A Global Phase 2 study of NS-089/NCNP-02 in Boys with Duchenne Muscular Dystrophy (DMD)

• Research type

  Research Study

• Full title

  A Phase 2 Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of NS-089/NCNP-02 in Boys with Duchenne Muscular Dystrophy (DMD)

• IRAS ID

  1011306

• Contact name

  Takeshi Seita

• Contact email

  a.pathak@nspharma.com

• Sponsor organisation

  NS Pharma, Inc.

• Clinicaltrials.gov Identifier

  NCT05996003

• Research summary

  Patients with Duchenne Muscular Dystrophy (DMD) have a mutation in the gene that makes a protein called dystrophin. With this mutation, the muscles of patients with DMD are unable to make dystrophin and the muscles become weak. Current treatment of DMD is with glucocorticoid (GC) steroid however these have significant side effects.

  This Phase 2 study will investigate the safety and tolerability of NS-089/NCNP-02, an investigational medicinal product. This is an open-label study therefore all participants will receive NS-089/NCNP-02.

  This study consists of 2 parts: Part 1 and Part 2.
  All UK participants will participate in Part 2 of the study only.

  The study will consist of 3 stages:
  - Pre-Treatment Phase (Screening + Pre-Infusion Visit) – 5 weeks
  - Treatment Phase - 24 weeks
  - Post-Treatment Phase (Week 25, Follow-up phone call and End of Trial) – 4 weeks

  Participants are eligible if they are male and ≥ 4 years and <15 years of age.
  Approximately 20 participants will take part in Part 1 and Part 2 of this study globally.

  The study is sponsored by NS Pharma, Inc.

• REC name

  South West - Central Bristol Research Ethics Committee

• REC reference

  25/SW/0083

• Date of REC Opinion

  29 Aug 2025

• REC opinion

  Further Information Favourable Opinion