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

Purpose

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.

Condition

  • Duchenne Muscular Dystrophy

Eligibility

Eligible Ages
Between 4 Years and 14 Years
Eligible Genders
Male
Accepts Healthy Volunteers
No

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

Study Design

Phase
Phase 2
Study Type
Interventional
Allocation
N/A
Intervention Model
Sequential Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
NS-089/NCNP-02 		Experimental: NS-089/NCNP-02 NS-089/NCNP-02 solution for infusion (Cohort 1) NS-089/NCNP-02 solution for infusion (Cohort 2)
  • 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
    Other names:
    • Brogidirsen

Recruiting Locations

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

More Details

Status
Recruiting
Sponsor
NS Pharma, Inc.

Study Contact

Trial info
1-866-677-6276
trialinfo@nspharma.com