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

Sponsor

NS Pharma, Inc. (Industry)

Overall Status

Approved for marketing

CT.gov ID

NCT04337112

Collaborator

(none)

Study Details

Study Description

Brief 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.

Condition or Disease	Intervention/Treatment	Phase
Muscular Dystrophy, Duchenne DMD	Drug: viltolarsen

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 Design

Study Type:

Expanded Access

Official Title:

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

Outcome Measures

Primary Outcome Measures

Eligibility Criteria

Criteria

Ages Eligible for Study:

3 Years to 12 Years

Sexes Eligible for Study:

Male

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.