Actively Recruiting
A Phase 2 Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of NS-089/NCNP-02 in Boys With Duchenne Muscular Dystrophy (DMD)
Led by NS Pharma, Inc. · Updated on 2026-03-06
20
Participants Needed
25
Research Sites
N/A
Total Duration
On this page
Sponsors
N
NS Pharma, Inc.
Lead Sponsor
N
Nippon Shinyaku Co., Ltd.
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating NS-089/NCNP-02 in a Phase 2, open-label study involving ambulant boys aged 4 to less than 15 years with Duchenne Muscular Dystrophy (DMD) caused by mutations suitable for exon 44 skipping. The study aims to assess the safety, tolerability, pharmacokinetics, and effects of the drug on muscle dystrophin levels and physical function. This research is sponsored by NS Pharma, Inc. and involves two parts with distinct participant cohorts. The study includes two parts: Part 1 involves six participants (Cohort 1) receiving escalating doses of NS-089/NCNP-02 by weekly intravenous infusion over a 4-week treatment phase per dose level. Part 2 involves both Cohort 1 and 14 additional participants (Cohort 2) receiving a selected dose of NS-089/NCNP-02 once weekly for 24 weeks. The drug is administered as a solution for infusion, and dosing in Part 2 is based on the maximum tolerated dose determined in Part 1. Participants will undergo assessments at baseline, during treatment, and follow-up visits including pharmacokinetic blood and urine tests, muscle biopsies to measure dystrophin protein and mRNA, and physical function tests such as the North Star Ambulatory Assessment, timed walking and climbing tasks, muscle strength tests, and grip and pinch strength. Safety monitoring includes tracking adverse events throughout the study and follow-up. The overall participation lasts through the treatment phases and a follow-up phone call after Part 2 completion.
CONDITIONS
Brief Title
NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male aged 4 years and under 15 years
- Confirmed Duchenne Muscular Dystrophy mutation amenable to exon 44 skipping
- Able to walk independently without assistive devices
- Able to complete the Time to Stand test in under 20 seconds without assistance
- Stable dose of glucocorticoid for at least 3 months, expected to remain stable during the study
- Other inclusion criteria may apply
You will not qualify if you...
- Body weight under 20 kg at informed consent (for Part 1 screening only)
- Evidence of symptomatic cardiomyopathy
- Treatment with anabolic steroids or products containing resveratrol or adenosine triphosphate within 3 months before first dose
- Treatment with any other investigational drug within 3 months before first dose or within 5 times the medication half-life
- Surgery within 3 months before first dose or planned during the study
- Previous participation in an interventional NS-089/NCNP-02 study
- Received exon skipping oligonucleotide treatment within 1 year before first dose
- Other exclusion criteria may apply
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 4 weeks for each dose level in Part 1, followed by 24 weeks in Part 2
Participants receive NS-089/NCNP-02 drug treatment in sequential phases: Cohort 1 includes a 4-week treatment phase for each dose level in Part 1, followed by a 24-week treatment phase at the maximum tolerated dose in Part 2; Cohort 2 receives a 24-week treatment phase at the maximum tolerated dose in Part 2.
Visits on Day 1 and Week 4 for each dose level in Part 1; Visits on Day 1 and Week 24 in Part 2
Trial Site Locations
Total: 25 locations
1
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
2
Rare Disease Research, LLC - FL
Kissimmee, Florida, United States, 34746
Actively Recruiting
3
Rare Disease Research
Atlanta, Georgia, United States, 30329
Actively Recruiting
4
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
5
University of Kansas Medical Center (KUMC)
Kansas City, Kansas, United States, 66160
Actively Recruiting
6
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Actively Recruiting
7
The Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
8
University of Pittsburgh School of Medicine
Pittsburgh, Pennsylvania, United States, 15224
Actively Recruiting
9
UT Southwestern/Children's Health
Dallas, Texas, United States, 75207
Actively Recruiting
10
Virginia Commonwealth University Health System
Richmond, Virginia, United States, 23298
Actively Recruiting
11
Perth Children's Hospital
Nedlands, Western Australia, Australia, 6009
Not Yet Recruiting
12
Alberta Children's Hospital
Calgary, Alberta, Canada
Not Yet Recruiting
13
British Columbia Children's Hospital
Vancouver, British Columbia, Canada
Not Yet Recruiting
14
London Health Sciences Centre
London, Ontario, Canada
Actively Recruiting
15
Fukui Prefectural Hospital
Fukui-shi, Fukui, Japan, 910-8526
Not Yet Recruiting
16
National Hospital Organization Nagara Medical Center
Nagara, Gifu-shi, Gifu, Japan, 502-8558
Actively Recruiting
17
NHO Osaka Toneyama Medical Center
Toyonaka, Osaka, Japan, 560-8552
Not Yet Recruiting
18
Shiga General Hospital
Moriyama-shi, Shiga, Japan, 524-8524
Not Yet Recruiting
19
National Center of Neurology and Psychiatry
Kodaira, Tokyo, Japan, 187-8551
Not Yet Recruiting
20
Starship Children's Hospital
Auckland, New Zealand, 1023
Not Yet Recruiting
21
Pusan National University Yangsan Hospital
Yangsan, Gyeongsangnam, South Korea
Not Yet Recruiting
22
Seoul National University Hospital
Seoul, South Korea
Not Yet Recruiting
23
Ankara Bilkent City Hospital
Ankara, Turkey (Türkiye), 06800
Not Yet Recruiting
24
Yeditepe University Kosuyolu Hospital
Istanbul, Turkey (Türkiye), 34718
Not Yet Recruiting
25
S.B.U. Dr. Behcet uz Pediatric Diseases and Surgery Training and Research Hospital
Izmir, Turkey (Türkiye), 11794
Not Yet Recruiting
Research Team
T
Trial info
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
1
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