A Phase 2 Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of NS-089/NCNP-02 in Boys with Duchenne Muscular Dystrophy (DMD)
Primary Objective
To evaluate the safety and tolerability of multiple-ascending dose (MAD) levels, 40 mg/kg, 60 mg/kg, and 80 mg/kg, of NS-089/NCNP-02 in ambulant boys with DMD after 4 weeks of once weekly intravenous (IV) treatment; and o evaluate the pharmacokinetics (PK) of MAD levels of NS-089/NCNP-02.
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
Michele Yang
Study ID
Protocol Number: 22-0121
Categories
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