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Clinical Trial Details — Status: Approved for marketing

Administrative data

NCT number NCT04337112
Other study ID # VILT-501
Secondary ID
Status Approved for marketing
Phase
First received
Last updated

Study information

Verified date August 2020
Source NS Pharma, Inc.
Contact n/a
Is FDA regulated No
Health authority
Study type Expanded Access

Clinical Trial 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.


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.


Recruitment information / eligibility

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.

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
viltolarsen
Intravenous (IV) infusions, weekly, at 80mg/kg, once weekly (approximately every 7 days).

Locations

Country Name City State
n/a

Sponsors (1)

Lead Sponsor Collaborator
NS Pharma, Inc.
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