Clinical trial NCT06053814: NS-050/NCNP-03 in Boys With DMD (Meteor50)
Official title: 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)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: Duchenne Muscular Dystrophy
Lead sponsor: NS Pharma, Inc.
Sponsor class: INDUSTRY
Collaborators: name: Nippon Shinyaku Co., Ltd.
First posted: 2023-09-26
Enrollment: 20
Sex: MALE
Minimum age (years): 4
Maximum age (years): 14
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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 criteria: Inclusion Criteria:

* 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.

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 outcomes:
measure: Part 1: Overall Summary of Treatment-emergent Adverse Events (TEAEs)
timeFrame: Baseline up to Week 24
measure: Part 1: Area Under the Plasma Concentration Versus Time Curve (AUC) of NS-050/NCNP-03
timeFrame: Day 1 (1st dose) for each dose level
measure: Part 1: Amount of Drug Excreted in Urine of NS-050/NCNP-03
timeFrame: Day 1 (1st dose) for each dose level
measure: Part 2: Change from baseline in skeletal muscle dystrophin protein by immunoblot (Western blot)
timeFrame: Baseline, Week25
Secondary outcomes:
measure: Part 2: Change from baseline in skeletal muscle dystrophin protein by mass spectrometry
timeFrame: Baseline, Week25
measure: Part 2: Change from baseline in skeletal muscle dystrophin protein levels by immunofluorescence staining
timeFrame: Baseline, Week25
measure: Part 2: Change from baseline in percentage of exon 50-skipped mRNA of skeletal muscle dystrophin
timeFrame: Baseline, Week25
measure: Part 2: North Star Ambulatory Assessment (NSAA) score
timeFrame: Baseline, Week13, Week25
measure: Part 2: Time to Stand (TTSTAND)
timeFrame: Baseline, Week13, Week25
measure: Part 2: Time to Run/Walk 10 Meters (TTRW)
timeFrame: Baseline, Week13, Week25
measure: Part 2: Time to Climb 4 Stairs (TTCLIMB)
timeFrame: Baseline, Week13, Week25
measure: Part 2: Total distance of 6 Minute Walk Test (6MWT)
timeFrame: Baseline, Week13, Week25
measure: Part 2: Muscle strength measured by Quantitative Muscle Testing (QMT)
timeFrame: Baseline, Week13, Week25
measure: Part 2: Grip/Pinch Strength
timeFrame: Baseline, Week13, Week25
measure: Part 2: Performance of Upper Limb (PUL) 2.0. score
timeFrame: Baseline, Week13, Week25
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles
state: California
city: Aurora
country: United States
facility: Children's Hospital Colorado
state: Colorado
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Portland
country: United States
facility: Shriners Children's Portland
state: Oregon
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center
state: Pennsylvania
city: Nagara
country: Japan
facility: National Hospital Organization Nagara Medical Center
state: Gifu
city: Nishinomiya
country: Japan
facility: Hyogo Medical University Hospital
state: Hyogo
city: Toyonaka
country: Japan
facility: NHO Osaka Toneyama Medical Center
state: Osaka
city: Kodaira
country: Japan
facility: National Center of Neurology and Psychiatry
state: Tokyo
Study officials:
References: