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The Expanded Access Use of Viltolarsen in Duchenne Muscular Dystrophy With Confirmed Exon 53 Amenable Mutation

NCT04337112 · Status: APPROVED_FOR_MARKETING · Type: EXPANDED_ACCESS

Last updated 2020-08-18

No results posted yet for this study

Summary

This is an open label expanded access program for boys, 3 to 12 years old, for the treatment of Duchenne muscular dystrophy (DMD) with confirmed mutation(s) in the dystrophin gene that is amenable to skipping of exon 53.

Conditions

  • Muscular Dystrophy, Duchenne
  • DMD

Interventions

DRUG

viltolarsen

Intravenous (IV) infusions, weekly, at 80mg/kg, once weekly (approximately every 7 days).

Sponsors & Collaborators

  • NS Pharma, Inc.

    lead INDUSTRY

Eligibility

Min Age
3 Years
Max Age
12 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04337112 on ClinicalTrials.gov