Trials / Recruiting
RecruitingNCT06053814
NS-050/NCNP-03 in Boys With DMD (Meteor50)
A Phase 1/2, First in Human, Multiple-dose, 2-part Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NS-050/NCNP-03 in Boys With Duchenne Muscular Dystrophy (DMD)
- Status
- Recruiting
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 20 (estimated)
- Sponsor
- NS Pharma, Inc. · Industry
- Sex
- Male
- Age
- 4 Years – 14 Years
- Healthy volunteers
- Not accepted
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | NS-050/NCNP-03 | NS-050/NCNP-03 solution for IV infusion. |
| DRUG | Placebo | NS-050/NCNP-03 placebo-matching solution for IV infusion. |
Timeline
- Start date
- 2024-09-18
- Primary completion
- 2027-05-01
- Completion
- 2027-05-01
- First posted
- 2023-09-26
- Last updated
- 2025-09-10
Locations
18 sites across 5 countries: United States, Canada, Japan, South Korea, Turkey (Türkiye)
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT06053814. Inclusion in this directory is not an endorsement.