A Global Phase 1/2 study of NS-050/NCNP-03 in Boys with Duchenne Muscular Dystrophy (DMD)
Research type
Research Study
Full title
A Phase 1/2, First in Human, Multiple-dose, 2-part Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NS-050/NCNP-03 in Boys with Duchenne Muscular Dystrophy (DMD)
IRAS ID
1012262
Contact name
Takeshi Seita
Contact email
Sponsor organisation
NS Pharma, Inc.
Clinicaltrials.gov Identifier
Research summary
This is a Phase 1/2 study that will investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NS-050/NCNP-03, an investigational medicinal product in Boys with Duchenne Muscular Dystrophy (DMD).
Patients with DMD have a mutation in the gene that makes a protein called dystrophin. With this mutation, the muscles are unable to make dystrophin, and the muscles become weak.
This study consists of 2 parts: Part 1 and Part 2.
Some participants will be invited to participate in both Part 1 and Part 2 of the study, and some will be invited to participate only in Part 2.
Part 1 is a double-blind, placebo-controlled study. Double-blind means neither the participant, nor the study doctor will know to which treatment group the participant has been assigned to.
Part 2 is an open-label study, meaning all participants in this part of the study will be treated with NS 050/NCNP-03.
Each part of the study will consist of 3 stages:
1. Pre-Treatment Phase (Screening + Pre-Infusion Visit)
2. Treatment Phase
3. Post-Treatment Phase (Week 25, Follow-up phone call and End of Trial)
The total duration of participation are as follows:
Part 1 – 54 Weeks
Part 2 – 33 Weeks
Part 1 and 2 - 25 months
Participants are eligible if they are male and ≥ 4 years and <15 years of age at the time of first infusion in the study.
It is expected that approximately 20 participants will take part in this study globally.The study is sponsored by NS Pharma, Inc.
REC name
London - Central Research Ethics Committee
REC reference
25/LO/0619
Date of REC Opinion
9 Oct 2025
REC opinion
Further Information Favourable Opinion