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

Description

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.

Conditions

Duchenne Muscular Dystrophy

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Study Overview

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Study Details

Study 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.

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

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

Condition
Duchenne Muscular Dystrophy
Intervention / Treatment

-

Contacts and Locations

Aurora

Children's Hospital Colorado, Aurora, Colorado, United States, 80045

Atlanta

Rare Disease Research, Atlanta, Georgia, United States, 30329

Chicago

Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States, 60611

Kansas City

University of Kansas Medical Center (KUMC), Kansas City, Kansas, United States, 66160

Cincinnati

Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, United States, 45229

Portland

Shriners Hospital for Children, Portland, Oregon, United States, 97239

Pittsburgh

University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania, United States, 15224

Dallas

UT Southwestern/Children's Health, Dallas, Texas, United States, 75207

Richmond

Virginia Commonwealth University Health System, Richmond, Virginia, United States, 23298

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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.
  • * 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 taken any gene therapy or other exon-skipping oligonucleotide
  • * Other exclusion criteria may apply.

Ages Eligible for Study

4 Years to 14 Years

Sexes Eligible for Study

MALE

Accepts Healthy Volunteers

No

Collaborators and Investigators

NS Pharma, Inc.,

Study Record Dates

2025-11-28