Long-term Viltolarsen for Duchenne Muscular Dystrophy

Not currently 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial examines the long-term effects of Viltolarsen, administered through weekly IVs, to assist boys with Duchenne Muscular Dystrophy (DMD), a condition that progressively weakens muscles. The main goal is to assess the safety and effectiveness of Viltolarsen over a period of up to 10 years. Boys who participated in a previous related study and do not have an allergy to the medication may qualify. As a Phase 4 trial, Viltolarsen has already received FDA approval and demonstrated effectiveness, and this research aims to understand its benefits for more patients.

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 is the safety track record for Viltolarsen?

Research has shown that viltolarsen is generally safe for people with Duchenne Muscular Dystrophy (DMD). Studies have found it to be an effective treatment for DMD. In long-term studies lasting over two years, patients using viltolarsen did not experience serious side effects. Administered once a week, the treatment has been used without major problems. The FDA has already approved viltolarsen for treating DMD, further confirming its safety for use in people. ^{1 2 3 4 5}

Why are researchers enthusiastic about this study treatment?

Researchers are excited about viltolarsen for Duchenne Muscular Dystrophy because it offers a novel approach to treating this progressive condition. Unlike other treatments that generally aim to manage symptoms or slow progression, viltolarsen works by targeting the genetic cause of the disease. Specifically, it uses exon skipping to enable the production of a functional dystrophin protein, which is crucial for muscle strength and stability. This innovative mechanism could potentially lead to more effective management and improved outcomes for individuals with Duchenne Muscular Dystrophy.

What is the effectiveness track record for Viltolarsen in treating Duchenne Muscular Dystrophy?

Research has shown that Viltolarsen helps treat Duchenne muscular dystrophy (DMD) in patients who can benefit from skipping exon 53. Studies have demonstrated significant improvement in muscle function in boys with DMD over four years. These studies confirm its safety and effectiveness when administered once a week. In this trial, participants will receive Viltolarsen to manage DMD symptoms and improve the quality of life for those eligible for this therapy. ^{3 5 6 7 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.

pubmed.ncbi.nlm.nih.gov

pubmed.ncbi.nlm.nih.gov/35634851/

Long-Term Functional Efficacy and Safety of Viltolarsen in ...

Based on these results at over 2 years, viltolarsen can be a new treatment option for patients with DMD amenable to exon 53 skipping.

2.

nature.com

nature.com/articles/s41598-024-70783-y

Safety and efficacy of viltolarsen in ambulatory ...

Efficacy and safety of viltolarsen in boys with Duchenne muscular dystrophy: Results from the phase 2, open-label, 4-year extension study. J ...

3.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC10200237/

Efficacy and Safety of Viltolarsen in Boys With Duchenne ...

Based on the results of this 4-year LTE, viltolarsen can be an important treatment strategy for DMD patients amenable to exon 53 skipping.

4.

institut-myologie.org

institut-myologie.org/en/2023/09/07/dmd-positive-results-from-viltolarsen-confirmed-over-four-years/

positive results from Viltolarsen confirmed over four years

The results of the phase II clinical trial of Viltolarsen in 16 boys with DMD aged between 4 and 9 years in May 2020 showed that it induced a significant ...

5.

sciencedirect.com

sciencedirect.com/science/article/abs/pii/S0387760424001384

Safety and efficacy of viltolarsen treatment in patients with ...

Viltolarsen was a safe and effective treatment for patients with DMD, and the findings highlighted the importance of once-weekly and uninterrupted viltolarsen ...

6.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC9398057/

Long-Term Functional Efficacy and Safety of Viltolarsen in ...

Based on these results at over 2 years, viltolarsen can be a new treatment option for patients with DMD amenable to exon 53 skipping.

7.

parentprojectmd.org

parentprojectmd.org/ns-pharma-announces-publication-of-long-term-efficacy-and-safety-data-of-viltolarsen-in-participants-with-duchenne/

NS Pharma Announces Publication of Long-Term Efficacy ...

NS Pharma, Inc. has announced the publication of long-term efficacy and safety data from the open-label extension trial of a Phase 2 study of VILTEPSO ( ...

8.

clinicaltrials.gov

clinicaltrials.gov/study/NCT04687020

NCT04687020 | Long-term Use of Viltolarsen in Boys With ...

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 ...