臨床研究等提出・公開システム

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)

NS-050/NCNP-03-101

Horiguchi Tatsuya

Nippon Shinyaku Co., Ltd.

14, Nishinosho-Monguchi-cho, Kisshoin, Minami-ku, Kyoto

zz_mail_clinical-trials@po.nippon-shinyaku.co.jp

Clinical Development Operations

Nippon Shinyaku Co., Ltd.

14, Nishinosho-Monguchi-cho, Kisshoin, Minami-ku, Kyoto

+81-120-40-8930

zz_mail_clinical-trials@po.nippon-shinyaku.co.jp

Recruiting

July. 31, 2024

Interventional

randomized controlled trial

double blind

placebo control

parallel assignment

treatment purpose

- Male >= 4 years and <16 years of age;
- Confirmed DMD exon deletion in the dystrophin gene that is amenable to skipping of exon 50 to restore the dystrophin mRNA reading frame;
- Able to walk independently without assistive devices;
- Able to complete the TTSTAND without assistance in <20 seconds;
- 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.

- 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.

Male

Duchenne Muscular Dystrophy

Experimental: Part 1: NS-050/NCNP-03
- Participants will be randomized and receive NS-050/NCNP-03 intravenous (IV) infusions once weekly for 2 weeks at each of MAD levels (1.95, 5, 10, 20, 40, and 80 mg/kg).
- Interventions: Drug: NS-050/NCNP-03

Placebo Comparator: Part 1: Placebo
- Participants will be randomized and receive NS-050/NCNP-03 placebo-matching IV infusions once weekly for 2 weeks at each of MAD levels.
- Interventions: Drug: Placebo

Experimental: Part 2: NS-050/NCNP-03
- Participants will receive NS-050/NCNP-03 IV infusions once weekly for 24 weeks at the dosage selected by the Data and Safety Monitoring Board (DSMB) at the conclusion of Part 1.
- Interventions: Drug: NS-050/NCNP-03

Biceps brachii muscle dystrophin protein by Western blot.

- Biceps brachii muscle dystrophin protein by mass spectrometry and immunofluorescence staining.
- Change from baseline in percentage of exon 50-skipped mRNA of skeletal muscle dystrophin.
- North Star Ambulatory Assessment (NSAA)
- TTSTAND
- TTRW
- TTCLIMB
- 6 Minute Walk Test (6MWT)
- Muscle strength measured by Quantitative Muscle Testing (QMT)
- Grip/Pinch Strength
- Performance of Upper Limb (PUL) 2.0

+81-58-232-7755

Jan. 10, 2024

No

United States/Canada/South Korea/Turkey