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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

20 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-02-22

Study Completion Date

2026-09-11

Brief Summary

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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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Detailed Description

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Conditions

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Duchenne Muscular Dystrophy Exon 44 DMD

Study Design

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Allocation Method

NA

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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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)

Group Type EXPERIMENTAL

NS-089/NCNP-02

Intervention Type 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

Interventions

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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

Intervention Type DRUG

Other Intervention Names

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Brogidirsen

Eligibility Criteria

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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.

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.
* Having received exon skipping oligonucleotide within 1 year prior to the first dose of IP or is expected to receive exon skipping oligonucleotide prior to completion of study.

Minimum Eligible Age

4 Years

Maximum Eligible Age

14 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Responsible Party

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Responsibility Role SPONSOR

Locations

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Children's Hospital Colorado

Aurora, Colorado, United States

Site Status RECRUITING

Rare Disease Research, LLC - FL

Kissimmee, Florida, United States

Site Status RECRUITING

Rare Disease Research

Atlanta, Georgia, United States

Site Status RECRUITING

Ann and Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Site Status RECRUITING

University of Kansas Medical Center (KUMC)

Kansas City, Kansas, United States

Site Status RECRUITING

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status RECRUITING

The Children's Hospital of Philadelphia (CHOP)

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

University of Pittsburgh School of Medicine

Pittsburgh, Pennsylvania, United States

Site Status RECRUITING

UT Southwestern/Children's Health

Dallas, Texas, United States

Site Status RECRUITING

Virginia Commonwealth University Health System

Richmond, Virginia, United States

Site Status RECRUITING