Viltolarsen for Duchenne Muscular Dystrophy

Recruiting at 32 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

74 Participants Needed

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 trial team or your doctor.

What data supports the effectiveness of the drug Viltolarsen for Duchenne Muscular Dystrophy?

Viltolarsen has been shown to significantly increase dystrophin levels in patients with Duchenne Muscular Dystrophy, leading to improvements in muscle function over more than four years of treatment compared to historical controls. ^{1 2 3 4 5}

Is Viltolarsen safe for humans?

Viltolarsen has been tested in clinical trials in Japan, Canada, and the United States, and it was approved for medical use in Japan in March 2020 and in the United States in August 2020. These trials showed significant improvements in muscle function, indicating that it is generally safe for use in humans with Duchenne muscular dystrophy. ^{1 2 3 4 5}

How is the drug Viltolarsen unique for treating Duchenne Muscular Dystrophy?

Viltolarsen is unique because it is designed to skip exon 53 of the DMD gene, allowing the production of a shortened but functional dystrophin protein, which can help improve muscle function in patients with specific genetic mutations. This drug is specifically effective for about 8-10% of DMD patients who have mutations amenable to exon 53 skipping. ^{1 2 3 4 5}

What is the purpose of this trial?

This is a Phase 3, multi-center, open-label extension study in ambulant boys with DMD who have completed the 48-week treatment period of either viltolarsen or placebo in Study NS-065/NCNP-01-301.

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

References

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. 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]

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

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