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The Expanded Access Use of Viltolarsen in Duchenne Muscular Dystrophy With Confirmed Exon 53 Amenable Mutation

Primary Purpose

Muscular Dystrophy, Duchenne, DMD

Status
Approved for marketing
Phase
Locations
Study Type
Expanded Access
Intervention
viltolarsen
Sponsored by
NS Pharma, Inc.
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for Muscular Dystrophy, Duchenne focused on measuring Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, Inborn, Genetic Diseases, X-Linked

Eligibility Criteria

3 Years - 12 Years (Child)Male

Inclusion Criteria:

  • Male ≥ 3 and ≤ 12 years of age
  • Clinical signs compatible with DMD
  • Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 53 to restore the dystrophin messenger ribonucleic acid (mRNA) reading frame
  • Able to walk independently without assistive device
  • Not able to participate in a Phase 3 trial

Exclusion Criteria:

  • Chronic systemic fungal or viral infections
  • An acute illness within 4 weeks prior to the first dose of viltolarsen
  • Symptomatic cardiomyopathy
  • Patient has a previous or ongoing medical condition, medical history, physical findings, or laboratory abnormality that could affect participant safety in the opinion of the treating physician
  • Surgery within the 3 months prior to the first anticipated administration of viltolarsen and in the opinion of the treating physician would impact weekly treatment schedule
  • Positive test results for hepatitis B antigen, hepatitis C antibody, or human immunodeficiency virus (HIV) antibody at screening
  • Currently taking any other investigational drug or has taken any other investigational drug within 3 months prior to the first dose of viltolarsen
  • Previously enrollment in any viltolarsen study.
  • Currently taking any other exon skipping agent or has taken any other exon skipping agent within 2 weeks prior to the first dose of viltolarsen (would need to be discontinued in order to be eligible)
  • Any gene therapy for DMD
  • Inadequate renal function as defined by a serum cystatin C > 1.5 x upper limit of normal (ULN). If the value is > 1.5 x ULN then the measurement can be repeated once. If repeat measurement is still > 1.5 x ULN then the patient should be excluded.

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    April 2, 2020
    Last Updated
    August 14, 2020
    Sponsor
    NS Pharma, Inc.
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    1. Study Identification

    Unique Protocol Identification Number
    NCT04337112
    Brief Title
    The Expanded Access Use of Viltolarsen in Duchenne Muscular Dystrophy With Confirmed Exon 53 Amenable Mutation
    Official Title
    The Expanded Access Use of Viltolarsen for the Treatment of Duchenne Muscular Dystrophy (DMD) Amenable to Exon 53 Skipping
    Study Type
    Expanded Access

    2. Study Status

    Record Verification Date
    August 2020
    Overall Recruitment Status
    Approved for marketing
    Study Start Date
    undefined (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    undefined (undefined)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    NS Pharma, Inc.

    4. Oversight

    5. Study Description

    Brief Summary
    This is an open label expanded access program for boys, 3 to 12 years old, for the treatment of Duchenne muscular dystrophy (DMD) with confirmed mutation(s) in the dystrophin gene that is amenable to skipping of exon 53.
    Detailed Description
    This expanded access program is designed to provide access to viltolarsen in patients with DMD with confirmed mutation(s) in the dystrophin gene amenable to skipping of exon 53, who in the opinion and clinical judgement of the treating physician, would benefit from treatment with viltolarsen.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Muscular Dystrophy, Duchenne, DMD
    Keywords
    Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, Inborn, Genetic Diseases, X-Linked

    7. Study Design

    8. Arms, Groups, and Interventions

    Intervention Type
    Drug
    Intervention Name(s)
    viltolarsen
    Other Intervention Name(s)
    NS-065/NCNP-01
    Intervention Description
    Intravenous (IV) infusions, weekly, at 80mg/kg, once weekly (approximately every 7 days).

    10. Eligibility

    Sex
    Male
    Minimum Age & Unit of Time
    3 Years
    Maximum Age & Unit of Time
    12 Years
    Eligibility Criteria
    Inclusion Criteria: Male ≥ 3 and ≤ 12 years of age Clinical signs compatible with DMD Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 53 to restore the dystrophin messenger ribonucleic acid (mRNA) reading frame Able to walk independently without assistive device Not able to participate in a Phase 3 trial Exclusion Criteria: Chronic systemic fungal or viral infections An acute illness within 4 weeks prior to the first dose of viltolarsen Symptomatic cardiomyopathy Patient has a previous or ongoing medical condition, medical history, physical findings, or laboratory abnormality that could affect participant safety in the opinion of the treating physician Surgery within the 3 months prior to the first anticipated administration of viltolarsen and in the opinion of the treating physician would impact weekly treatment schedule Positive test results for hepatitis B antigen, hepatitis C antibody, or human immunodeficiency virus (HIV) antibody at screening Currently taking any other investigational drug or has taken any other investigational drug within 3 months prior to the first dose of viltolarsen Previously enrollment in any viltolarsen study. Currently taking any other exon skipping agent or has taken any other exon skipping agent within 2 weeks prior to the first dose of viltolarsen (would need to be discontinued in order to be eligible) Any gene therapy for DMD Inadequate renal function as defined by a serum cystatin C > 1.5 x upper limit of normal (ULN). If the value is > 1.5 x ULN then the measurement can be repeated once. If repeat measurement is still > 1.5 x ULN then the patient should be excluded.

    12. IPD Sharing Statement

    Learn more about this trial

    The Expanded Access Use of Viltolarsen in Duchenne Muscular Dystrophy With Confirmed Exon 53 Amenable Mutation

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