Clinical trial NCT04060199: Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)
Official title: A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With Duchenne Muscular Dystrophy (DMD)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Duchenne Muscular Dystrophy
Lead sponsor: NS Pharma, Inc.
Sponsor class: INDUSTRY
Collaborators: name: Nippon Shinyaku Co., Ltd.
First posted: 2019-08-19
Enrollment: 77
Sex: MALE
Minimum age (years): 4
Maximum age (years): 7
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The main objective of this study is to evaluate the efficacy of Viltolarsen compared to placebo in Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping.

Detailed description: This is a Phase 3 randomized, double-blind, placebo-controlled, multi-center study to assess the efficacy and safety of Viltolarsen in ambulant boys with Duchenne muscular dystrophy. Eligible patients with out-of-frame deletion mutations amenable to exon 53 skipping will be randomized to receive once weekly intravenous (IV) infusions of 80 mg/kg Viltolarsen or placebo for up to 48 weeks.

The study will enroll approximately 74 patients amenable to exon 53 skipping. Clinical efficacy will be assessed at regularly scheduled study visits, including functional tests such as Time to Stand Test (TTSTAND), Time to Run/Walk 10 Meters Test (TTRW), Six-minute Walk Test (6MWT), North Star Ambulatory Assessment (NSAA), Time to Climb 4 Steps Test (TTCLIMB) and Hand-held dynamometer (elbow extension, elbow flexion, knee extension and knee flexion on the dominant side only).

Safety will be assessed through the collection of adverse events (AEs), laboratory tests, electrocardiograms (ECGs), vital signs, and physical examinations throughout the study.

Blood samples will be taken periodically throughout the study to assess the pharmacokinetics of study drug.

Eligibility criteria: Inclusion Criteria:

* Male ≥ 4 years and \< 8 years of age
* Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 53 to restore the dystrophin mRNA reading frame
* Able to walk independently without assistive devices
* TTSTAND \< 10 seconds
* Stable dose of glucocorticoid (GC) for at least 3 months prior to study entry and is expected to remain on stable dose of GC treatment for the duration of the study
* Other inclusion criteria may apply

Exclusion Criteria:

* Current or history of chronic systemic fungal or viral infections
* Acute illness within 4 weeks prior to the first dose of study drug
* Evidence of symptomatic cardiomyopathy (Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary)
* Allergy or hypersensitivity to the study drug or to any of its constituents
* Severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the investigator
* Previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect safety, make it unlikely that treatment and follow-up will be correctly completed or impair the assessment of study results, in the opinion of the investigator;
* Surgery within the 3 months prior to the first dose of study drug or surgery is planned for anytime during the duration of the study
* Participant has positive test results for hepatitis B antigen, hepatitis C antibody or human immunodeficiency virus (HIV)
* Currently taking any other investigational drug or has taken any other investigational drug within 3 months prior to the first dose of study drug or within 5 times the half-life of a medication, whichever is longer
* Previously enrolled in an interventional study of viltolarsen
* Currently taking any other exon skipping agent or has taken any other exon skipping agent within 3 months prior to the first dose of study drug
* Having taken any gene therapy
* Other exclusion criteria may apply

Primary outcomes:
measure: Change From Baseline in Time to Stand (TTSTAND) Velocity
timeFrame: baseline, Week 13, 25, 37, 49
Secondary outcomes:
Other outcomes:
Locations:
city: Sacramento
country: United States
facility: University of California Davis Medical Center
state: California
city: Chicago
country: United States
facility: Ann and Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Brisbane
country: Australia
facility: Queensland Children's Hospital
city: Nedlands
country: Australia
facility: Perth Children's Hospital
city: Westmead
country: Australia
facility: The Childrens Hospital at Westmead
city: Toronto
country: Canada
facility: The Hospital for Sick Children (SickKids)
state: Ontario
city: Calgary
country: Canada
facility: Alberta Children's Hospital
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: Beijing
country: China
facility: The Third Medical Center of 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: Athens
country: Greece
facility: Agia Sofia Children's Hospital
city: Thessaloníki
country: Greece
facility: Hippokration General Hospital of Thessaloniki
city: Kowloon Bay
country: Hong Kong
facility: Hong Kong Children's Hospital
city: Rome
country: Italy
facility: Fondazione Policlinico Universitario A. Gemelli - Universita Cattolica del Sacro Cuore
city: Pusan
country: Korea, Republic of
facility: Pusan National University Yangsan Hospital
city: Seoul
country: Korea, Republic of
facility: Seoul National University Hospital
city: Chihuahua
country: Mexico
facility: Hospital Angeles Chihuahua
city: Ciudad de mexico
country: Mexico
facility: Instituto Nacional de Pediatria
city: Nijmegen
country: Netherlands
facility: Radboud Universitair Medisch Centrum
state: Gelderland
city: Leiden
country: Netherlands
facility: Leids 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 n.a. N.I.Pirogov, structural branch - Research Clinical Institute of Pediatrics n.a. Academician Yu. E. Veltishchev
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: Kaohsiung
country: Taiwan
facility: Kaohsiung Medical University Chung-Ho Memorial Hospital
city: Taipei
country: Taiwan
facility: National Taiwan University Hospital
city: Istanbul
country: Turkey
facility: Yeditepe University Kosuyolu Hospital
city: Kyiv
country: Ukraine
facility: State Institution "Ukrainian Medical rehabilitation Center for Children with organic disorders of the nervous system of the Ministry of Health of Ukraine"
city: Birmingham
country: United Kingdom
facility: Birmingham Heartlands Hospital
city: Glasgow
country: United Kingdom
facility: Royal Hospital for Children - Glasgow
city: London
country: United Kingdom
facility: University College London Institute of Child Health
city: Manchester
country: United Kingdom
facility: Royal Manchester Children's Hospital
Study officials:
References: