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NCT Number: NCT05996003

NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)

This is a Phase 2, open-label, multi-center, 2-part study of NS-089/NCNP-02 administered by weekly IV infusion to ambulant boys aged ≥4 to <15 years with DMD due to mutations amenable to exon 44 skipping. Participants will receive a selected dose of NS-089/NCNP-02 administered once weekly.

The study consists of 2 parts: Part 1 and Part 2. Six participants (Cohort 1) will participate in both Part 1 and Part 2, and 14 participants (Cohort 2) will be added for Part 2.

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Key information

Conditions

Duchenne Muscular Dystrophy Congenital, Hereditary, and Neonatal Diseases and Abnormalities DMD Exon 44 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–14 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 2

Primary location

Perth Children's Hospital, Nedlands, Western Australia, Australia

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Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Male ≥ 4 years and <15 years of age
  • Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 44 to restore the dystrophin mRNA reading frame
  • Able to walk independently without assistive devices
  • Ability 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

  • Has a body weight of <20 kg at the time of informed consent (applies to participants screening for Part 1 only)
  • Evidence of symptomatic cardiomyopathy
  • Current or previous treatment with anabolic steroids (e.g., oxandrolone) or products containing resveratrol or adenosine triphosphate within 3 months prior to first dose of study drug
  • Current or previous treatment with any other investigational drug within 3 months prior to the first dose of study drug or within 5 times the half-life of a medication, whichever is longer
  • Surgery within the 3 months prior to the first dose of study drug or planned during the study duration
  • Previously treated in an interventional study of NS-089/NCNP-02
  • Having received exon skipping oligonucleotide within 1 year prior to the first dose of IP
  • Other exclusion criteria may apply.

Treatment and study plan

NS-089/NCNP-02

Drug

Cohort 1:

Part 1 Dose Level 1-3: a 4-week Treatment Phase at each treatment dose level

Part 2 Single Dose Level: a 24-week Treatment Phase at the MTD of Part 1

Cohort 2:

Part 2 Single Dose Level: a 24-week Treatment Phase at the MTD of Part 1

Other names: Brogidirsen

Primary outcomes

  1. Adverse Event and Adverse Drug Reaction

    Time frame: through study completion, up to follow-up phone call for Part 2

  2. Plasma pharmacokinetic (PK) parameters

    Time frame: Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Maximum plasma concentration (Cmax) of NS-089/NCNP-02

  3. Plasma pharmacokinetic (PK) parameters

    Time frame: Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Time of the maximum plasma concentration (Tmax) of NS-089/NCNP-02

  4. Plasma pharmacokinetic (PK) parameters

    Time frame: Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Terminal half-life (T1/2) of NS-089/NCNP-02

  5. Plasma pharmacokinetic (PK) parameters

    Time frame: Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Area under the concentration-time curve from time 0 to the last time point (AUC0-t) of NS-089/NCNP-02

  6. Plasma pharmacokinetic (PK) parameters

    Time frame: Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Area under the concentration-time curve from time 0 to infinity (AUC0-∞) of NS-089/NCNP-02

  7. Plasma pharmacokinetic (PK) parameters

    Time frame: [Time Frame: Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Total body clearance (CLtot) of NS-089/NCNP-02

  8. Plasma pharmacokinetic (PK) parameters

    Time frame: Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] The volume in the terminal state (Vz) of NS-089/NCNP-02

  9. Urine pharmacokinetic parameters

    Time frame: Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Urinary excretion of NS-089/NCNP-02

  10. Change from baseline in skeletal muscle dystrophin protein by immunoblot (Western blot).

    Time frame: Baseline, Week25

Secondary outcomes

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

    Time frame: Baseline, Week25

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

    Time frame: Baseline, Week25

  3. Change from baseline in percentage of exon 44-skipped mRNA of skeletal muscle dystrophin

    Time frame: Baseline, Week25

  4. 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. Time to Run/Walk 10 Meters (TTRW)

    Time frame: Baseline, Week13, Week25

  6. Time to Stand (TTSTAND)

    Time frame: Baseline, Week13, Week25

  7. Total distance of 6 Minute Walk Test (6MWT)

    Time frame: Baseline, Week13, Week25

  8. Time to Climb 4 Stairs (TTCLIMB)

    Time frame: Baseline, Week13, Week25

  9. Muscle strength measured by Quantitative Muscle Testing (QMT)

    Time frame: Baseline, Week13, Week25

  10. Grip and pinch strength

    Time frame: Baseline, Week13, Week25

  11. 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).

Study contacts

Contact information is provided by the study sponsor or research team.

Trial info

CONTACT

[email protected]

1-866-677-6276

Sponsors and collaborators

Lead sponsor

NS Pharma, Inc.

Industry

Collaborators

  • Nippon Shinyaku Co., Ltd.

Registry information

Official study title

A Phase 2 Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of NS-089/NCNP-02 in Boys With Duchenne Muscular Dystrophy (DMD)

Important dates

Study start
2024
Primary completion
2026
Study completion
2026
First posted
Aug 16, 2023
Registry last updated
Mar 6, 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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