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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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline to 96 weeks of treatment

Awards & highlights

Study 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.Check my eligibility

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.See study design

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.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline to 96 weeks of treatment

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Number of participants with treatment related Adverse Events as assessed by CTCAE v4.03

Secondary outcome measures

Muscle Strength Measured by Hand-Held Dynamometer

North Star Ambulatory Assessment (NSAA)

Six-minute Walk Test (6MWT)

+3 more

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

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

NS Pharma, Inc.Lead Sponsor

13 Previous Clinical Trials

500 Total Patients Enrolled

Nippon Shinyaku Co., Ltd.Industry Sponsor

12 Previous Clinical Trials

527 Total Patients Enrolled

Frequently Asked Questions

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

Is the enrollment process for this study currently ongoing?

"As per clinicaltrials.gov, this specific research is not currently open for enrollment. The trial was initially listed on April 13th, 2021 and last modified on February 13th, 2024. Nonetheless, there are numerous other trials seeking participants at present - a total of 98 to be precise."

Answered by AI

What are the safety considerations for patients taking Viltolarsen?

"The safety evaluation conducted by our team at Power rated Viltolarsen a 3 due to it being in Phase 3 of the trial. This indicates existing efficacy data and numerous rounds of safety data."

Answered by AI

Are there several medical centers in the city conducting this research project?

"The current phase of this medical investigation is ongoing at 33 locations. Noteworthy sites include Westmead, Tomsk, and Moscow among other unspecified areas. Opting for a facility in close proximity to your residence can help reduce the burden of travel if you decide to partake in the trial."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

2021. "Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04768062.

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