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. Patient (if age 18 years or older) or patient's parent(s) or legal guardian(s) has (have) provided written informed consent and Health Insurance Portability and Accountability Act authorization, where applicable, prior to any study-related procedures; patients younger than age 18 years will be asked to give written or verbal assent according to local requirements; 2. Patient has a confirmed diagnosis of DMD defined as: a. Patient is male with clinical signs compatible with DMD; and b. Patient has a confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 53 to restore the dystrophin messenger ribonucleic acid reading frame including determination of unambiguously defined exon boundaries (using techniques such as multiplex ligation-dependent probe amplification, comparative genomic hybridization array, or other techniques with similar capability); 3. Patient is >= 8 years of age at time of first infusion in the study; 4. Patient has a Brooke scale rating of 3 or better OR an upright FVC 30% or greater at Screening; 5. Patient, if sexually active, is willing to abstain from sexual intercourse or employ a barrier or medical method of contraception during and for 3 months following completion of IP administration; 6. Patient and patient's parent(s)/guardian(s) (if patient is =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Patient has had an acute illness within 4 weeks prior to the first dose of IP; 2. Patient has evidence of symptomatic cardiomyopathy (New York Heart Association Class III or higher); 3. Patient requires ventilation support while awake during the day; 4. Patient has an allergy or hypersensitivity to IP or any of its constituents; 5. Patient has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the investigator; 6. Patient has a previous or ongoing medical condition, medical history, physical findings, or laboratory abnormalities that could affect patient 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. Patient has had surgery within 3 months prior to the first anticipated administration of IP or has known plans to have surgery during the Treatment Period; 8. Patient has positive test results for hepatitis B antigen, hepatitis C antibody, or human immunodeficiency virus antibody at Screening; 9. Patient has been diagnosed with asthma that requires chronic treatment with a long-acting beta agonist; 10. Patient has relevant history of or current drug or alcohol abuse or use of any tobacco/marijuana products by smoking or vaping within 3 months prior to treatment with IP; 11. Patient is currently taking any other investigational drug or has taken any other investigational drug within 3 months prior to the first dose of IP or within 5 times the half life of a medication, whichever is longer; 12. Patient has taken any gene therapy; 13. Patient 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 IP; 14. Patient has hydronephrosis, hydroureter, renal or urinary tract calculi, or ureteral stenosis by renal ultrasound; 15. Patient was previously enrolled in an interventional study of viltolarsen.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety and tolerability of viltolarsen administered intravenously (IV) at weekly doses of 80 mg/kg in ambulant and non ambulant boys >= 8 years of age with DMD;Secondary Objective: To compare the efficacy of viltolarsen administered IV at weekly doses of 80 mg/kg over a 48 week Treatment Period versus natural history controls in ambulant and non ambulant boys >= 8 years of age with DMD;Primary end point(s): • Vital signs • Physical examination • Renal ultrasound • Echocardiogram • Clinical laboratory tests : Hematology and clinical chemistry, Urinalysis, Urine cytology • 12-lead electrocardiogram (ECG) • Anti-viltolarsen antibodies • Anti-dystrophin antibodies • Treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs);Timepoint(s) of evaluation of this end point: • Vital signs & TEAE's/SAE's: every study visit • Physical examination: Day 1 and Weeks 5, 9, 13, 17, 21, 25, 37, 49 • Renal Ultrasound & Echocardiogram: Weeks 25 and 49 • Clinical Laboratory: - Hematology & Chemistry: Day 1 and Weeks 3, 5, 9, 13, 17, 21, 25, 37, 49 - Urinalysis: Weeks 3, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49 - Urine Cytology: Weeks 13, 25, 27, 49 • 12-Lead ECG: Weeks 13, 25, 37, 49 • Anti-viltolarsen and anti-dystrophin antibodies: Weeks 13, 25, 37, 48 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Peak Expiratory Flow (PEF) • Forced Vital Capacity (FVC) • Forced expiratory volume in 1 second (FEV1) • Function and Strength tests: - Performance of Upper Limb (PUL) test - Brooke scale - Vignos scale - Hand-held dynamometer - North Star Ambulatory Assessment (NSAA);Timepoint(s) of evaluation of this end point: • PEF, FVC, FEV1: Day 1 and Weeks 13, 25, 37, 49 • Function and Strength tests: Weeks 13, 25, 37, 49 | — |
Countries
China, Italy, Russian Federation, Spain, Turkey, United States
Contacts
Public ContactClinical Trial Management
Medpace
Outcome results
None listed