Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Sponsors
NS Pharma, Inc.
Eligibility
Sex/Gender
Male
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 =65 years) no F.1.3.1 Number of subjects for this age range
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 previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect 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 halflife 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., oxendolone), 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. Participant has inadequate renal function, as defined by serum cystatin C greater than 1.5 × upper limit of normal (ULN) at the Screening Visit. If the value is greater than 1.5 × ULN, the serum cystatin C will be repeated once; if the repeated test result is still greater than 1.5 × ULN, the participant should be excluded; 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 |
|---|---|
| Main Objective: To compare the efficacy of viltolarsen administered intravenously (IV) at weekly doses of 80 mg/kg over a 48-week treatment period vs. placebo controls in ambulant boys ages 4 to <8 years with DMD using Time to Stand Test (TTSTAND) as a measure of strength and function.;Secondary Objective: -To compare the efficacy of viltolarsen administered intravenously (IV) at weekly doses of 80 mg/kg in ambulant boys ages 4 to <8 years with DMD over a 48-week treatment period vs. placebo controls using hierarchical strength and endurance outcomes -To evaluate the safety and tolerability of viltolarsen administered intravenously at weekly doses of 80 mg/kg in ambulant boys ages 4 to <8 years with DMD ;Primary end point(s): TTSTAND ;Timepoint(s) of evaluation of this end point: At 48 weeks treatment | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): -Hierarchical analyses of the following strength and endurance measures: - Time to Run/Walk 10 Meters Test (TTRW) - Six-minute Walk Test (6MWT) - North Star Ambulatory Assessment (NSAA) - Time to Climb 4 Steps Test (TTCLIMB) - Quantitative muscle strength measured BY hand-held dynamometer (elbow extension, elbow flexion, knee extension and knee flexion on the dominant side only);Timepoint(s) of evaluation of this end point: At 48 weeks treatment (first 5 bullets) At each study visit (last 10 bullets) | — |
Countries
Australia, Belgium, Brazil, Canada, Chile, France, Italy, Japan, Korea, Republic of, Netherlands, Poland, Russian Federation, Spain, Sweden, Taiwan, Turkey, United Kingdom, United States
Contacts
Public ContactKyle Haas
Medpace
Outcome results
None listed