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

August 14, 2020 updated by: NS Pharma, Inc.

The Expanded Access Use of Viltolarsen for the Treatment of Duchenne Muscular Dystrophy (DMD) Amenable to Exon 53 Skipping

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.

Study Overview

Status

Approved for marketing

Conditions

Intervention / Treatment

Detailed Description

This expanded access program is designed to provide access to viltolarsen in patients with DMD with confirmed mutation(s) in the dystrophin gene amenable to skipping of exon 53, who in the opinion and clinical judgement of the treating physician, would benefit from treatment with viltolarsen.

Study Type

Expanded Access

Expanded Access Type

  • Individual Patients

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

3 years to 12 years (CHILD)

Accepts Healthy Volunteers

N/A

Genders Eligible for Study

Male

Description

Inclusion Criteria:

  • Male ≥ 3 and ≤ 12 years of age
  • Clinical signs compatible with DMD
  • Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 53 to restore the dystrophin messenger ribonucleic acid (mRNA) reading frame
  • Able to walk independently without assistive device
  • Not able to participate in a Phase 3 trial

Exclusion Criteria:

  • Chronic systemic fungal or viral infections
  • An acute illness within 4 weeks prior to the first dose of viltolarsen
  • Symptomatic cardiomyopathy
  • Patient has a previous or ongoing medical condition, medical history, physical findings, or laboratory abnormality that could affect participant safety in the opinion of the treating physician
  • Surgery within the 3 months prior to the first anticipated administration of viltolarsen and in the opinion of the treating physician would impact weekly treatment schedule
  • Positive test results for hepatitis B antigen, hepatitis C antibody, or human immunodeficiency virus (HIV) antibody at screening
  • Currently taking any other investigational drug or has taken any other investigational drug within 3 months prior to the first dose of viltolarsen
  • Previously enrollment in any viltolarsen study.
  • Currently taking any other exon skipping agent or has taken any other exon skipping agent within 2 weeks prior to the first dose of viltolarsen (would need to be discontinued in order to be eligible)
  • Any gene therapy for DMD
  • Inadequate renal function as defined by a serum cystatin C > 1.5 x upper limit of normal (ULN). If the value is > 1.5 x ULN then the measurement can be repeated once. If repeat measurement is still > 1.5 x ULN then the patient should be excluded.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

NS Pharma, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

April 2, 2020

First Submitted That Met QC Criteria

April 2, 2020

First Posted (ACTUAL)

April 7, 2020

Study Record Updates

Last Update Posted (ACTUAL)

August 18, 2020

Last Update Submitted That Met QC Criteria

August 14, 2020

Last Verified

August 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VILT-501

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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