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

• ClinicalTrials.gov Identifier: NCT04768062
• Recruitment Status: Enrolling by invitation
• First Posted: February 24, 2021
• Last Update Posted: November 16, 2021
• Sponsor: NS Pharma, Inc.
• Collaborator: Nippon Shinyaku Co., Ltd.
• Information provided by (Responsible Party): NS Pharma, Inc.

Study Description

Brief Summary:

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.

Condition or disease	Intervention/treatment	Phase
Duchenne Muscular Dystrophy	Drug: Viltolarsen	Phase 3

Detailed 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.

Study Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 74 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Phase 3, Multi-center, Open-label Extension Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With Duchenne Muscular Dystrophy (DMD)
• Actual Study Start Date: April 13, 2021
• Estimated Primary Completion Date: June 2026
• Estimated Study Completion Date: June 2026

Arms and Interventions

Arm	Intervention/treatment
Experimental: Viltolarsen; Patients amenable to exon 53 skipping will receive viltolarsen intravenous (IV) infusions, weekly, at 80 mg/kg for up to 96 weeks.	Drug: Viltolarsen; Received during weekly intravenous infusions; Other Name: NS-065/NCNP-01

Outcome Measures

Primary Outcome Measures :

1. Number of participants with treatment related Adverse Events as assessed by CTCAE v4.03 [ Time Frame: baseline to up to 96 weeks of treatment ]

Secondary Outcome Measures :

1. Time to Stand Test (TTSTAND) [ Time Frame: baseline to 96 weeks of treatment ]
   Change in Time to Stand
2. Time to Run/Walk 10 Meters Test (TTRW) [ Time Frame: baseline to 96 weeks of treatment ]
   Change in Time to Run/Walk 10 meters
3. Six-minute Walk Test (6MWT) [ Time Frame: baseline to 96 weeks of treatment ]
   Change in Six-minute Walk
4. North Star Ambulatory Assessment (NSAA) [ Time Frame: baseline to 96 weeks of treatment ]
   Change in North Star Ambulatory Assessment
5. Time to Climb 4 Stairs Test (TTCLIMB) [ Time Frame: baseline to 96 weeks of treatment ]
   Change in Time to Climb 4 Stairs
6. Muscle Strength Measured by Hand-Held Dynamometer [ Time Frame: baseline to 96 weeks of treatment ]
   Change in Muscle Strength Measured by Hand-Held Dynamometer

Eligibility Criteria

• Ages Eligible for Study: Child, Adult, Older Adult
• Sexes Eligible for Study: Male
• Accepts Healthy Volunteers: No

Criteria

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.

Contacts and Locations

Locations

Italy

Fondazione Policlinico Universitario A. Gemelli - Universita Cattolica del Sacro Cuore

Rome, Italy

Japan

National Center of Neurology and Psychiatry

Tokyo, Japan

Korea, Republic of

Pusan National University Yangsan Hospital

Pusan, Korea, Republic of

Seoul National University Hospital

Seoul, Korea, Republic of

Netherlands

Leids Universitair Medisch Centrum

Leiden, Netherlands

New Zealand

Christchurch Clinical Studies Trust

Auckland, New Zealand

New Zealand Clinical Research Ltd.

Auckland, New Zealand

Russian Federation

Russian National Research Medical University

Moscow, Russian Federation

"Saint Petersburg State Paediatric Medical University" based at Consultative and Diagnostic Centre

Saint Petersburg, Russian Federation

Tomsk National Research Medical Center of Russian Academy of Sciences

Tomsk, Russian Federation

Spain

Hospital Sant Joan de Deu

Barcelona, Spain

Turkey

Yeditepe University Kosuyolu Hospital

Istanbul, Turkey

Sponsors and Collaborators

NS Pharma, Inc.

Nippon Shinyaku Co., Ltd.

More Information

Additional Information:

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

• Responsible Party: NS Pharma, Inc.
• ClinicalTrials.gov Identifier: NCT04768062
• Other Study ID Numbers: NS-065/NCNP-01-302
• First Posted: February 24, 2021
• Last Update Posted: November 16, 2021
• Last Verified: June 2021

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Muscular Dystrophies; Muscular Dystrophy, Duchenne; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked