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

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ClinicalTrials.gov Identifier: NCT04060199
Recruitment Status : Recruiting
First Posted : August 19, 2019
Last Update Posted : October 1, 2019
Sponsor:
Collaborator:
Nippon Shinyaku Co., Ltd.
Information provided by (Responsible Party):
NS Pharma, Inc.

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.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: Viltolarsen Drug: Placebo Phase 3

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.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 74 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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)
Estimated Study Start Date : September 2019
Estimated Primary Completion Date : November 2024
Estimated Study Completion Date : December 2024


Arm Intervention/treatment
Experimental: Viltolarsen
Patients amenable to exon 53 skipping will receive viltolarsen intravenous (IV) infusions, weekly, at 80 mg/kg for up to 48 weeks.
Drug: Viltolarsen
IV infusion
Other Name: NS-065/NCNP-01

Placebo Comparator: Placebo
Patients amenable to exon 53 skipping will receive placebo intravenous (IV) infusions, weekly, for up to 48 weeks.
Drug: Placebo
IV infusion




Primary Outcome Measures :
  1. TTSTAND [ Time Frame: baseline to 48 weeks of treatment ]
    Change in Time to Stand (TTSTAND)


Secondary Outcome Measures :
  1. TTRW [ Time Frame: baseline to 48 weeks of treatment ]
    Change in Time to Run/Walk 10 Meters Test (TTRW)

  2. 6MWT [ Time Frame: baseline to 48 weeks of treatment ]
    Change in Six-minutes Walk Test (6MWT)

  3. 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.


  4. TTCLIMB [ Time Frame: baseline to 48 weeks of treatment ]
    Change in Time to Climb 4 Steps Test (TTCLIMB)

  5. 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.



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Ages Eligible for Study:   4 Years to 7 Years   (Child)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04060199


Contacts
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Contact: Trial Info 201-986-3860 trialinfo@nspharma.com

Locations
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Japan
National Center of Neurology and Psychiatry Recruiting
Tokyo, Japan
Sponsors and Collaborators
NS Pharma, Inc.
Nippon Shinyaku Co., Ltd.

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Responsible Party: NS Pharma, Inc.
ClinicalTrials.gov Identifier: NCT04060199     History of Changes
Other Study ID Numbers: NS-065/NCNP-01-301
First Posted: August 19, 2019    Key Record Dates
Last Update Posted: October 1, 2019
Last Verified: September 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked