Viltolarsen for Duchenne Muscular Dystrophy

Recruiting in Palo Alto (17 mi)

+32 other locations

Age: Any Age

Sex: Male

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: NS Pharma, Inc.

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

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.

Research Team

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

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Treatment Details

Interventions

Viltolarsen (Antisense Oligonucleotide)

Trial OverviewThe 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

1Treatment groups

Experimental Treatment

Group I: ViltolarsenExperimental Treatment1 Intervention

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