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Recruiting NCT05996003

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

Phase II Interventional Duchenne Muscular Dystrophy Exon 44 DMD

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: NS-089/NCNP-02.
Who it may be relevant to
Registry conditions: Duchenne Muscular Dystrophy, Exon 44, DMD. Basic parameters: 4 years — 14 years · Male.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Australia, Canada, Japan, New Zealand +2
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official 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)

Overview

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.

Interventions

  • Drug NS-089/NCNP-02
    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

Primary outcome measures

  • Adverse Event and Adverse Drug Reaction [Time frame: through study completion, up to follow-up phone call for Part 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]
  • 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]
  • 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]
  • 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]
  • 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]
  • 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]
  • 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]
  • 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]
  • Change from baseline in skeletal muscle dystrophin protein by immunoblot (Western blot). [Time frame: Baseline, Week25]
Secondary outcome measures (11)
  • Change from baseline in skeletal muscle dystrophin protein by mass spectrometry. [Time frame: Baseline, Week25]
  • Change from baseline in skeletal muscle dystrophin protein levels by immunofluorescence staining. [Time frame: Baseline, Week25]
  • Change from baseline in percentage of exon 44-skipped mRNA of skeletal muscle dystrophin [Time frame: Baseline, Week25]
  • North Star Ambulatory Assessment (NSAA) score [Time frame: Baseline, Week13, Week25]
  • Time to Run/Walk 10 Meters (TTRW) [Time frame: Baseline, Week13, Week25]
  • Time to Stand (TTSTAND) [Time frame: Baseline, Week13, Week25]
  • Total distance of 6 Minute Walk Test (6MWT) [Time frame: Baseline, Week13, Week25]
  • Time to Climb 4 Stairs (TTCLIMB) [Time frame: Baseline, Week13, Week25]
  • Muscle strength measured by Quantitative Muscle Testing (QMT) [Time frame: Baseline, Week13, Week25]
  • Grip and pinch strength [Time frame: Baseline, Week13, Week25]
  • Performance of Upper Limb (PUL) 2.0. score [Time frame: Baseline, Week13, Week25]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Sequential
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 10 centers
  • Children's Hospital Colorado — Aurora
  • Rare Disease Research, LLC - FL — Kissimmee
  • Rare Disease Research — Atlanta
  • Ann and Robert H. Lurie Children's Hospital of Chicago — Chicago
  • University of Kansas Medical Center (KUMC) — Kansas City
  • Cincinnati Children's Hospital Medical Center — Cincinnati
  • The Children's Hospital of Philadelphia (CHOP) — Philadelphia
  • University of Pittsburgh School of Medicine — Pittsburgh
  • … and 2 more centers
Japan · 5 centers
  • Fukui Prefectural Hospital — Fukui-shi
  • National Hospital Organization Nagara Medical Center — Nagara
  • NHO Osaka Toneyama Medical Center — Toyonaka
  • Shiga General Hospital — Moriyama-shi
  • National Center of Neurology and Psychiatry — Kodaira
Canada · 3 centers
  • Alberta Children's Hospital — Calgary
  • British Columbia Children's Hospital — Vancouver
  • London Health Sciences Centre — London
Turkey (Türkiye) · 3 centers
  • Ankara Bilkent City Hospital — Ankara
  • Yeditepe University Kosuyolu Hospital — Istanbul
  • S.B.U. Dr. Behcet uz Pediatric Diseases and Surgery Training and Research Hospital — Izmir
South Korea · 2 centers
  • Pusan National University Yangsan Hospital — Yangsan
  • Seoul National University Hospital — Seoul
Australia · 1 center
  • Perth Children's Hospital — Nedlands
New Zealand · 1 center
  • Starship Children's Hospital — Auckland

Identifiers

NCT: NCT05996003 · NS-089/NCNP-02-201

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗