Back to results

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

Primary Purpose

Duchenne Muscular Dystrophy

Status

Enrolling by invitation

Phase

Phase 3

Locations

International

Study Type

Interventional

Intervention

Viltolarsen

About this trial

This is an interventional treatment trial for Duchenne Muscular Dystrophy

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

Inclusion Criteria:

Patient has completed the NS-065/NCNP-01-301 study;
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;
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:

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;
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;
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.

Sites / Locations

The Childrens Hospital at Westmead
Agia Sofia Children's Hospital
Fondazione Policlinico Universitario A. Gemelli - Universita Cattolica del Sacro Cuore
National Center of Neurology and Psychiatry
Pusan National University Yangsan Hospital
Seoul National University Hospital
Leids Universitair Medisch Centrum
Radboud Universitair Medisch Centrum
New Zealand Clinical Research Ltd.
Russian National Research Medical University
"Saint Petersburg State Paediatric Medical University" based at Consultative and Diagnostic Centre
Tomsk National Research Medical Center of Russian Academy of Sciences
Hospital Sant Joan de Deu
Yeditepe University Kosuyolu Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Viltolarsen

Arm Description

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

Outcomes

Primary Outcome Measures

Number of participants with treatment related Adverse Events as assessed by CTCAE v4.03

Secondary Outcome Measures

Time to Stand Test (TTSTAND)

Change in Time to Stand

Time to Run/Walk 10 Meters Test (TTRW)

Change in Time to Run/Walk 10 meters

Six-minute Walk Test (6MWT)

Change in Six-minute Walk

North Star Ambulatory Assessment (NSAA)

Change in North Star Ambulatory Assessment

Time to Climb 4 Stairs Test (TTCLIMB)

Change in Time to Climb 4 Stairs

Muscle Strength Measured by Hand-Held Dynamometer

Change in Muscle Strength Measured by Hand-Held Dynamometer

Full Information

NCT ID

NCT04768062

First Posted

February 19, 2021

Last Updated

May 24, 2022

Sponsor

NS Pharma, Inc.

Collaborators

Nippon Shinyaku Co., Ltd.

1. Study Identification

Unique Protocol Identification Number

NCT04768062

Brief Title

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

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)

Study Type

Interventional

2. Study Status

Record Verification Date

June 2021

Overall Recruitment Status

Enrolling by invitation

Study Start Date

April 13, 2021 (Actual)

Primary Completion Date

June 2026 (Anticipated)

Study Completion Date

June 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

NS Pharma, Inc.

Collaborators

Nippon Shinyaku Co., Ltd.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

5. 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.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Duchenne Muscular Dystrophy

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

74 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Viltolarsen

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

Viltolarsen

Other Intervention Name(s)

NS-065/NCNP-01

Intervention Description

Received during weekly intravenous infusions

Primary Outcome Measure Information:

Title

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 Measure Information:

Title

Time to Stand Test (TTSTAND)

Description

Change in Time to Stand

Time Frame