Long-term Viltolarsen for Duchenne Muscular Dystrophy
What You Need to Know Before You Apply
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.
Do I need to stop my current medications to join the trial?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.
Is Viltolarsen safe for humans?
Viltolarsen has been approved for use in treating Duchenne muscular dystrophy in Japan and the United States, indicating it has passed safety evaluations in clinical trials. While specific safety data is not detailed in the provided research, its approval suggests it is generally considered safe for human use in the context of this condition.12345
How is the drug Viltolarsen unique in treating Duchenne muscular dystrophy?
Viltolarsen is unique because it is designed to skip exon 53 of the DMD gene, allowing the production of a shorter but partially functional dystrophin protein, which can help improve muscle function in patients with specific genetic mutations. This drug is specifically targeted for about 8-10% of Duchenne muscular dystrophy patients who have mutations amenable to exon 53 skipping.12367
What data supports the effectiveness of the drug Viltolarsen for treating Duchenne Muscular Dystrophy?
Viltolarsen has shown significant improvements in muscle function in clinical trials conducted in Japan, Canada, and the United States, leading to its approval for medical use in these countries. It works by skipping exon 53 of the DMD gene, allowing the production of a partially functional dystrophin protein, which can help restore muscle function in about 8-10% of patients with specific genetic mutations.23468
Are You a Good Fit for This Trial?
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
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive viltolarsen intravenously once weekly for up to 120 months
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Viltolarsen
Find a Clinic Near You
Who Is Running the Clinical Trial?
NS Pharma, Inc.
Lead Sponsor