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

NCT04337112 | Unknown

• 1 other identifier: VILT-501
• Study Type: expanded_access
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Muscular Dystrophy, Duchenne; DMD

Keywords

Muscular Dystrophies; Muscular Dystrophy, Duchenne; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked

Interventions

viltolarsen DRUG

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

Also known as: NS-065/NCNP-01

Eligibility Criteria

• Age: 3 Years - 12 Years
• Sex: male
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• NS Pharma, Inc.: lead

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne; Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked

Interventions

viltolarsen

Condition Hierarchy (Ancestors)

Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 2, 2020
• First Posted: April 7, 2020
• Last Updated: August 18, 2020

Record last verified: 2020-08