A Phase 2 Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of NS-089/NCNP-02 in Boys with Duchenne Muscular Dystrophy (DMD)
Study ID: STU-2022-0205
Summary
This is a Phase 2, open-label, multi-center study of nS-089/nCnP-02 administered by iV infusion once weekly for 24 weeks to ambulant boys aged [GreaterThanorequalTo]4 to [Less Than]15 years with DMD due to mutations amenable to exon 44 skipping. Patients will receive 80 mg/kg/week nS-089/nCnP-02. The study is comprised of a Pre-Treatment Phase of approximately 5 weeks (including screening and pre-treatment muscle biopsy and baseline measures), a 24-week Treatment Phase, and a Post-Treatment Phase of approximately 4 weeks of follow-up. Patients completing the study will be eligible for an open-label extension study (nS-089/nCnP-02-202) under a separate protocol.
* To evaluate the safety and tolerability of 80 mg/kg weekly intravenous (iV) doses of nS-089/nCnP-02 in ambulant boys with DMD over 24 weeks of treatment; * To evaluate the effects of 80 mg/kg weekly iV doses of nS-089/nCnP-02 on induction of dystrophin protein in muscle after 24 weeks of treatment, as measured by Western blot; and * To evaluate the pharmacokinetics (PK) of 80 mg/kg weekly iV doses of nS-089/nCnP-02.
