Open-Label, Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)


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Primary Outcome Measures:

  • Number of participants with treatment related Adverse Events as assessed by CTCAE v4.0. [ Time Frame: 24 weeks of treatment ]
  • Change in Time to Stand (TTSTAND) versus matched historical controls. [ Time Frame: 24 weeks of treatment ]
Secondary Outcome Measures:

  • Change in Time to Run/Walk 10 meters (TTRW) versus matched historical controls. [ Time Frame: 24 weeks of treatment ]
  • Change in Time to Climb 4 stairs (TTCLIMB) versus matched historical controls. [ Time Frame: 24 weeks of treatment ]
  • North Star Ambulatory Assessment (NSAA) results versus matched historical controls. [ Time Frame: 24 weeks of treatment ]
  • Change in distance traveled in the Six-Minute Walk Test (6MWT) versus matched historical controls. [ Time Frame: 24 weeks of treatment ]
  • Muscle strength as measured by Quantitative Muscle Testing (QMT) versus matched historical controls. [ Time Frame: 24 weeks of treatment ]

This is a Phase II, multicenter, open-label, extension study of NS-065/NCNP-01 administered intravenously once weekly for an additional 24 weeks to boys with DMD who complete Study NS-065/NCNP-01-201. This study will evaluate the safety, tolerability, and clinical efficacy of NS-065/NCNP-01 at dose levels of up to 80 mg/kg/week administered by weekly IV infusion over an additional treatment period of 24 weeks.

Patients who complete the Phase II Dose-finding Study NS-065/NCNP-01-201 are eligible to enroll.