Clinical trial NCT04768062: 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
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Duchenne Muscular Dystrophy
Lead sponsor: NS Pharma, Inc.
Sponsor class: INDUSTRY
Collaborators: name: Nippon Shinyaku Co., Ltd.
First posted: 2021-02-24
Enrollment: 74
Sex: MALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility 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.

Primary outcomes:
measure: Number of participants with treatment related Adverse Events as assessed by CTCAE v4.03
timeFrame: baseline to up to 96 weeks of treatment
Secondary outcomes:
measure: Time to Stand Test (TTSTAND)
timeFrame: baseline to 96 weeks of treatment
measure: Time to Run/Walk 10 Meters Test (TTRW)
timeFrame: baseline to 96 weeks of treatment
measure: Six-minute Walk Test (6MWT)
timeFrame: baseline to 96 weeks of treatment
measure: North Star Ambulatory Assessment (NSAA)
timeFrame: baseline to 96 weeks of treatment
measure: Time to Climb 4 Stairs Test (TTCLIMB)
timeFrame: baseline to 96 weeks of treatment
measure: Muscle Strength Measured by Hand-Held Dynamometer
timeFrame: baseline to 96 weeks of treatment
Other outcomes:
Locations:
city: Brisbane
country: Australia
facility: Queensland Children's Hospital
city: Westmead
country: Australia
facility: The Childrens Hospital at Westmead
city: Quebec City
country: Canada
facility: CHU de Quebec Research Centre
city: Santiago
country: Chile
facility: Hospital de Niños Roberto del Rio
city: Santiago
country: Chile
facility: Pontificia Universidad Católica de Chile
city: Beijing
country: China
facility: Chinese PLA General Hospital
city: Changsha
country: China
facility: Hunan Children's Hospital
city: Shanghai
country: China
facility: Children's Hospital of Fudan University
city: Shenzhen
country: China
facility: Shenzhen Children's Hospital
city: Nový Hradec Králové
country: Czechia
facility: Fakultni nemocnice Hradec Kralove
city: Athens
country: Greece
facility: Agia Sofia Children's Hospital
city: Thessaloníki
country: Greece
facility: Hippokration General Hospital of Thessaloniki
city: Rome
country: Italy
facility: Fondazione Policlinico Universitario A. Gemelli - Universita Cattolica del Sacro Cuore
city: Tokyo
country: Japan
facility: National Center of Neurology and Psychiatry
city: Pusan
country: Korea, Republic of
facility: Pusan National University Yangsan Hospital
city: Seoul
country: Korea, Republic of
facility: Seoul National University Hospital
city: Ciudad de mexico
country: Mexico
facility: Instituto Nacional de Pediatría
city: Leiden
country: Netherlands
facility: Leids Universitair Medisch Centrum
city: Nijmegen
country: Netherlands
facility: Radboud Universitair Medisch Centrum
city: Auckland
country: New Zealand
facility: New Zealand Clinical Research Ltd.
city: Oslo
country: Norway
facility: Rikshospitalet
city: Moscow
country: Russian Federation
facility: Russian National Research Medical University
city: Saint Petersburg
country: Russian Federation
facility: "Saint Petersburg State Paediatric Medical University" based at Consultative and Diagnostic Centre
city: Tomsk
country: Russian Federation
facility: Tomsk National Research Medical Center of Russian Academy of Sciences
city: Barcelona
country: Spain
facility: Hospital Sant Joan de Deu
city: Madrid
country: Spain
facility: Hospital Universitario La Paz
city: Istanbul
country: Turkey
facility: Yeditepe University Kosuyolu Hospital
city: Birmingham
country: United Kingdom
facility: Birmingham Heartlands Hospital
city: Glasgow
country: United Kingdom
facility: Royal Hospital for Children
city: Glasgow
country: United Kingdom
facility: Royal Manchester Children's Hospital
city: London
country: United Kingdom
facility: University College London Institute of Child Health
Study officials:
References: