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Antisense Oligonucleotide

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
Must not have
Patient had a treatment which was made for the purpose of dystrophin or dystrophin-related protein induction after completion of Study NS-065/NCNP-01-301
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline to 96 weeks of treatment
Awards & highlights
No Placebo-Only Group
Pivotal Trial

Summary

This trial is for boys with Duchenne Muscular Dystrophy who have finished a 48-week treatment period in a previous study.

Who is the study for?
This trial is for boys who can walk (ambulant) and have Duchenne Muscular Dystrophy (DMD). They must have finished a previous 48-week study of viltolarsen or placebo. Participants need consent from parents or guardians, and they should be able to follow the study schedule and procedures.
What is being tested?
The trial is testing the safety and effectiveness of a drug called Viltolarsen in boys with DMD. It's an extension of a prior study where participants received either Viltolarsen or a placebo for 48 weeks.
What are the potential side effects?
Possible side effects are not specified here, but generally, treatments for DMD may cause potential reactions at the injection site, kidney problems, changes in mood or behavior, nausea, vomiting, and heart issues.

Eligibility Criteria

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I have received treatment aimed at increasing dystrophin after completing a specific study.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline to 96 weeks of treatment
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to 96 weeks of treatment for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Secondary study objectives
Muscle Strength Measured by Hand-Held Dynamometer
North Star Ambulatory Assessment (NSAA)
Six-minute Walk Test (6MWT)
+3 more

Side effects data

From 2023 Phase 2 trial • 20 Patients • NCT04956289
30%
COVID-19
25%
Diarrhoea
20%
Haematuria
20%
Headache
15%
Nasopharyngitis
15%
Upper respiratory tract infection
10%
Pain in extremity
10%
Abdominal pain upper
10%
Food poisoning
10%
Pyrexia
10%
Joint injury
10%
Influenza
10%
Rhinitis
5%
Hypertension
5%
Gait inability
5%
Myalgia
5%
Toothache
5%
Tension headache
5%
Blister
5%
Rash
5%
Vomiting
5%
Pharyngitis
5%
Drug hypersensitivity
5%
Nephrolithiasis
5%
Urine cytology abnormal
5%
Ligament sprain
5%
Non-cardiac chest pain
5%
Contusion
5%
Back pain
5%
Angina pectoris
5%
Otitis media
5%
Tachycardia
5%
Protein urine
5%
Nasal congestion
5%
Oropharyngeal pain
100%
80%
60%
40%
20%
0%
Study treatment Arm
Viltolarsen 80mg/kg

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups
Experimental Treatment
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 96 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
387 Total Patients Enrolled
Nippon Shinyaku Co., Ltd.Industry Sponsor
13 Previous Clinical Trials
422 Total Patients Enrolled
~14 spots leftby Oct 2025
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