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Active, Not Recruiting

NCT Number: NCT06053814

NS-050/NCNP-03 in Boys With DMD (Meteor50)

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Duchenne Muscular Dystrophy Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Muscular Dystrophy, Duchenne Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Age range

4 year–15 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Alberta Children's Hospital, Calgary, Alberta, Canada

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

NS-050/NCNP-03

Drug

NS-050/NCNP-03 solution for IV infusion.

Placebo

Drug

NS-050/NCNP-03 placebo-matching solution for IV infusion.

Primary outcomes

  1. Part 1: Overall Summary of Treatment-emergent Adverse Events (TEAEs)

    Time frame: 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.

  2. Part 1: Area Under the Plasma Concentration Versus Time Curve (AUC) of NS-050/NCNP-03

    Time frame: 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.

  3. Part 1: Amount of Drug Excreted in Urine of NS-050/NCNP-03

    Time frame: 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.

  4. Part 2: Change from baseline in skeletal muscle dystrophin protein by immunoblot (Western blot)

    Time frame: Baseline, Week25

Secondary outcomes

  1. Part 2: Change from baseline in skeletal muscle dystrophin protein by mass spectrometry

    Time frame: Baseline, Week25

  2. Part 2: Change from baseline in skeletal muscle dystrophin protein levels by immunofluorescence staining

    Time frame: Baseline, Week25

  3. Part 2: Change from baseline in percentage of exon 50-skipped mRNA of skeletal muscle dystrophin

    Time frame: Baseline, Week25

  4. Part 2: North Star Ambulatory Assessment (NSAA) score

    Time frame: Baseline, Week13, Week25

    The NSAA is a functional scale devised for use in ambulant children with Duchenne muscular dystrophy (DMD). It consists of 17 activities graded 0 (unable to perform), 1 (performs with modifications), 2 (normal movement). It assesses abilities necessary to remain ambulant that have been found to progressively deteriorate in untreated DMD patients, as well as in other muscular dystrophies such as Becker Muscular Dystrophy. NSAA Total Score ranges from 0 to 34, with a score of 34 implying normal function.

  5. Part 2: Time to Stand (TTSTAND)

    Time frame: Baseline, Week13, Week25

  6. Part 2: Time to Run/Walk 10 Meters (TTRW)

    Time frame: Baseline, Week13, Week25

  7. Part 2: Time to Climb 4 Stairs (TTCLIMB)

    Time frame: Baseline, Week13, Week25

  8. Part 2: Total distance of 6 Minute Walk Test (6MWT)

    Time frame: Baseline, Week13, Week25

  9. Part 2: Muscle strength measured by Quantitative Muscle Testing (QMT)

    Time frame: Baseline, Week13, Week25

  10. Part 2: Grip/Pinch Strength

    Time frame: Baseline, Week13, Week25

  11. Part 2: Performance of Upper Limb (PUL) 2.0. score

    Time frame: Baseline, Week13, Week25

    The PUL 2.0 provides both a total score and sub-scores for the 3 domains (shoulder, middle, and distal) that in DMD are progressively involved with a proximal to distal gradient. The PUL includes 22 items with an entry item to define the starting functional level. The 22 items are subdivided into the high level shoulder dimension (6 items), middle level elbow dimension (9 items), and distal wrist and hand dimension (7 items). For weaker patients, a low score on the entry item (0 2) means high level items do not need to be performed. Scoring options vary across the scale between 0-1 and 0-2 according to performance. Each dimension can be scored separately with a maximum score of 12 for the high level shoulder dimension, 17 for the middle level elbow dimension, and 13 for the distal wrist and hand dimension. A total score can be achieved by adding the 3 level scores (maximum total score of 42).

Sponsors and collaborators

Lead sponsor

NS Pharma, Inc.

Industry

Collaborators

  • Nippon Shinyaku Co., Ltd.

Registry information

Official study 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)

Important dates

Study start
2024
Primary completion
2028
Study completion
2028
First posted
Sep 26, 2023
Registry last updated
Jun 16, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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