Actively Recruiting
NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
Led by NS Pharma, Inc. · Updated on 2026-03-06
20
Participants Needed
25
Research Sites
133 weeks
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
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
Official 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 or older 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 glucocorticoid dose for at least 3 months and expected to remain stable during the study
- Other inclusion criteria may apply
You will not qualify if you...
- Body weight under 20 kg at consent (for Part 1 screening only)
- Evidence of symptomatic cardiomyopathy
- Treatment with anabolic steroids or products containing resveratrol or adenosine triphosphate within 3 months prior to first dose
- Treatment with any investigational drug within 3 months prior to first dose or within 5 times the drug half-life, whichever is longer
- Surgery within 3 months prior to first dose or planned during study
- Previous participation in an interventional study of NS-089/NCNP-02
- Received exon skipping oligonucleotide within 1 year prior to first dose
- Other exclusion criteria may apply
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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
CONTACT
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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