Long-term Viltolarsen for Duchenne Muscular Dystrophy

Recruiting at 4 trial locations

Overseen By Trial info

Age: Any Age

Sex: Male

Trial Phase: Phase 4

Sponsor: NS Pharma, Inc.

Must not be taking: Investigational drugs

Disqualifiers: Allergy, Behavioral problems, Medical condition, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 120 months

9 Participants Needed

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. ^{1 2 3 4 5}

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. ^{1 2 3 6 7}

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. ^{2 3 4 6 8}

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

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.

Timeline for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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).

How Is the Trial Designed?

Group I: Non-interventional (United States) / Low-interventional (Canada) : Viltolarsen Experimental Treatment 1 Intervention

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

Who Is Running the Clinical Trial?

NS Pharma, Inc.

Lead Sponsor

Trials

14

Recruited

460+

Published Research Related to This Trial

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy. Frank, DE., Schnell, FJ., Akana, C., et al.[2023]

Citations

1. New Zealand pubmed.ncbi.nlm.nih.gov

Viltolarsen: First Approval. [2021]

2. Spain pubmed.ncbi.nlm.nih.gov

Viltolarsen for the treatment of Duchenne muscular dystrophy. [2020]

3. New Zealand pubmed.ncbi.nlm.nih.gov

Pharmacological Profile of Viltolarsen for the Treatment of Duchenne Muscular Dystrophy: A Japanese Experience. [2021]

4. United States pubmed.ncbi.nlm.nih.gov

Viltolarsen: From Preclinical Studies to FDA Approval. [2022]

5. England pubmed.ncbi.nlm.nih.gov

Long term treatment with ataluren-the Swedish experience. [2021]

6. Netherlands pubmed.ncbi.nlm.nih.gov

Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial. [2022]

7. United States pubmed.ncbi.nlm.nih.gov

Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy. [2023]

8. Netherlands pubmed.ncbi.nlm.nih.gov

Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study. [2023]