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NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
Sponsor: NS Pharma, Inc.
Summary
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.
Official title: A Phase 2 Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of NS-089/NCNP-02 in Boys With Duchenne Muscular Dystrophy (DMD)
Key Details
Gender
MALE
Age Range
4 Years - 14 Years
Study Type
INTERVENTIONAL
Enrollment
20
Start Date
2024-02-22
Completion Date
2027-02-26
Last Updated
2026-09-15
Healthy Volunteers
No
Conditions
Interventions
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
Locations (29)
Children's Hospital Colorado
Aurora, Colorado, United States
Rare Disease Research
Atlanta, Georgia, United States
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
University of Kansas Medical Center (KUMC)
Kansas City, Kansas, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Columbia University Pediatric Neuromuscular Center
New York, New York, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
The Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, United States
University of Pittsburgh School of Medicine
Pittsburgh, Pennsylvania, United States
UT Southwestern/Children's Health
Dallas, Texas, United States
Virginia Commonwealth University Health System
Richmond, Virginia, United States
Queensland Children's Hospital
South Brisbane, Queensland, Australia
Perth Children's Hospital
Nedlands, Western Australia, Australia
Alberta Children's Hospital
Calgary, Alberta, Canada
British Columbia Children's Hospital
Vancouver, British Columbia, Canada
London Health Sciences Centre
London, Ontario, Canada
Fukui Prefectural Hospital
Fukui-shi, Fukui, Japan
National Hospital Organization Nagara Medical Center
Nagara, Gifu-shi, Gifu, Japan
NHO Osaka Toneyama Medical Center
Toyonaka, Osaka, Japan
Shiga General Hospital
Moriyama-shi, Shiga, Japan
National Center of Neurology and Psychiatry
Kodaira, Tokyo, Japan
Starship Children's Hospital
Auckland, New Zealand
Seoul National University Bundang Hospital
Seongnam-si, Gyeonggi-do, South Korea
Pusan National University Yangsan Hospital
Yangsan, Gyeongsangnam, South Korea
Seoul National University Hospital
Seoul, South Korea
Ankara Bilkent City Hospital
Ankara, Turkey (Türkiye)
Istanbul University- Istanbul Faculty of Medicine
Istanbul, Turkey (Türkiye)
Yeditepe University Kosuyolu Hospital
Istanbul, Turkey (Türkiye)
S.B.U. Dr. Behcet uz Pediatric Diseases and Surgery Training and Research Hospital
Izmir, Turkey (Türkiye)