Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)

• ClinicalTrials.gov Identifier: NCT04060199
• Recruitment Status: Completed
• First Posted: August 19, 2019
• Last Update Posted: October 27, 2023
• Sponsor: NS Pharma, Inc.
• Collaborator: Nippon Shinyaku Co., Ltd.
• Information provided by (Responsible Party): NS Pharma, Inc.

Tracking Information

• First Submitted Date: August 15, 2019
• First Posted Date: August 19, 2019
• Last Update Posted Date: October 27, 2023
• Actual Study Start Date: April 14, 2020
• Actual Primary Completion Date: October 19, 2023 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: August 15, 2019)

TTSTAND [ Time Frame: baseline to 48 weeks of treatment ]

Change in Time to Stand (TTSTAND)

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (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 (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: 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)
• 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: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
• Condition: Duchenne Muscular Dystrophy

Intervention

• Drug: Viltolarsen
  IV infusion
  Other Name: NS-065/NCNP-01
• Drug: Placebo
  IV infusion

Study Arms

• 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

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: October 25, 2023): 77
• Original Estimated Enrollment (submitted: August 15, 2019): 74
• Actual Study Completion Date: October 19, 2023
• Actual Primary Completion Date: October 19, 2023 (Final data collection date for primary outcome measure)

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

Sex/Gender

• Sexes Eligible for Study: Male
• Gender Based Eligibility: Yes

• Ages: 4 Years to 7 Years (Child)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Australia, Canada, Chile, China, Greece, Hong Kong, Italy, Korea, Republic of, Mexico, Netherlands, New Zealand, Norway, Russian Federation, Spain, Taiwan, Turkey, Ukraine, United Kingdom, United States
• Removed Location Countries: Japan, Sweden

Administrative Information

• NCT Number: NCT04060199
• Other Study ID Numbers: 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

• Plan to Share IPD: No

• Current Responsible Party: NS Pharma, Inc.
• Original Responsible Party: Same as current
• Current Study Sponsor: NS Pharma, Inc.
• Original Study Sponsor: Same as current
• Collaborators: Nippon Shinyaku Co., Ltd.
• Investigators: Not Provided
• PRS Account: NS Pharma, Inc.
• Verification Date: October 2023