Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD

JRCT ID: jRCT2080224893

Registered date:27/09/2019

Basic Information

• Recruitment status: completed
• Health condition(s) or Problem(s) studied: Duchenne Muscular Dystrophy
• Date of first enrollment: 14/04/2020
• Target sample size: 74
• Countries of recruitment: Japan,Asia except Japan,North America,South America,Europe,Oceania
• Study type: Interventional

Intervention(s)

investigational material(s)
Generic name etc : NS-065/NCNP-01
INN of investigational material : viltolarsen
Therapeutic category code : 19- Other agents affecting nervous system and sensory organs
Dosage and Administration for Investigational material : IV infusion

control material(s)
Generic name etc : Placebo
INN of investigational material : -
Therapeutic category code : --- Other
Dosage and Administration for Investigational material : -

Outcome(s)

Primary Outcome

efficacy
confirmatory
TTSTAND [ Time Frame: baseline to 48 weeks of treatment ]
Change in Time to Stand (TTSTAND)

Secondary Outcome

efficacy
confirmatory
TTRW [ Time Frame: baseline to 48 weeks of treatment ]
Change in Time to Run/Walk 10 Meters Test (TTRW)
efficacy
confirmatory
6MWT [ Time Frame: baseline to 48 weeks of treatment ]
Change in Six-minutes Walk Test (6MWT)
efficacy
confirmatory
NSAA [ Time Frame: baseline to 48 weeks of treatment ]
Change in North Star Ambulatory Assessment (NSAA)

The NSAA is a functional scale devised for use in ambulant children with Duchenne muscular dystrophy (DMD). It consists of 17 activities graded 0 (unable to perform), 1 (performs with modifications), 2 (normal movement). It assesses abilities necessary to remain ambulant that have been found to progressively deteriorate in untreated DMD patients, as well as in other muscular dystrophies such as Becker Muscular Dystrophy. NSAA Total Score ranges from 0 to 34, with a score of 34 implying normal function.
efficacy
confirmatory
TTCLIMB [ Time Frame: baseline to 48 weeks of treatment ]
Change in Time to Climb 4 Steps Test (TTCLIMB)
efficacy
confirmatory
Hand-held dynamometer [ Time Frame: baseline to 48 weeks of treatment ]
The force generated for each muscle strength (elbow extension, elbow flexion, knee extension, and knee flexion on the dominant side only) will be measured by Hand-held dynamometer.

Key inclusion & exclusion criteria

• Age minimum: >= 4age old
• Age maximum: <= 7age old
• Gender: Male

Include criteria

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 the first dose of study drug and is expected to remain on stable dose of GC treatment for the duration of the study
Other inclusion criteria may apply

Exclude 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

Related Information

• Primary Sponsor: Nippon Shinyaku Co., Ltd.
• Secondary Sponsor: NS Pharma, Inc.
• Source(s) of Monetary Support: -
• Secondary ID(s): NCT04060199,JapicCTI-194978,2019-002076-13