This is a Phase 3, multi-center, open-label extension study in ambulant boys with DMD who have completed the 48-week treatment period of either viltolarsen or placebo in Study NS-065/NCNP-01-301.
This Phase 3 study is a multi-center, open-label extension study in ambulant boys with DMD who have completed the 48-week treatment period of either viltolarsen or placebo in Study NS-065/NCNP-01-301. Patients will receive viltolarsen administered IV at weekly doses of 80 mg/kg. Study NS-065/NCNP-01-302 will be comprised of a 96-week treatment period.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
74
Received during weekly intravenous infusions
Number of participants with treatment related Adverse Events as assessed by CTCAE v4.03
Time frame: baseline to up to 96 weeks of treatment
Time to Stand Test (TTSTAND)
Change in Time to Stand
Time frame: baseline to 96 weeks of treatment
Time to Run/Walk 10 Meters Test (TTRW)
Change in Time to Run/Walk 10 meters
Time frame: baseline to 96 weeks of treatment
Six-minute Walk Test (6MWT)
Change in Six-minute Walk
Time frame: baseline to 96 weeks of treatment
North Star Ambulatory Assessment (NSAA)
Change in North Star Ambulatory Assessment
Time frame: baseline to 96 weeks of treatment
Time to Climb 4 Stairs Test (TTCLIMB)
Change in Time to Climb 4 Stairs
Time frame: baseline to 96 weeks of treatment
Muscle Strength Measured by Hand-Held Dynamometer
Change in Muscle Strength Measured by Hand-Held Dynamometer
Time frame: baseline to 96 weeks of treatment
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Queensland Children's Hospital
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Pontificia Universidad Católica de Chile
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Fakultni nemocnice Hradec Kralove
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...and 21 more locations