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

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ClinicalTrials.gov Identifier: NCT04337112
Expanded Access Status : Approved for marketing
First Posted : April 7, 2020
Last Update Posted : August 18, 2020
Sponsor:
Information provided by (Responsible Party):
NS Pharma, Inc.

Study Description
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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.

Condition or disease Intervention/treatment
Muscular Dystrophy, Duchenne DMD Drug: viltolarsen

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.
Study Design
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Study Type : Expanded Access
Expanded Access Type : Individual Patients
Official Title: The Expanded Access Use of Viltolarsen for the Treatment of Duchenne Muscular Dystrophy (DMD) Amenable to Exon 53 Skipping

Resource links provided by the National Library of Medicine


Interventions
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Intervention Details:
  • Drug: viltolarsen
    Intravenous (IV) infusions, weekly, at 80mg/kg, once weekly (approximately every 7 days).
    Other Name: NS-065/NCNP-01
Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   3 Years to 12 Years   (Child)
Sexes Eligible for Study:   Male
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.
Contacts and Locations
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More Information
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Responsible Party: NS Pharma, Inc.
ClinicalTrials.gov Identifier: NCT04337112    
Other Study ID Numbers: VILT-501
First Posted: April 7, 2020    Key Record Dates
Last Update Posted: August 18, 2020
Last Verified: August 2020
Keywords provided by NS Pharma, Inc.:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
 Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
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