NS Pharma Exon 53 Skipping Trial


The study is a safety and dose finding clinical trial for boys with Duchenne muscular dystrophy amenable to exon 53 skipping.

The study will enroll approximately 16 boys with Duchenne in the United States. NSPharma, Inc. (US subsidiary of Nippon Shinyaku, Co.) is fully supporting this trial and the clinical development of the studydrug, NS-065/NCNP-01.

The study is comprised of a screening period with pre-treatment muscle biopsy and baseline measures. Eligible participants will then have a 4-week double-blind period where randomized patients will receive weekly intravenous (IV) infusions of NS-065/NCNP-01 or placebo followed by a 20-week open label period where all participants will receive weekly treatment with NS-065/NCNP-01 by IV infusion.

Patients completing the 24-week study will be eligible for an open-label extension study.

Some of the enrollment criteria required for participation include:

  • Boys with a diagnosis of Duchenne between ages 4 and less than 10 years old
  • Confirmed DMD Glossary Link mutation amenable to skipping of exon 53
  • Able to walk independently
  • Able to complete the time to stand, time to run/walk 10 meters, and time to climb 4 steps
  • Stable dose of glucocorticoid for at least 3 months prior to screening

Full eligibility requirements for this study will be assessed at participating study sites. For more information on additional enrollment criteria, please visit clinicaltrials.gov (NCT02740972).

This study is being set-up at several sites across the United States. Please continue to check clinicaltrials.gov as additional sites are under evaluation and updates will be made as sites are activated. Reasonable travel and accommodations will be provided or reimbursed.

If you wish to participate or have questions, please visit clinicaltrials.gov for site contact information. You may also contact the study patient advocate Lauren Morgenroth at This email address is being protected from spambots. You need JavaScript enabled to view it..

Please view the NS Pharma Trial webinar hosted by Parent Project Muscular Dystrophy for a closer look into the study.

Posted 12/8/2016