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Antisense Oligonucleotide

Viltolarsen for Duchenne Muscular Dystrophy

Phase 2
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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline to 48 weeks of treatment
Awards & highlights
No Placebo-Only Group

Summary

This trial tests viltolarsen in DMD patients over 8 years old. It includes both those who can walk and those who cannot. The treatment helps produce dystrophin to improve muscle function and slow disease progression. Viltolarsen is a medication approved for treating DMD patients with a specific gene deletion.

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

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Number of Participants With Adverse Event of Special Interest
Number of Participants With Investigational Product-related Treatment Emergent Adverse Events
Number of Participants With Investigational Product-related Treatment Emergent Adverse Events by Highest Intervention Level Regarding Investigational Product
+6 more
Secondary study objectives
Brooke scale
Forced Vital Capacity (FVC)
Forced expiratory volume in 1 second (FEV1)
+5 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%
Non-cardiac chest pain
5%
Contusion
5%
Ligament sprain
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.

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 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
441 Total Patients Enrolled
Nippon Shinyaku Co., Ltd.Industry Sponsor
13 Previous Clinical Trials
476 Total Patients Enrolled
~5 spots leftby Nov 2025
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