NCT04687020

Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)

• Status: Active Not Recruiting
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 9 (actual)
• Sponsor: NS Pharma, Inc. · Industry
• Sex: Male
• Age: —
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

The VILT-502 study is an open-label, single-arm study to assess the long-term safety and effectiveness of viltolarsen, an exon skipping therapy for the treatment of DMD. Patients who complete the Phase II long-term extension study and meet the additional inclusion and exclusion criteria of the present protocol will be invited to enroll. Viltolarsen will be administered through weekly IV infusions, at the study site or at home. The VILT-502 study will be conducted as a non-interventional study in the US, and as a low-intervention clinical trial in Canada where viltolarsen is not yet commercially available, owing to differences in the stage of regulatory approval in the two countries.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	Viltolarsen	Received during weekly intravenous infusions

Timeline

• Start date: 2021-06-10
• Primary completion: 2032-09-01
• Completion: 2032-10-01
• First posted: 2020-12-29
• Last updated: 2022-10-19

Locations

5 sites across 2 countries: United States, Canada

Regulatory

• FDA-regulated drug study