NS-050/NCNP-03 / Nippon Shinyaku, National Center of Neurology and Psychiatry - LARVOL DELTA

NS-050/NCNP-03 in Boys With DMD (Meteor50) (clinicaltrials.gov) - P1/2 | N=20 | Recruiting | Sponsor: NS Pharma, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

FDA Grants Rare Pediatric Disease Designation to NS-050/NCNP-03 for the Treatment of Duchenne Muscular Dystrophy (PRNewswire) - "NS Pharma, Inc...announced that the Food and Drug Administration (FDA) has granted rare pediatric disease designation to NS-050/NCNP-03 which is being developed for the treatment of Duchenne muscular dystrophy (Duchenne)....We are preparing a Phase 1 / 2 study to evaluate the safety and efficacy of NS-050/NCNP-03 in patients with Duchenne in Japan and the United States."

FDA event • New P1/2 trial • Duchenne Muscular Dystrophy

A Phase 1/2 study of NS-050/NCNP-03, an investigational exon 50 skipping therapy, in boys with Duchenne muscular dystrophy (Meteor50): Trial design (MDA 2024) - P1/2 | "This Phase 1/2 study is a first-in-human trial to evaluate safety, tolerability, pharmacodynamics, pharmacokinetics, and efficacy of an exon 50 skipping therapy in ambulatory boys with DMD."

P1/2 data • Cardiomyopathy • Cardiovascular • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

NS-050/NCNP-03 in Boys With DMD (Meteor50) (clinicaltrials.gov) - P1/2 | N=20 | Not yet recruiting | Sponsor: NS Pharma, Inc. | Trial completion date: Jul 2026 ➔ May 2027 | Initiation date: Oct 2023 ➔ Jul 2024 | Trial primary completion date: Jun 2026 ➔ May 2027

Trial completion date • Trial initiation date • Trial primary completion date • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

A Phase 1/2 of NS-050/NCNP-03 in Boys With DMD (Meteor50) (clinicaltrials.gov) - P1/2 | N=20 | Not yet recruiting | Sponsor: NS Pharma, Inc.

New P1/2 trial • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy