NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
Start Date
2/22/2024
Completion Date
9/11/2026
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.
Eligibility Criteria
Age Range: 4 years to 14 years
Interventions
NS-089/NCNP-02
Conditions
Locations
Children's Hospital Colorado
Aurora, Colorado 80045
United States
Rare Disease Research, LLC - FL
Kissimmee, Florida 34746
United States
Rare Disease Research
Atlanta, Georgia 30329
United States
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois 60611
United States
University of Kansas Medical Center (KUMC)
Kansas City, Kansas 66160
United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio 45229
United States
The Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania 19104
United States
University of Pittsburgh School of Medicine
Pittsburgh, Pennsylvania 15224
United States
UT Southwestern/Children's Health
Dallas, Texas 75207
United States
Virginia Commonwealth University Health System
Richmond, Virginia 23298
United States
Perth Children's Hospital
Nedlands, Western Australia 6009
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 910-8526
Japan
National Hospital Organization Nagara Medical Center
Nagara, Gifu-shi, Gifu 502-8558
Japan
NHO Osaka Toneyama Medical Center
Toyonaka, Osaka 560-8552
Japan
Shiga General Hospital
Moriyama-shi, Shiga 524-8524
Japan
National Center of Neurology and Psychiatry
Kodaira, Tokyo 187-8551
Japan
Starship Children's Hospital
Auckland, 1023
New Zealand
Pusan National University Yangsan Hospital
Yangsan, Gyeongsangnam
South Korea
Seoul National University Hospital
Seoul,
South Korea
Ankara Bilkent City Hospital
Ankara, 06800
Turkey (Türkiye)
Yeditepe University Kosuyolu Hospital
Istanbul, 34718
Turkey (Türkiye)
S.B.U. Dr. Behcet uz Pediatric Diseases and Surgery Training and Research Hospital
Izmir, 11794
Turkey (Türkiye)