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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

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 <7 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

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.