NS-050/NCNP-03-101 in Boys With Duchenne Muscular Dystrophy (DMD)
Active, Not RecruitingPhase 1/2Exon Skipping
Study Overview
- Age
- 4–15 years
- Phase
- Phase 1/2
- Sponsor
- NS Pharma
- Therapeutic Approach
- Exon Skipping
- Variant Requirement
- Confirmed DMD exon deletion in the dystrophin gene that is amenable to skipping of exon 50 to restore the dystrophin mRNA reading frame.
- Eligible Sex
- Male
- Ambulation
- Ambulatory
- Study Start (Actual)
- 2024-09-18
- Primary Completion (Estimated)
- 2028-03
- Study Completion (Estimated)
- 2028-03
- Enrollment (Estimated)
- 20
- Countries
- United StatesCanadaJapanSouth KoreaTurkey
Study Requirements and Criteria
Steroid Use
Stable dose of glucocorticoid for at least 3 months and the dose is expected to remain on a stable dose for the duration of the study.
Inclusion Criteria
- Male ≥ 4 years and <16 years of age;
- Able to walk independently without assistive devices;
- Able to complete the TTSTAND without assistance in <20 seconds;
Exclusion Criteria
- Evidence of symptomatic cardiomyopathy;
- Current or previous treatment with anabolic steroids (e.g., oxendolone, oxandrolone) or products containing resveratrol or adenosine triphosphate within 3 months prior to first dose of study drug;
- Currently taking another investigational drug or has taken another investigational drug within 3 months prior to the first dose of study drug;
- Surgery within the 3 months prior to the first dose of study drug or planned during the study duration;
- Having taken any gene therapy.
- Other exclusion criteria may apply.
Contact Information
This section provides contact details for people who can answer questions about joining this study
- Name: Trial info
- Phone Number: 1-866-677-6276
- Email: trialinfo@nspharma.com
Clinical Trial Registry
NCT ID
NCT06053814This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.