JRCT ID: jRCT2080224893
Registered date:27/09/2019
Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD
Basic Information
Recruitment status | completed |
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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) |
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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 |
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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. |
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Secondary Sponsor | NS Pharma, Inc. |
Source(s) of Monetary Support | - |
Secondary ID(s) | NCT04060199,JapicCTI-194978,2019-002076-13 |
Contact
Public contact | |
Name | Clinical Development Operations |
Address | 14 Kisshouin Nishinoshou Monnguchi-chou, Minami-ku, Kyoto |
Telephone | +81-120-40-8930 |
zz_mail_clinical-trials@po.nippon-shinyaku.co.jp | |
Affiliation | Nippon Shinyaku Co., Ltd. |
Scientific contact | |
Name | Takeshi Seita |
Address | 14 Kisshouin Nishinoshou Monnguchi-chou, Minami-ku, Kyoto |
Telephone | +81-120-40-8930 |
zz_mail_clinical-trials@po.nippon-shinyaku.co.jp | |
Affiliation | Nippon Shinyaku Co., Ltd. |