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)

Nippon Shinyaku Co., Ltd.0 个研究点目标入组 20 人开始时间: 2024年9月18日最近更新: 2025年2月4日

适应症

试验速览

暂无简介。

•Male >= 4 years and <15 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.

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

发起方