Trial Radar
Sign in

NCT lookup

Pull any trial record directly from ClinicalTrials.gov.

NS-050/NCNP-03 in Boys With DMD (Meteor50)
NS-050/NCNP-03 in Boys With DMD (Meteor50)

NCT06053814

Active Not RecruitingPhase 1, Phase 2

Sponsor: NS Pharma, Inc.

Conditions: Duchenne Muscular Dystrophy

Interventions: NS-050/NCNP-03, Placebo

Countries: United States, Canada, Japan, South Korea, Turkey (Türkiye)

This is a Phase 1/2 study of Multiple-Ascending Dose (MAD) levels for 12 weeks of treatment followed by 24 weeks of open-label treatment with a selected dose of NS-050/NCNP-03 administered once weekly to ambulant boys with DMD, who have a DMD exon deletion amenable to exon 50 skipping.

Eligibility overview

Sex: MALE

Age: 4 Years to 15 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria
Inclusion Criteria:

* Male ≥ 4 years and \<16 years of age;
* Confirmed DMD exon deletion in the dystrophin gene that is amenable to skipping of exon 50 to restore the dystrophin mRNA reading frame;
* Able to walk independently without assistive devices;
* Able to complete the TTSTAND without assistance in \<20 seconds;
* Stable dose of glucocorticoid for at least 3 months and the dose is expected to remain on a stable dose for the duration of the study.

Other inclusion criteria may apply.

Exclusion Criteria:

* Evidence of symptomatic cardiomyopathy;
* Current or previous treatment with anabolic steroids (e.g., oxendolone, oxandrolone) or products containing resveratrol or adenosine triphosphate within 3 months prior to first dose of study drug;
* Currently taking another investigational drug or has taken another investigational drug within 3 months prior to the first dose of study drug;
* Surgery within the 3 months prior to the first dose of study drug or planned during the study duration;
* Having taken any gene therapy.

Other exclusion criteria may apply.
Locations (19)
  • Los Angeles, California, United States
  • Aurora, Colorado, United States
  • Chicago, Illinois, United States
  • Kansas City, Kansas, United States
  • Philadelphia, Pennsylvania, United States
  • Pittsburgh, Pennsylvania, United States
  • Calgary, Alberta, Canada
  • Vancouver, British Columbia, Canada
  • London, Ontario, Canada
  • Nagara, Gifu, Japan
  • Nishinomiya, Hyōgo, Japan
  • Sendai, Miyagi, Japan
  • Toyonaka, Osaka, Japan
  • Kodaira, Tokyo, Japan
  • Yangsan, Gyeongsangnam-do, South Korea
  • Seoul, South Korea
  • Ankara, Turkey (Türkiye)
  • Istanbul, Turkey (Türkiye)
  • Istanbul, Turkey (Türkiye)