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Trial record 1 of 1 for:    Viltolarsen | Duchenne Muscular Dystrophy
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Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)

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ClinicalTrials.gov Identifier: NCT04060199
Recruitment Status : Recruiting
First Posted : August 19, 2019
Last Update Posted : February 19, 2020
Sponsor:
Collaborator:
Nippon Shinyaku Co., Ltd.
Information provided by (Responsible Party):
NS Pharma, Inc.

Tracking Information
First Submitted Date  ICMJE August 15, 2019
First Posted Date  ICMJE August 19, 2019
Last Update Posted Date February 19, 2020
Actual Study Start Date  ICMJE October 23, 2019
Estimated Primary Completion Date November 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 15, 2019)
TTSTAND [ Time Frame: baseline to 48 weeks of treatment ]
Change in Time to Stand (TTSTAND)
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: August 19, 2019)
  • TTRW [ Time Frame: baseline to 48 weeks of treatment ]
    Change in Time to Run/Walk 10 Meters Test (TTRW)
  • 6MWT [ Time Frame: baseline to 48 weeks of treatment ]
    Change in Six-minutes Walk Test (6MWT)
  • 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.
  • TTCLIMB [ Time Frame: baseline to 48 weeks of treatment ]
    Change in Time to Climb 4 Steps Test (TTCLIMB)
  • 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.
Original Secondary Outcome Measures  ICMJE
 (submitted: August 15, 2019)
  • TTRW [ Time Frame: baseline to 48 weeks of treatment ]
    Change in Time to Run/Walk 10 Meters Test (TTRW)
  • 6MWT [ Time Frame: baseline to 48 weeks of treatment ]
    Change in Six-minutes Walk Test (6MWT)
  • NSAA [ Time Frame: baseline to 48 weeks of treatment ]
    Change in North Star Ambulatory Assessment (NSAA)
  • TTCLIMB [ Time Frame: baseline to 48 weeks of treatment ]
    Change in Time to Climb 4 Steps Test (TTCLIMB)
  • Hand-held dynamometer [ Time Frame: baseline to 48 weeks of treatment ]
    Change in Hand-held dynamometer (elbow extension, elbow flexion, knee extension and knee flexion on the dominant side only)
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)
Official Title  ICMJE 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)
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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Duchenne Muscular Dystrophy
Intervention  ICMJE
  • Drug: Viltolarsen
    IV infusion
    Other Name: NS-065/NCNP-01
  • Drug: Placebo
    IV infusion
Study Arms  ICMJE
  • Experimental: Viltolarsen
    Patients amenable to exon 53 skipping will receive viltolarsen intravenous (IV) infusions, weekly, at 80 mg/kg for up to 48 weeks.
    Intervention: Drug: Viltolarsen
  • Placebo Comparator: Placebo
    Patients amenable to exon 53 skipping will receive placebo intravenous (IV) infusions, weekly, for up to 48 weeks.
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: August 15, 2019)
74
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 2024
Estimated Primary Completion Date November 2024   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Gender Based Eligibility: Yes
Ages  ICMJE 4 Years to 7 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Trial Info 201-986-3860 trialinfo@nspharma.com
Listed Location Countries  ICMJE Canada,   Italy,   Japan,   Korea, Republic of,   New Zealand,   Russian Federation,   Spain,   Sweden,   Taiwan,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04060199
Other Study ID Numbers  ICMJE NS-065/NCNP-01-301
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party NS Pharma, Inc.
Study Sponsor  ICMJE NS Pharma, Inc.
Collaborators  ICMJE Nippon Shinyaku Co., Ltd.
Investigators  ICMJE Not Provided
PRS Account NS Pharma, Inc.
Verification Date February 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP