9 Participants Needed

Long-term Viltolarsen for Duchenne Muscular Dystrophy

Recruiting at 4 trial locations
Ti
Overseen ByTrial info
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial

Trial Summary

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.

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.12345

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.13467

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.12368

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.

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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Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive viltolarsen intravenously once weekly for up to 120 months

120 months
Weekly visits (in-person or at home)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4-8 weeks

Treatment Details

Interventions

  • Viltolarsen
Trial Overview The 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.

Find a Clinic Near You

Who Is Running the Clinical Trial?

NS Pharma, Inc.

Lead Sponsor

Trials
14
Recruited
460+

Findings from Research

In a phase 3 study involving 79 patients aged 7-16 with Duchenne muscular dystrophy, eteplirsen treatment for 96 weeks resulted in a significant increase in dystrophin production (7-fold) and exon skipping (18.7-fold), indicating its efficacy in addressing the underlying cause of the disease.
The study also demonstrated a favorable safety profile, with most adverse events being mild to moderate and unrelated to the treatment, while showing a notable slowing of disease progression compared to untreated controls.
Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial.McDonald, CM., Shieh, PB., Abdel-Hamid, HZ., et al.[2022]
Golodirsen was found to be well-tolerated in patients with Duchenne muscular dystrophy (DMD), with safety results consistent with previous studies in pediatric patients.
Treatment with golodirsen led to a significant increase in exon 53 skipping and a 16-fold increase in dystrophin protein expression at week 48, indicating its potential efficacy in restoring dystrophin production in muscle tissue.
Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy.Frank, DE., Schnell, FJ., Akana, C., et al.[2023]

References

Viltolarsen: First Approval. [2021]
Viltolarsen for the treatment of Duchenne muscular dystrophy. [2020]
Pharmacological Profile of Viltolarsen for the Treatment of Duchenne Muscular Dystrophy: A Japanese Experience. [2021]
Viltolarsen: From Preclinical Studies to FDA Approval. [2022]
Long term treatment with ataluren-the Swedish experience. [2021]
Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial. [2022]
Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy. [2023]
Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study. [2023]
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