Viltolarsen for Duchenne Muscular Dystrophy

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
Duchenne Muscular DystrophyViltolarsen - Drug
Eligibility
4 - 7
Male
What conditions do you have?
Select

Study Summary

This trial is testing a new drug for Duchenne muscular dystrophy patients who are eligible for exon 53 skipping.

Eligible Conditions
  • Duchenne Muscular Dystrophy

Treatment Effectiveness

Effectiveness Progress

2 of 3
This is further along than 85% of similar trials

Similar Trials

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Myoblast transplantation

Study Objectives

1 Primary · 5 Secondary · Reporting Duration: baseline to 48 weeks of treatment

Week 48
6MWT
Hand-held dynamometer
NSAA
TTCLIMB
TTRW
TTSTAND

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Similar Trials

1
AOC 1044-CS1 Part A - Single Dose Levels 1-5
2
Vamorolone
1
Myoblast transplantation

Trial Design

2 Treatment Groups

Viltolarsen
1 of 2
Placebo
1 of 2

Experimental Treatment

Non-Treatment Group

74 Total Participants · 2 Treatment Groups

Primary Treatment: Viltolarsen · Has Placebo Group · Phase 3

Viltolarsen
Drug
Experimental Group · 1 Intervention: Viltolarsen · Intervention Types: Drug
Placebo
Drug
PlaceboComparator Group · 1 Intervention: Placebo · Intervention Types: Drug

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: baseline to 48 weeks of treatment

Who is running the clinical trial?

NS Pharma, Inc.Lead Sponsor
10 Previous Clinical Trials
412 Total Patients Enrolled
Nippon Shinyaku Co., Ltd.Industry Sponsor
9 Previous Clinical Trials
439 Total Patients Enrolled

Eligibility Criteria

Age 4 - 7 · Male Participants · 6 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You are male and younger than 8 years of age.
You are expected to be on a stable dose of glucocorticoid for at least 3 months prior to study entry and is expected to remain on stable dose of glucocorticoid for the duration of the study.
References

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