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

study id #: NCT04060199

condition: Duchenne Muscular Dystrophy

status: recruiting

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.

intervention: Viltolarsen, Placebo

mechanism of action: Exon-skipping to promote dystrophin production

results: https://clinicaltrials.gov/ct2/show/results/NCT04060199

last updated: October 31, 2020

start date: April 14, 2020

estimated completion: November 2024

phase of development: Phase 3

size / enrollment: 74

study 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.

primary outcomes:

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

secondary outcomes:

• 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.

inclusion criteria:

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

sponsor: NS Pharma, Inc.

contacts: 201-986-3860, trialinfo@nspharma.com

• United States, California
  University of California Davis Medical Center
  
  
• United States, Illinois
  Ann & Robert H. Lurie Children’s Hospital of Chicago
  
  
• United States, Kansas
  University of Kansas Medical Center
  
  
• Canada
  CHU de Quebec Research Centre
  
  
• Canada, Alberta
  Alberta Children’s Hospital
  
  
• Italy
  Universita Cattolica del Sacro Cuore
  
  
• Japan
  Hyogo College of Medicine Hospital
  
  
• Japan
  Kumamoto University Hospital
  
  
• Japan
  National Center of Neurology and Psychiatry
  
  
• Netherlands
  Leids Universitair Medisch Centrum
  
  
• Netherlands
  Radboud University Medical Centre
  
  
• New Zealand
  Auckland Clinical Studies Ltd
  
  
• Republic Of Korea
  Seoul National University Hospital
  
  
• Republic Of Korea, Gyeongsangnamdo
  Pusan National University Yangsan Hospital
  
  
• Russian Federation
  Russian National Research Medical University
  
  
• Russian Federation
  Saint-Petersburg State Pediatric Medical University
  
  
• Russian Federation
  Tomsk National Research Medical Center of Russian Academy of Sciences
  
  
• Spain
  Hospital Sant Joan de Deu
  
  
• Sweden
  Queen Silvia Children’s Hospital
  
  
• Taiwan
  Kaohsiung Medical University Chung-Ho Memorial Hospital
  
  
• Taiwan
  National Taiwan University Hospital
  
  
• Turkey
  Yeditepe University Kosuyolu Hospital