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
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
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
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
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Primary Completion Date
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Study Completion Date
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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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