A Phase 2 Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of NS-089/NCNP-02 in Boys With Duchenne Muscular Dystrophy (DMD)
Status: Recruiting
Location: See all (17) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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
Participation Requirements
Sex: Male
Minimum Age: 4
Maximum Age: 14
Healthy Volunteers: f
View:
• Male ≥ 4 years and \<15 years of age
• Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 44 to restore the dystrophin mRNA reading frame
• Able to walk independently without assistive devices
• Ability to complete the TTSTAND without assistance in \<20 seconds
• 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.
• Other inclusion criteria may apply.
Locations
United States
Colorado
Children's Hospital Colorado
RECRUITING
Aurora
Florida
Rare Disease Research, LLC - FL
RECRUITING
Kissimmee
Georgia
Rare Disease Research
RECRUITING
Atlanta
Illinois
Ann and Robert H. Lurie Children's Hospital of Chicago
RECRUITING
Chicago
Kansas
University of Kansas Medical Center (KUMC)
RECRUITING
Kansas City
Ohio
Cincinnati Children's Hospital Medical Center
RECRUITING
Cincinnati
Pennsylvania
University of Pittsburgh School of Medicine
RECRUITING
Pittsburgh
Texas
UT Southwestern/Children's Health
RECRUITING
Dallas
Virginia
Virginia Commonwealth University Health System
RECRUITING
Richmond
Other Locations
Canada
Alberta Children's Hospital
NOT_YET_RECRUITING
Calgary
London Health Sciences Centre
RECRUITING
London
Japan
National Center of Neurology and Psychiatry
NOT_YET_RECRUITING
Kodaira
Shiga General Hospital
NOT_YET_RECRUITING
Moriyama-shi
National Hospital Organization Nagara Medical Center
RECRUITING
Nagara
NHO Osaka Toneyama Medical Center
NOT_YET_RECRUITING
Toyonaka
Republic of Korea
Seoul National University Hospital
NOT_YET_RECRUITING
Seoul
Pusan National University Yangsan Hospital
NOT_YET_RECRUITING
Yangsan
Contact Information
Primary
Trial info
trialinfo@nspharma.com
1-866-677-6276
Time Frame
Start Date:2024-02-22
Estimated Completion Date:2026-09-11
Participants
Target number of participants:20
Treatments
Experimental: NS-089/NCNP-02
Experimental: NS-089/NCNP-02~NS-089/NCNP-02 solution for infusion (Cohort 1)~NS-089/NCNP-02 solution for infusion (Cohort 2)