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Exon Skipping Agent

Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)

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, week 13, 25, 37, 49
Awards & highlights
Pivotal Trial

Summary

This trial is testing Viltolarsen, an IV medication given regularly, in boys with Duchenne muscular dystrophy who have specific genetic mutations. The medication helps the body skip over a faulty gene part to improve muscle function.

Eligible Conditions
  • Duchenne Muscular Dystrophy

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline, week 13, 25, 37, 49
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, week 13, 25, 37, 49 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Change From Baseline in Time to Stand (TTSTAND) Velocity
Secondary study objectives
6MWT
Hand-held dynamometer
NSAA
+2 more

Side effects data

From 2023 Phase 3 trial • 77 Patients • NCT04060199
32%
Cough
32%
COVID-19
26%
Pyrexia
26%
Upper respiratory tract infection
24%
Nasopharyngitis
21%
Diarrhea
16%
Rhinorrhea
16%
Rhinitis
13%
Fall
11%
Vomiting
11%
Abdominal pain
8%
Blood triglycerides increased
8%
Fatigue
8%
Gastroenteritis
8%
Pain in extremity
8%
Ear pain
8%
Rhinitis allergic
8%
Urine abnormality
8%
Eczema
8%
Headache
8%
Tachycardia
5%
Protein urine present
5%
Contusion
5%
Pharyngitis
5%
Abdominal pain upper
5%
Influenza-like illness
5%
Urine protein:creatinine ratio increased
5%
Urinary tract infection
5%
Hematuria
5%
Dermatitis allergic
3%
Back pain
3%
Hypercholesterolemia
3%
Influenza
3%
Rash
3%
Constipation
3%
Bronchitis
3%
Nausea
3%
Blood cholesterol increased
3%
Adverse drug reaction
100%
80%
60%
40%
20%
0%
Study treatment Arm
Placebo
Viltolarsen

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

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
384 Total Patients Enrolled
Nippon Shinyaku Co., Ltd.Industry Sponsor
13 Previous Clinical Trials
419 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
~14 spots leftby Dec 2025
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