study only enrolls patients that are between the ages of 4 and 7. Out of the 118 total clinical trials, this is the 65th for patients under 18 and the 53rd for patients over 65."

Answered by AI

For whom is this clinical trial designed?

"This study is looking for 74 boys that have muscular dystrophy, duchenne. The participants must be between 4 and 7 years old. Most importantly, the applicants must meet the following requirements: Be a male ≥ 4 years and < 8 years of age, Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 53 to restore the dystrophin mRNA reading frame, Able to walk independently without assistive devices, TTSTAND < 10 seconds, Stable dose of glucocorticoid (GC) for at least 3 months prior to study entry and"

Answered by AI

Could you please list any other times Viltolarsen has been studied?

"Currently, there are 4 different studies being conducted to research Viltolarsen. Out of those 4, 2 are in Phase 3. Although the majority of Viltolarsen trials are based in Roma, Oregon, there are a total of 70 locations running these trials."

Answered by AI

How many people are being test subjects in this experiment?

"This research project needs 74 individuals that meet the necessary requirements in order to commence. The pharmaceutical company NS Pharma, Inc. will be managing the trial from different hospitals, which are located in Quebec City, California, and Sacramento, Ontario."

Answered by AI

Have similar studies been conducted in the past?

"Viltolarsen has been researched since 2020 when the first clinical trial was completed. The initial study, which was backed by NS Pharma, Inc., only included 74 patients. After the 2020 trial, Viltolarsen was approved for Phase 3 drug testing. There are currently 4 trials underway involving 20 countries and 8 cities."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)
2020. "Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04060199.
~15 spots leftby Apr 2025
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