Viltolarsen for Duchenne Muscular Dystrophy

Not currently recruiting at 41 trial locations

Overseen By Trial Info

Age: Any Age

Sex: Male

Trial Phase: Phase 3

Sponsor: NS Pharma, Inc.

Must not be taking: Investigational drugs

Disqualifiers: Adverse event, others

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

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Compensation: Varies

Number of Visits: Unknown

Duration: 96 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests viltolarsen, a treatment for Duchenne muscular dystrophy (DMD), a condition that causes muscle weakness and loss. The goal is to determine if viltolarsen is safe and effective when administered weekly for up to 96 weeks. It targets boys who have completed a previous study involving viltolarsen or a placebo and can adhere to the trial schedule. As a Phase 3 trial, this represents the final step before FDA approval, providing an opportunity to contribute to a potentially groundbreaking treatment.

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 trial team or your doctor.

Is there any evidence suggesting that viltolarsen is likely to be safe for humans?

Research has shown that viltolarsen has been carefully studied for safety in boys with Duchenne muscular dystrophy (DMD). Studies have found that viltolarsen is generally safe. In one study, boys taking viltolarsen demonstrated better movement abilities compared to those who did not receive the treatment. This suggests that the treatment is not only effective but also safe over time.

Another study followed patients for more than two years and found that viltolarsen was well-tolerated, with most patients not experiencing serious side effects. The treatment is administered as weekly intravenous (IV) infusions, which many patients managed without major problems.

Overall, the evidence suggests that viltolarsen is a safe option for treating boys with DMD who can benefit from exon 53 skipping, a specific type of genetic therapy. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for Duchenne Muscular Dystrophy, which often focus on managing symptoms or slowing progression, Viltolarsen targets the genetic root of the disease. It works by skipping exon 53 during the production of dystrophin, a crucial protein that patients with this condition lack. This targeted approach offers the potential to significantly increase dystrophin levels, which could lead to better muscle function and a slower disease progression. Researchers are excited about Viltolarsen because it represents a more precise and potentially more effective way to address the underlying cause of Duchenne Muscular Dystrophy.

What evidence suggests that viltolarsen might be an effective treatment for Duchenne Muscular Dystrophy?

Research has shown that viltolarsen holds promise for treating Duchenne muscular dystrophy (DMD) in patients who can benefit from exon 53 skipping. Studies have found that this treatment significantly increases dystrophin production to about 6% of normal levels. Long-term studies, lasting over four years, have demonstrated that viltolarsen is safe and effective. Patients experienced improved muscle function, making it a potential new option for managing DMD. In this trial, participants will receive weekly intravenous infusions of viltolarsen, which previous trials have shown to be well-tolerated. ^{1 2 6 7 8}

Are You a Good Fit for This Trial?

This trial is for boys who can walk (ambulant) and have Duchenne Muscular Dystrophy (DMD). They must have finished a previous 48-week study of viltolarsen or placebo. Participants need consent from parents or guardians, and they should be able to follow the study schedule and procedures.

Inclusion Criteria

Patient has completed the NS-065/NCNP-01-301 study

Patient and parent(s)/guardian(s) are willing and able to comply with scheduled visits, investigational product (IP) administration plan, and study procedures

Patient's parent(s) or legal guardian(s) has (have) provided written informed consent and Health Insurance Portability and Accountability Act authorization, where applicable, prior to any study-related procedures; patients will be asked to give written or verbal assent according to local requirements

Exclusion Criteria

Patient took any other investigational drug(s) during or after completion of Study NS-065/NCNP-01-301

Patient is judged by the investigator and/or the sponsor not to be appropriate to participate in the extension study for any reason

Patient had an adverse event in Study NS-065/NCNP-01-301 that, in the opinion of the investigator and/or the sponsor, precludes safe use of viltolarsen for the patient in this study

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive viltolarsen intravenous (IV) infusions weekly at 80 mg/kg for up to 96 weeks

96 weeks

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 is testing the safety and effectiveness of a drug called Viltolarsen in boys with DMD. It's an extension of a prior study where participants received either Viltolarsen or a placebo for 48 weeks.

How Is the Trial Designed?

Group I: Viltolarsen Experimental Treatment 1 Intervention

Patients amenable to exon 53 skipping will receive viltolarsen intravenous (IV) infusions, weekly, at 80 mg/kg for up to 96 weeks.

Who Is Running the Clinical Trial?

NS Pharma, Inc.

Lead Sponsor

Trials

14

Recruited

460+

Nippon Shinyaku Co., Ltd.

Industry Sponsor

Trials

14

Recruited

500+

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. nspharma.com nspharma.com/wp-content/uploads/2024/08/MDA_2023_4-year_data_poster_2March2023_FINAL.pdf

Efficacy and safety of viltolarsen in boys with Duchenne ... treatment with viltolarsen in participants with DMD amenable to exon 53 skipping resulted in significant increases in dystrophin production (~6% of normal) at a ...

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. mdaconference.org mdaconference.org/abstract-library/results-of-a-4-year-viltolarsen-extension-study-of-functional-and-safety-outcomes/

Results of a 4 Year Viltolarsen Extension Study ... Conclusions: The study outcome of better motor function vs historical controls, and favorable safety profile, are demonstrated in this longest exon 53 skipping ...

7. jamanetwork.com jamanetwork.com/journals/jamaneurology/fullarticle/2766519

Safety, Tolerability, and Efficacy of Viltolarsen in Boys With ... This phase 2 randomized clinical trial evaluates the safety, tolerability, and efficacy of viltolarsen in boys with Duchenne muscular ...

8. clinicaltrials.gov clinicaltrials.gov/study/NCT04060199

NCT04060199 | Study to Assess the Efficacy and Safety of ... This is a Phase 3 randomized, double-blind, placebo-controlled, multi-center study to assess the efficacy and safety of Viltolarsen in ambulant boys with ...