drugset / Trial / NCT04129294
Exploratory Study of NS-089/NCNP-02 in DMD
Phase 1/2
Completed
6 enrolled
National Center of Neurology and Psychiatry, Japan
Nippon Shinyaku Co., Ltd. · collab
NaSingle-groupOpen-labelTreatment
Summary
This study is designed to assess the safety, tolerability, efficacy and pharmacokinetics (PK) of NS-089/NCNP-02 in subjects diagnosed with Duchenne muscular dystrophy (DMD), and to determine the dosage for subsequent studies.
Timeline
- Start
- 2019-12-02
- Primary completion
- 2022-05-31
- Completion
- 2022-05-31
Publications
- Komaki H, Takeshita E, Kunitake K, Ishizuka T, Shimizu-Motohashi Y, Ishiyama A, Sasaki M, Yonee C, Maruyama S, Hida E, Aoki Y. Phase 1/2 trial of brogidirsen: Dual-targeting antisense oligonucleotides for exon 44 skipping in Duchenne muscular dystrophy. Cell Rep Med. 2025 Jan 21;6(1):101901. doi: 10.1016/j.xcrm.2024.101901. Epub 2025 Jan 9.
- Ishizuka T, Komaki H, Asahina Y, Nakamura H, Motohashi N, Takeshita E, Shimizu-Motohashi Y, Ishiyama A, Yonee C, Maruyama S, Hida E, Aoki Y. Systemic administration of the antisense oligonucleotide NS-089/NCNP-02 for skipping of exon 44 in patients with Duchenne muscular dystrophy: Study protocol for a phase I/II clinical trial. Neuropsychopharmacol Rep. 2023 Jun;43(2):277-286. doi: 10.1002/npr2.12335. Epub 2023 Apr 3.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | brogidirsen | Unknown | 1.62 mg/kg | Intravenous |
| Subject | brogidirsen | Unknown | 10 mg/kg | Intravenous |
| Subject | brogidirsen | Unknown | 40 mg/kg | Intravenous |
| Subject | brogidirsen | Unknown | 80 mg/kg | Intravenous |