Muscular Dystrophy, Duchenne Clinical Trial
Official title:
The Expanded Access Use of Viltolarsen for the Treatment of Duchenne Muscular Dystrophy (DMD) Amenable to Exon 53 Skipping
NCT number | NCT04337112 |
Other study ID # | VILT-501 |
Secondary ID | |
Status | Approved for marketing |
Phase | |
First received | |
Last updated |
Verified date | August 2020 |
Source | NS Pharma, Inc. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Expanded Access |
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.
Status | Approved for marketing |
Enrollment | 0 |
Est. completion date | |
Est. primary completion date | |
Accepts healthy volunteers | |
Gender | Male |
Age group | 3 Years to 12 Years |
Eligibility |
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. |
Country | Name | City | State |
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n/a |
Lead Sponsor | Collaborator |
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NS Pharma, Inc. |
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