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Antisense Oligonucleotide

Long-term Viltolarsen for Duchenne Muscular Dystrophy

Phase 4
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 its related protein induction after completing the NS-065/NCNP-01-202 study.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline to up to 120 months of treatment
Awards & highlights
No Placebo-Only Group
Drug Has Already Been Approved
Pivotal Trial

Summary

This trial will study the long-term effects of Viltolarsen, given intravenously once a week, in boys with Duchenne Muscular Dystrophy who have completed another study.

Who is the study for?
This trial is for boys with Duchenne Muscular Dystrophy who completed the NS-065/NCNP-01-202 study. They must be able to follow the study plan and have consent from a parent or guardian. Boys with allergies to Viltolarsen, severe behavioral issues, other medical conditions that could affect safety, or those taking investigational drugs post-study are excluded.
What is being tested?
The trial tests long-term use of Viltolarsen given intravenously once weekly over 10 years in boys with Duchenne Muscular Dystrophy. It's designed to see how they do on this drug after finishing an earlier study (NS-065/NCNP-01-202).
What are the potential side effects?
While specific side effects for Viltolarsen in this context aren't listed here, common ones may include injection site reactions, kidney damage, skin changes, and potential allergic reactions.

Eligibility Criteria

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I received treatment aimed at increasing dystrophin or related proteins after completing the NS-065/NCNP-01-202 study.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline to up to 120 months of treatment
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to up to 120 months 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
Change in Performance of Upper Limb (PUL)
Loss of Ambulation (LOA)

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.
Drug Has Already Been Approved
The FDA has already approved this drug, and is just seeking more data.
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: Non-interventional (United States) / Low-interventional (Canada) : ViltolarsenExperimental Treatment1 Intervention
Patients will receive viltolarsen (recommended dose 80mg/kg/week) during a treatment period up to 120 months.
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
452 Total Patients Enrolled

Media Library

Viltolarsen (Antisense Oligonucleotide) Clinical Trial Eligibility Overview. Trial Name: NCT04687020 — Phase 4
Duchenne Muscular Dystrophy Research Study Groups: Non-interventional (United States) / Low-interventional (Canada) : Viltolarsen
Duchenne Muscular Dystrophy Clinical Trial 2023: Viltolarsen Highlights & Side Effects. Trial Name: NCT04687020 — Phase 4
Viltolarsen (Antisense Oligonucleotide) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04687020 — Phase 4
~6 spots leftby Sep 2032
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