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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
- Patient is male with clinical signs compatible with DMD; and
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline to 48 weeks of treatment
Awards & highlights

Study Summary

This trial is testing a new drug for Duchenne Muscular Dystrophy. The drug is given intravenously once a week for 48 weeks. The trial is open to patients who are 8 years or older, and who can walk or not walk.

Eligible Conditions
  • Duchenne Muscular Dystrophy

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
You are a male with symptoms that match Duchenne muscular dystrophy (DMD).

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

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
Brooke scale
Forced Vital Capacity (FVC)
Forced expiratory volume in 1 second (FEV1)
+5 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 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
554 Total Patients Enrolled
Nippon Shinyaku Co., Ltd.Industry Sponsor
12 Previous Clinical Trials
581 Total Patients Enrolled

Frequently Asked Questions

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

What assessments have been made to determine the safety of Viltolarsen for human consumption?

"The safety profile of Viltolarsen was scored a 2. This is because despite some evidence supporting its security, no data has yet been found to prove the drug's effectiveness at this time."

Answered by AI

Is this a groundbreaking research endeavor?

"Currently, 4 ongoing clinical trials with Viltolarsen are occuring in 8 cities and 20 countries. The first trial involving this drug was conducted by NS Pharma, Inc. back in 2020; it had 74 participants and finished its Phase 3 process of medication approval. Since then, three further investigations have concluded their course."

Answered by AI

Is there presently an enrollment process for this clinical experiment?

"Clinicaltrials.gov confirms that this medical trial is presently not enrolling any patients, as the last update was made on June 30th 2022. Despite this, there are 90 other research projects actively seeking volunteers at present."

Answered by AI

How many participants are currently engaged in this clinical trial?

"Unfortunately, this research trial is no longer open to new participants. It was posted on the 1st of July 2021 and concluded at the end of June 2022. However, there are still 86 trials recruiting individuals with becker muscular dystrophy and 4 studies for Viltolarsen actively interviewing potential volunteers."

Answered by AI

Has Viltolarsen been tested in prior laboratory experiments?

"Currently, four clinical trials are exploring Viltolarsen with two of the studies in Phase 3. While largely held in Rome, Oregon, there is a total of 70 trial sites across the world looking into potential applications for this treatment."

Answered by AI

Who else is applying?

What portion of applicants met pre-screening criteria?
Did not meet criteria
How many prior treatments have patients received?
1

Why did patients apply to this trial?

My contact number 9642641281 my son DMD.
PatientReceived 2+ prior treatments
~5 spots leftby Mar 2025