Long-term Viltolarsen for Duchenne Muscular Dystrophy

Recruiting in Palo Alto (17 mi)

+4 other locations

Age: Any Age

Sex: Male

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 4

Waitlist Available

Sponsor: NS Pharma, Inc.

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?

The VILT-502 study is Non-interventional Study(United States)/Low-intervention Clinical Trial (Canada) of Viltolarsen administered intravenously once weekly for 10 years to boys with DMD who complete the NS-065/NCNP-01-202 study.

Research Team

Eligibility Criteria

This trial is for boys with Duchenne Muscular Dystrophy who completed the NS-065/NCNP-01-202 study. They must be able to follow the study plan and have consent from a parent or guardian. Boys with allergies to Viltolarsen, severe behavioral issues, other medical conditions that could affect safety, or those taking investigational drugs post-study are excluded.

Inclusion Criteria

Patient and parent or legal guardian are willing and able to comply with scheduled visits, study treatment administration plan, and study procedures.

Patient, patient's parent or legal guardian have provided written informed consent/medical record release authorization prior to any extension study-specific procedures, and the patient has provided assent appropriate for his age and developmental status.

Patient completed the NS-065/NCNP-01-202 study and was judged by the investigator as appropriate to participate in the VILT-502 study.

Exclusion Criteria

Patient took any other investigational drugs after completing the NS-065/NCNP-01-202 study.

I received treatment aimed at increasing dystrophin or related proteins after completing the NS-065/NCNP-01-202 study.

Patient has an allergy or hypersensitivity to the study drug or to any of its constituents.

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Treatment Details

Interventions

Viltolarsen (Antisense Oligonucleotide)

Trial OverviewThe trial tests long-term use of Viltolarsen given intravenously once weekly over 10 years in boys with Duchenne Muscular Dystrophy. It's designed to see how they do on this drug after finishing an earlier study (NS-065/NCNP-01-202).

Participant Groups

1Treatment groups

Experimental Treatment

Group I: Non-interventional (United States) / Low-interventional (Canada) : ViltolarsenExperimental Treatment1 Intervention

Patients will receive viltolarsen (recommended dose 80mg/kg/week) during a treatment period up to 120 months.