DMD Duchenne Muscular Dystrophy
Conditions
Interventions
Intravenous administration of the study drug.
Sponsors
NS Pharma, Inc.
Eligibility
Age
2 Years to 11 Years
Inclusion criteria
Inclusion criteria: 1. Participant*s parent(s) or legal guardian(s) has (have) provided written informed consent and Health Insurance Portability and Accountability Act (HIPAA) authorization, where applicable, prior to any study-related procedures; participants will be asked to give written or verbal assent according to local requirements 2. Participant has a confirmed diagnosis of DMD defined as: a. Participant is male with clinical signs compatible with DMD; and b. Participant has a confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 53 to restore the dystrophin mRNA reading frame including determination of unambiguously defined exon boundaries (using techniques such as Multiplex Ligation-dependent Probe Amplification [MLPA], comparative genomic hybridization [CGH] array or other techniques with similar capability) 3. Participant is >= 4 years and
Exclusion criteria
Exclusion criteria: 1. Participant has current or history of chronic systemic fungal or viral infections 2. Participant has had an acute illness within 4 weeks prior to the first dose of study drug based on the Principal Investigator*s judgment/discretion 3. Participant has evidence of symptomatic cardiomyopathy (Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary) 4. Participant has an allergy or hypersensitivity to the study drug or to any of its constituents 5. Participant has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the investigator 6. Participant has a previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect participant safety, make it unlikely that treatment and follow-up will be correctly completed or impair the assessment of study results, in the opinion of the investigator 7. Participant has had surgery within the 3 months prior to the first anticipated administration of study drug or surgery is planned for anytime during the duration of the study 8. Participant has positive test results for hepatitis B antigen, hepatitis C antibody or human immunodeficiency virus (HIV) antibody at screening. (Note: A positive hepatitis C antibody result is acceptable if accompanied by a negative hepatitis C RNA test and normal bilirubin and gamma glutamyl transferase results.) 9. Participant is currently taking any other investigational drug or has taken any other investigational drug within 3 months prior to the first dose of study drug or within 5 times the half-life of a medication, whichever is longer 10. Participant was previously enrolled in an interventional study of viltolarsen 11. Participant is currently taking any other exon skipping agent or has taken any other exon skipping agent within 3 months prior to the first dose of study drug 12. Participant has taken any gene therapy 13. Participant is currently taking idebenone, anabolic steroids (e.g., oxandrolone), or products containing resveratrol or adenosine triphosphate, or has taken such within 3 months prior to first dose of study drug. Coenzyme Q10 or creatine are permitted only if the participant is receiving a stable dose for at least 3 months prior to the first dose of study drug and for the duration of the study 14. Note: There is no exclusion criterion #14. This criterion was removed from the protocol with Amendment 4 (version 3.0, dated 08 January 2021); however, the numbering was maintained to avoid documentation errors; 15. Participant has hydronephrosis, hydroureter, renal or urinary tract calculi, or ureteral stenosis by medical history or renal ultrasound.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| • TTSTAND at 48 weeks of treatment | — |
Secondary
| Measure | Time frame |
|---|---|
| Hierarchical analysis at 48 weeks treatment of the following strength and endurance measures: • TTRW • 6MWT • NSAA • TTCLIMB • Quantitative muscle strength measured by hand-held dynamometer (elbow extension, elbow flexion, knee extension and knee flexion on the dominant side only) • Vital signs (blood pressure, heart rate, respiratory rate, and body temperature [modality for determining temperature should be consistent for each participant at all assessment time points throughout the study]) • Physical examination • Clinical laboratory tests: • Hematology and clinical chemistry • Urinalysis • Urine cytology • Exogenous tracer glomerular filtration rate (GFR) • Antibodies to dystrophin and viltolarsen • 12-lead electrocardiogram (ECG) • Clinical signs and symptoms (AEs and SAEs) • Grading of clinical and clinical laboratory AEs will be according to the Common Terminology Criteria for Adverse Events (CTCAE), v.4.03 | — |
Countries
Netherlands
Outcome results
None listed