NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)

Condition(s) studied

Investigational drug(s) / intervention(s)

NS-089/NCNP-02: Cohort 1: Part 1 Dose Level 1-3: a 4-week Treatment Phase at each treatment dose level Part 2 Single Dose Level: a 24-week Treatment Phase at the MTD of Part 1 Cohort 2: Part 2 Single Dose Level: a 24-week Treatment Phase at the MTD of Part 1

Study summary

This is a Phase 2, open-label, multi-center, 2-part study of NS-089/NCNP-02 administered by weekly IV infusion to ambulant boys aged ≥4 to \<15 years with DMD due to mutations amenable to exon 44 skipping. Participants will receive a selected dose of NS-089/NCNP-02 administered once weekly.

The study consists of 2 parts: Part 1 and Part 2. Six participants (Cohort 1) will participate in both Part 1 and Part 2, and 14 participants (Cohort 2) will be added for Part 2.

Eligibility

Primary outcome measure(s)

• Adverse Event and Adverse Drug Reaction — through study completion, up to follow-up phone call for Part 2
• Plasma pharmacokinetic (PK) parameters — Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Maximum plasma concentration (Cmax) of NS-089/NCNP-02
• Plasma pharmacokinetic (PK) parameters — Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Time of the maximum plasma concentration (Tmax) of NS-089/NCNP-02
• Plasma pharmacokinetic (PK) parameters — Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Terminal half-life (T1/2) of NS-089/NCNP-02
• Plasma pharmacokinetic (PK) parameters — Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Area under the concentration-time curve from time 0 to the last time point (AUC0-t) of NS-089/NCNP-02
• Plasma pharmacokinetic (PK) parameters — Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Area under the concentration-time curve from time 0 to infinity (AUC0-∞) of NS-089/NCNP-02
• Plasma pharmacokinetic (PK) parameters — Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Total body clearance (CLtot) of NS-089/NCNP-02
• Plasma pharmacokinetic (PK) parameters — Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] The volume in the terminal state (Vz) of NS-089/NCNP-02
• Urine pharmacokinetic parameters — Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Urinary excretion of NS-089/NCNP-02
• Change from baseline in skeletal muscle dystrophin protein by immunoblot (Western blot). — Baseline, Week25

Trial sites (29)

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