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

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Description

Treatment of Duchenne muscular dystrophy (DMD) with dystrophin deletion amenable to exon 44 skipping

Details
Age

Child

Eligibility

- Participant has a confirmed diagnosis of DMD - Participant is >=4 and <15 years of age at the time of first infusion in the study - Participant is able to walk independently without assistive devices - Participant is able to complete the TTSTAND without assistance in <7 seconds, as assessed at the Screening Visit and the Pre-Infusion Visit; - Participant has QTc <450 msec on standard 12-lead electrocardiogram (ECG) or, if participant has bundle branch block, QTc <480 msec; - Participant and parent(s) or legal guardian(s) are willing and able to comply with scheduled visits, investigational product (IP) administration plan, and study procedures - Participant must be on a stable dose of glucocorticoid (GC) steroids for at least 3 months prior to the first dose of IP and is expected to remain on a stable dose of GC steroid treatment for the duration of the study - Participants must use contraception during and for 3 months following completion of IP administration, if sexually active

Locations

Childrens Hospital Colorado

Principal Investigator
Photograph of Michele Yang

Michele Yang

Study ID

Protocol Number: 22-0121

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