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

Duchenne Muscular Dystrophy Clinical Trial

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)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04768062
• Other study ID #: NS-065/NCNP-01-302
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: April 13, 2021
• Est. completion date: November 2025

Study information

• Verified date: February 2024
• Source: NS Pharma, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 74
• Est. completion date: November 2025
• Est. primary completion date: October 2025
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• Viltolarsen
Received during weekly intravenous infusions

Locations

Country	Name	City	State
Australia	Queensland Children's Hospital	Brisbane
Australia	The Childrens Hospital at Westmead	Westmead
Canada	CHU de Quebec Research Centre	Quebec City
Chile	Hospital de Niños Roberto del Rio	Santiago
Chile	Pontificia Universidad Católica de Chile	Santiago
China	Chinese PLA General Hospital	Beijing
China	Hunan Children's Hospital	Changsha
China	Children's Hospital of Fudan University	Shanghai
China	Shenzhen Children's Hospital	Shenzhen
Czechia	Fakultni nemocnice Hradec Kralove	Nový Hradec Králové
Greece	Agia Sofia Children's Hospital	Athens
Greece	Hippokration General Hospital of Thessaloniki	Thessaloníki
Italy	Fondazione Policlinico Universitario A. Gemelli - Universita Cattolica del Sacro Cuore	Rome
Japan	National Center of Neurology and Psychiatry	Tokyo
Korea, Republic of	Pusan National University Yangsan Hospital	Pusan
Korea, Republic of	Seoul National University Hospital	Seoul
Mexico	Instituto Nacional de Pediatría	Ciudad de mexico
Netherlands	Leids Universitair Medisch Centrum	Leiden
Netherlands	Radboud Universitair Medisch Centrum	Nijmegen
New Zealand	New Zealand Clinical Research Ltd.	Auckland
Norway	Rikshospitalet	Oslo
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
Spain	Hospital Sant Joan de Deu	Barcelona
Spain	Hospital Universitario La Paz	Madrid
Turkey	Yeditepe University Kosuyolu Hospital	Istanbul
United Kingdom	Birmingham Heartlands Hospital	Birmingham
United Kingdom	Royal Hospital for Children	Glasgow
United Kingdom	Royal Manchester Children's Hospital	Glasgow
United Kingdom	University College London Institute of Child Health	London

Sponsors (2)

Lead Sponsor	Collaborator
NS Pharma, Inc.	Nippon Shinyaku Co., Ltd.

Countries where clinical trial is conducted

Australia,  Canada,  Chile,  China,  Czechia,  Greece,  Italy,  Japan,  Korea, Republic of,  Mexico,  Netherlands,  New Zealand,  Norway,  Russian Federation,  Spain,  Turkey,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of participants with treatment related Adverse Events as assessed by CTCAE v4.03		baseline to up to 96 weeks of treatment
Secondary	Time to Stand Test (TTSTAND)	Change in Time to Stand	baseline to 96 weeks of treatment
Secondary	Time to Run/Walk 10 Meters Test (TTRW)	Change in Time to Run/Walk 10 meters	baseline to 96 weeks of treatment
Secondary	Six-minute Walk Test (6MWT)	Change in Six-minute Walk	baseline to 96 weeks of treatment
Secondary	North Star Ambulatory Assessment (NSAA)	Change in North Star Ambulatory Assessment	baseline to 96 weeks of treatment
Secondary	Time to Climb 4 Stairs Test (TTCLIMB)	Change in Time to Climb 4 Stairs	baseline to 96 weeks of treatment
Secondary	Muscle Strength Measured by Hand-Held Dynamometer	Change in Muscle Strength Measured by Hand-Held Dynamometer	baseline to 96 weeks of treatment

External Links

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