Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X)

NS Pharma

Status and phase: Active, not recruiting; Phase 3

Conditions: Duchenne Muscular Dystrophy

Treatments: Drug: Viltolarsen

Study type: Interventional

Funder types: Industry

Identifiers

NCT04768062

NS-065/NCNP-01-302

Details and patient eligibility

About

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.

Full description

This Phase 3 study is a 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. Patients will receive viltolarsen administered IV at weekly doses of 80 mg/kg.

Study NS-065/NCNP-01-302 will be comprised of a 96-week treatment period.

Enrollment: 74 patients

Sex: Male

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Patient has completed the NS-065/NCNP-01-301 study;
2. 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;
3. Patient and parent(s)/guardian(s) are willing and able to comply with scheduled visits, investigational product (IP) administration plan, and study procedures.

Exclusion criteria

1. 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;
2. Patient had a treatment which was made for the purpose of dystrophin or dystrophin-related protein induction after completion of Study NS-065/NCNP-01-301;
3. Patient took any other investigational drug(s) during or after completion of Study NS-065/NCNP-01-301;
4. Patient is judged by the investigator and/or the sponsor not to be appropriate to participate in the extension study for any reason.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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74 participants in 1 patient group

Viltolarsen

Experimental group

Description:

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

Treatment:

Drug: Viltolarsen