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

• Sponsor: NS Pharma, Inc. (Industry)
• Overall Status: Enrolling by invitation
• CT.gov ID: NCT04768062
• Collaborator: Nippon Shinyaku Co., Ltd. (Industry)
• Enrollment: 74
• Locations: 14
• Arm: 1
• Anticipated Duration (Months): 61.6
• Patients Per Site: 5.3
• Patients Per Site Per Month: 0.1

Study Details

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
• Anticipated 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: Apr 13, 2021
• Anticipated Primary Completion Date: Jun 1, 2026
• Anticipated Study Completion Date: Jun 1, 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 Names: NS-065/NCNP-01

Outcome Measures

Primary Outcome Measures

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

Secondary Outcome Measures

1. Time to Stand Test (TTSTAND) [baseline to 96 weeks of treatment]

   Change in Time to Stand

2. Time to Run/Walk 10 Meters Test (TTRW) [baseline to 96 weeks of treatment]

   Change in Time to Run/Walk 10 meters

3. Six-minute Walk Test (6MWT) [baseline to 96 weeks of treatment]

   Change in Six-minute Walk

4. North Star Ambulatory Assessment (NSAA) [baseline to 96 weeks of treatment]

   Change in North Star Ambulatory Assessment

5. Time to Climb 4 Stairs Test (TTCLIMB) [baseline to 96 weeks of treatment]

   Change in Time to Climb 4 Stairs

6. Muscle Strength Measured by Hand-Held Dynamometer [baseline to 96 weeks of treatment]

   Change in Muscle Strength Measured by Hand-Held Dynamometer

Eligibility Criteria

Criteria

• Ages Eligible for Study: N/A and Older
• Sexes Eligible for Study: Male
• Accepts Healthy Volunteers: No

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

	Site	City	State	Country	Postal Code
1	The Childrens Hospital at Westmead	Westmead		Australia
2	Agia Sofia Children's Hospital	Athens		Greece
3	Fondazione Policlinico Universitario A. Gemelli - Universita Cattolica del Sacro Cuore	Rome		Italy
4	National Center of Neurology and Psychiatry	Tokyo		Japan
5	Pusan National University Yangsan Hospital	Pusan		Korea, Republic of
6	Seoul National University Hospital	Seoul		Korea, Republic of
7	Leids Universitair Medisch Centrum	Leiden		Netherlands
8	Radboud Universitair Medisch Centrum	Nijmegen		Netherlands
9	New Zealand Clinical Research Ltd.	Auckland		New Zealand
10	Russian National Research Medical University	Moscow		Russian Federation
11	"Saint Petersburg State Paediatric Medical University" based at Consultative and Diagnostic Centre	Saint Petersburg		Russian Federation
12	Tomsk National Research Medical Center of Russian Academy of Sciences	Tomsk		Russian Federation
13	Hospital Sant Joan de Deu	Barcelona		Spain
14	Yeditepe University Kosuyolu Hospital	Istanbul		Turkey

Sponsors and Collaborators

• NS Pharma, Inc.
• Nippon Shinyaku Co., Ltd.

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

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

Publications

None provided.

• Responsible Party: NS Pharma, Inc.
• ClinicalTrials.gov Identifier: NCT04768062
• Other Study ID Numbers: NS-065/NCNP-01-302
• First Posted: Feb 24, 2021
• Last Update Posted: May 26, 2022
• Last Verified: Jun 1, 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

Study Results

No Results Posted as of May 26, 2022