NS-050/NCNP-03: NS-050/NCNP-03 solution for IV infusion.
Placebo: NS-050/NCNP-03 placebo-matching solution for IV infusion.
Study 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.
Eligibility
Sex
MALE
Min age
4 Years
Max age
15 Years
Healthy volunteers
No
Inclusion Criteria:
* 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.
Other inclusion criteria may apply.
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.
Primary outcome measure(s)
Part 1: Overall Summary of Treatment-emergent Adverse Events (TEAEs) — Baseline up to Week 24 TEAEs will be summarized both at the patient level for number of TEAEs, highest severity, relationship, action, and outcome, and at the TEAE level (summarizing events) by system organ class (SOC) and preferred term (PT) as well as severity, relationship, action, and outcome.
The most recent version of the Medical Dictionary for Regulatory Activities (MedDRA) will be used for coding TEAEs.
Part 1: Area Under the Plasma Concentration Versus Time Curve (AUC) of NS-050/NCNP-03 — Day 1 (1st dose) for each dose level Blood samples will be collected at the designated time frame. Pharmacokinetic (PK) parameters of NS-050/NCNP-03 will be calculated using non-compartmental methods.
Part 1: Amount of Drug Excreted in Urine of NS-050/NCNP-03 — Day 1 (1st dose) for each dose level Urine samples will be collected at the designated time frame. PK parameters of NS-050/NCNP-03 will be calculated using non-compartmental methods.
Part 2: Change from baseline in skeletal muscle dystrophin protein by immunoblot (Western blot) — Baseline, Week25
Trial sites (19)
Facility
City
Region
Status
Children's Hospital Los Angeles
Los Angeles
California
Children's Hospital Colorado
Aurora
Colorado
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago
Illinois
University of Kansas Medical Center
Kansas City
Kansas
Children's Hospital of Philadelphia
Philadelphia
Pennsylvania
University of Pittsburgh Medical Center
Pittsburgh
Pennsylvania
Alberta Children's Hospital
Calgary
Alberta
British Columbia Children's Hospital
Vancouver
British Columbia
London Health Sciences Centre
London
Ontario
National Hospital Organization Nagara Medical Center
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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