NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
Study Overview
- Age
- 4–14 years
- Phase
- Phase 2
- Sponsor
- NS Pharma
- Therapeutic Approach
- Exon Skipping
- Variant Requirement
- Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 44 to restore the dystrophin mRNA reading frame.
- Eligible Sex
- Male
- Ambulation
- Ambulatory
- Study Start (Actual)
- 2024-02-22
- Primary Completion (Estimated)
- 2026-09-11
- Study Completion (Estimated)
- 2026-09-11
- Enrollment (Estimated)
- 20
- Countries
- United StatesAustraliaCanadaJapanNew ZealandSouth KoreaTurkiye
Study Requirements and Criteria
Steroid Use
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.
Inclusion Criteria
- Male ≥ 4 years and <15 years of age
- Able to walk independently without assistive devices
- Ability to complete the TTSTAND without assistance in <20 seconds
- 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 received exon skipping oligonucleotide within 1 year prior to the first dose of IP
- Other exclusion criteria may apply.
Contacts and Locations
Contacts and Locations
Contact
- Name: Trial info
- Phone Number: 1-866-677-6276
- Email: [email protected]
Locations
This study has 25 locations
United States
Colorado Locations
Aurora, Colorado, United States, 80045
Actively Recruiting
Children's Hospital Colorado
Contact: Hannah Kleiner
Contact Mail: [email protected]
Florida Locations
Kissimmee, Florida, United States, 34746
Actively Recruiting
Rare Disease Research, LLC - FL
Contact: Marcial Almaraz
Contact Mail: [email protected]
Georgia Locations
Atlanta, Georgia, United States, 30329
Actively Recruiting
Rare Disease Research
Contact: Julia Zhu
Contact Mail: [email protected]
Illinois Locations
Chicago, Illinois, United States, 60611
Actively Recruiting
Ann and Robert H. Lurie Children's Hospital of Chicago
Contact: Raven Hill
Contact Mail: [email protected]
Kansas Locations
Kansas City, Kansas, United States, 66160
Actively Recruiting
University of Kansas Medical Center (KUMC)
Contact: Rebecca Clay
Contact Mail: [email protected]
Ohio Locations
Cincinnati, Ohio, United States, 45229
Actively Recruiting
Cincinnati Children's Hospital Medical Center
Contact: Angela Edmondson
Contact Mail: [email protected]
Pennsylvania Locations
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
The Children's Hospital of Philadelphia (CHOP)
Contact: Karen Monono
Contact Mail: [email protected]
Pittsburgh, Pennsylvania, United States, 15224
Actively Recruiting
University of Pittsburgh School of Medicine
Contact: Vibha Chauhan
Contact Mail: [email protected]
Texas Locations
Dallas, Texas, United States, 75207
Actively Recruiting
UT Southwestern/Children's Health
Contact: Deniece Winston
Contact Mail: [email protected]
Virginia Locations
Richmond, Virginia, United States, 23298
Actively Recruiting
Virginia Commonwealth University Health System
Contact: Andrea Jewell
Contact Mail: [email protected]
Australia
Western Australia Locations
Nedlands, Western Australia, Australia, 6009
Not Yet Recruiting
Perth Children's Hospital
Contact: Jane Jones
Contact Mail: [email protected]
Canada
Alberta Locations
Calgary, Alberta, Canada
Not Yet Recruiting
Alberta Children's Hospital
Contact: Julie Dao
Contact Mail: [email protected]
British Columbia Locations
Vancouver, British Columbia, Canada
Not Yet Recruiting
British Columbia Children's Hospital
Contact: Gabe Massarotto
Contact Mail: [email protected]
Ontario Locations
London, Ontario, Canada
Actively Recruiting
London Health Sciences Centre
Contact: Rhiannon Hicks
Contact Mail: [email protected]
Japan
Fukui Locations
Fukui-shi, Fukui, Japan, 910-8526
Not Yet Recruiting
Fukui Prefectural Hospital
Gifu-shi, Gifu Locations
Nagara, Gifu-shi, Gifu, Japan, 502-8558
Actively Recruiting
National Hospital Organization Nagara Medical Center
Contact: Clinical Trial Management Office
Contact Mail: [email protected]
Osaka Locations
Toyonaka, Osaka, Japan, 560-8552
Not Yet Recruiting
NHO Osaka Toneyama Medical Center
Contact: Clinical Trial Office
Contact Mail: [email protected]
Shiga Locations
Moriyama-shi, Shiga, Japan, 524-8524
Not Yet Recruiting
Shiga General Hospital
Contact: Children's Building
Contact Mail: [email protected]
Tokyo Locations
Kodaira, Tokyo, Japan, 187-8551
Not Yet Recruiting
National Center of Neurology and Psychiatry
New Zealand
Auckland, New Zealand, 1023
Not Yet Recruiting
Starship Children's Hospital
Contact: Margaret Joppa
Contact Mail: [email protected]
South Korea
Gyeongsangnam Locations
Yangsan, Gyeongsangnam, South Korea
Not Yet Recruiting
Pusan National University Yangsan Hospital
Contact: JaeYoon Kim
Contact Mail: [email protected]
Seoul, South Korea
Not Yet Recruiting
Seoul National University Hospital
Contact: Hyeon Ju Kwon
Contact Mail: [email protected]; [email protected]
Turkey (Türkiye)
Ankara, Turkey (Türkiye), 06800
Not Yet Recruiting
Ankara Bilkent City Hospital
Contact: Gozde Akgun
Contact Mail: [email protected]
Istanbul, Turkey (Türkiye), 34718
Not Yet Recruiting
Yeditepe University Kosuyolu Hospital
Contact: Onur Güner
Contact Mail: [email protected]
Izmir, Turkey (Türkiye), 11794
Not Yet Recruiting
S.B.U. Dr. Behcet uz Pediatric Diseases and Surgery Training and Research Hospital
Contact: Zeynep Erturk
Contact Mail: [email protected]
Clinical Trial Registry
NCT ID
NCT05996003This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.