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Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
study id #: NCT03167255
condition: Duchenne Muscular Dystrophy
status: active, not recruitingpurpose:
This is an open-label, extension study of NS-065/NCNP-01 administered intravenously once weekly for an additional 168 weeks to boys with DMD who complete Study NS-065/NCNP-01-201.
mechanism of action: Exon-skipping to promote dystrophin production
last updated: May 03, 2020
start date: July 6, 2017
estimated completion: May 2021
phase of development: Phase 2
size / enrollment: 16
This is a Phase II, multicenter, open-label, extension study of NS-065/NCNP-01 administered intravenously once weekly for an additional 168 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 168 weeks or until NS-065/NCNP-01 is commercially available, whichever is earlier.
Patients who complete the Phase II Dose-finding Study NS-065/NCNP-01-201 are eligible to enroll.
- Number of participants with treatment related Adverse Events as assessed by CTCAE v4.0. [ Time Frame: 168 weeks of treatment ]
- Change in Time to Stand (TTSTAND) versus matched historical controls. [ Time Frame: 168 weeks of treatment ]
- Change in Time to Run/Walk 10 meters (TTRW) versus matched historical controls. [ Time Frame: 168 weeks of treatment ]
- Change in Time to Climb 4 stairs (TTCLIMB) versus matched historical controls. [ Time Frame: 168 weeks of treatment ]
- North Star Ambulatory Assessment (NSAA) results versus matched historical controls. [ Time Frame: 168 weeks of treatment ]
- Change in distance traveled in the Six-Minute Walk Test (6MWT) versus matched historical controls. [ Time Frame: 168 weeks of treatment ]
- Muscle strength as measured by Quantitative Muscle Testing (QMT) versus matched historical controls. [ Time Frame: 168 weeks of treatment ]
• Eligible Sexes: male
• Completed Study NS-065/NCNP-01-201 through Week 25.
• Willing and able to comply with scheduled visits, investigational product administration plan, and study procedures.
• Stable dose of glucocorticoid (GC), and is expected to remain on the stable dose for the duration of the study.
• Serious or severe adverse event in Study NS-065/NCNP-01-201 that precludes safe use of NS-065/NCNP-01.
• Patient had a treatment which was made for the purpose of dystrophin or its related protein induction after completion of Study NS-065/NCNP-01-201.
• Patient took any other investigational drugs after completion of Study NS-065/NCNP-01-201.
• Patient was judged by the investigator and/or the Sponsor that it was not appropriate to participate in the extension study for other reasons.
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