NS-065/NCNP-01 for Exon 53 Skipping - Safety and Dose Finding Study of NS-065/NCNP-01 in Boys with Duchenne


Frequently Asked Questions (FAQs) about this research:


What stage is this research?

  • Study NS-065/NCNP-01-201 is a Phase II study that is recruiting participants at clinical sites in North America (United States and Canada).


What is the goal or purpose of this study?

  • This study is being conducted to evaluate the safety and tolerability of a low dose and high dose of NS-065/NCNP-01 injection delivered as a weekly intravenous infusion in patients with Duchenne who are amenable to exon 53 skipping. NS-065/NCNP-01 is a new drug designed to skip exon 53 in the dystrophin Glossary Link gene. Additional objectives of the study will assess its effect on induction of dystrophin, muscle function and strength, pharmacokinetics and pharmacodynamics.


Who is funding this study?

  • The study is sponsored by NS Pharma, Inc.


Who is eligible to paricipate in this study?

  • Approximately 16 boys with Duchenne will be enrolled. The main eligibility criteria are:
    - Boys between the ages of 4 and less than 10 years old;
    - Confirmed Duchenne diagnosis with a specific change in the dystrophin gene that is amenable to skipping of exon 53;
    - Able to walk independently without assistive devices;
    - Able to complete the time to stand, time to run walk and time to climb assessments;
    - Currently on a stable dose of glucocorticoid for at least 3 months


Will all boys enrolled in this trial receive treatment with the investigational therapy?

  • This study is a 2-period, randomized, placebo-controlled, dose finding study for 24 weeks.
    -Period 1 is a 4-week, blinded, placebo-controlled phase. This means that neither you nor your study doctor will know if your son is receiving the study medication or an inactive solution (placebo).
    -Period 2 is a 20-week, open-label phase. This means that all participants receive the study medication.
    -The escalation to the high dose will occur once all participants have completed the first period of 4 weeks in the low dose and safety of the low dose is confirmed


What do I have to do if I decide to participate in this study?

  • The Principal Investigator (study doctor) will review study requirements with all participants during the screening process.
  • Participants enrolled in this study will receive weekly infusions of NS-065/NCNP-01 or placebo for the first 4 weeks. Then all participants will receive weekly infusions of NS-065/NCNP-01 for the following 20 weeks.
  • Participants will have study visits for safety laboratory tests, muscle function and strength testing, and other safety monitoring such as adverse event collection. Participants will also have 2 muscle biopsies, of the bicep muscle, during the study.


How long will this study last, and will I have access to the drug/treatment once the study has ended?

  • This study will last up to 28 weeks (weekly infusion for 24 weeks).
  • Participants completing the study will be eligible for a separate 24-week open-label extension study.


Why is Study NS-065/NCNP-01-201 including a placebo group of participants?

  • During the initial 4 weeks (Period 1), participants are randomized to receive NS-065/NCNP-01 or placebo. Participants in the placebo arm serve as a control arm to participants in the NS-065/NCNP-01 arm. The safety information collected during Period 1 will be compared between the placebo and NS-065/NCNP-01 groups to help evaluate the safety of NS-065/NCNP-01.


 Where does this study take place?

  • This study is conducted at selected participating centers of the Cooperative International Neuromuscular Research Group (CINRG) in the North America. For more information on site locations, please visit www.clinicaltrials.gov. Identifier NCT02740972).


 Will I get paid for participating in this study?

  • Reimbursement is available for travel expenses and meals for study visits.
  • Support and travel arrangements are made by Greenphire, a travel agency specializing in clinical research travel arrangements.


 Why should I consider participating in this study?

  • There are no guarantees that participating in this research will benefit you or your son. But, by participating you and your son can:
  • Help to advance research on the safety and effectiveness of NS-065/NCNP-01
  • Help researchers to better understand exon skipping in DMD.


Where can I learn more about this research?