drugset / Trial / NCT06053814
NS-050/NCNP-03 in Boys With DMD (Meteor50)
RandomizedParallel-groupQuadruple-blindTreatment
Summary
This is a Phase 1/2 study of Multiple-Ascending Dose (MAD) levels for 12 weeks of treatment followed by 24 weeks of open-label treatment with a selected dose of NS-050/NCNP-03 administered once weekly to ambulant boys with DMD, who have a DMD exon deletion amenable to exon 50 skipping.
Timeline
- Start
- 2024-09-18
- Primary completion
- 2028-03
- Completion
- 2028-03
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | NS-050/NCNP-03 | Unknown | 1.95 mg/kg | Intravenous |
| Subject | NS-050/NCNP-03 | Unknown | 5 mg/kg | Intravenous |
| Subject | NS-050/NCNP-03 | Unknown | 10 mg/kg | Intravenous |
| Subject | NS-050/NCNP-03 | Unknown | 20 mg/kg | Intravenous |
| Subject | NS-050/NCNP-03 | Unknown | 40 mg/kg | Intravenous |
| Subject | NS-050/NCNP-03 | Unknown | 80 mg/kg | Intravenous |