Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) | DuchenneXchange

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enrolling by invitation

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

key information

study id #: NCT03167255

condition: Duchenne Muscular Dystrophy

status: enrolling by invitation

purpose:

This is an open-label, extension study of NS-065/NCNP-01 administered intravenously once weekly for an additional 72 weeks to boys with DMD who complete Study NS-065/NCNP-01-201.

intervention: NS-065/NCNP-01

mechanism of action: Exon-skipping to promote dystrophin production

results: https://clinicaltrials.gov/ct2/show/results/NCT03167255

study details

start date: July 6, 2017

estimated completion: July 2019

phase of development: Phase 2

size / enrollment: 16

study description:
This is a Phase II, multicenter, open-label, extension study of NS-065/NCNP-01 administered intravenously once weekly for an additional 72 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 72 weeks.
Patients who complete the Phase II Dose-finding Study NS-065/NCNP-01-201 are eligible to enroll.

primary outcomes:

  • Number of participants with treatment related Adverse Events as assessed by CTCAE v4.0. [Time Frame: 72 weeks of treatment]
  • Change in Time to Stand (TTSTAND) versus matched historical controls. [Time Frame: 72 weeks of treatment]

secondary outcomes:

  • Change in Time to Run/Walk 10 meters (TTRW) versus matched historical controls. [Time Frame: 72 weeks of treatment]
  • Change in Time to Climb 4 stairs (TTCLIMB) versus matched historical controls. [Time Frame: 72 weeks of treatment]
  • North Star Ambulatory Assessment (NSAA) results versus matched historical controls. [Time Frame: 72 weeks of treatment]
  • Change in distance traveled in the Six-Minute Walk Test (6MWT) versus matched historical controls. [Time Frame: 72 weeks of treatment]
  • Muscle strength as measured by Quantitative Muscle Testing (QMT) versus matched historical controls. [Time Frame: 72 weeks of treatment]

inclusion criteria:
• 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.

exclusion criteria:
• 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.

study contacts

sponsor: NS Pharma, Inc.

investigators: Paula R. Clemens, MD

locations: United States, Canada