A Phase 1/2, First in Human, Multiple-dose, 2-part Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NS-050/NCNP-03 in Boys With Duchenne Muscular Dystrophy (DMD)
Status: Recruiting
Location: See all (18) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1/Phase 2
SUMMARY
This is a Phase 1/2 study of Multiple-Ascending Dose (MAD) levels for 12 weeks of treatment followed by 24 weeks of open-label treatment with a selected dose of NS-050/NCNP-03 administered once weekly to ambulant boys with DMD, who have a DMD exon deletion amenable to exon 50 skipping.
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 exon deletion in the dystrophin gene that is amenable to skipping of exon 50 to restore the dystrophin mRNA reading frame;
• Able to walk independently without assistive devices;
• Able 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
California
Children's Hospital Los Angeles
NOT_YET_RECRUITING
Los Angeles
Colorado
Children's Hospital Colorado
RECRUITING
Aurora
Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITING
Chicago
Kansas
University of Kansas Medical Center
RECRUITING
Kansas City
Pennsylvania
Children's Hospital of Philadelphia
RECRUITING
Philadelphia
University of Pittsburgh Medical Center
RECRUITING
Pittsburgh
Other Locations
Canada
Alberta Children's Hospital
NOT_YET_RECRUITING
Calgary
London Health Sciences Centre
RECRUITING
London
British Columbia Children's Hospital
NOT_YET_RECRUITING
Vancouver
Japan
National Center of Neurology and Psychiatry
RECRUITING
Kodaira
National Hospital Organization Nagara Medical Center
RECRUITING
Nagara
Hyogo Medical University Hospital
RECRUITING
Nishinomiya
NHO Osaka Toneyama Medical Center
RECRUITING
Toyonaka
Republic of Korea
Seoul National University Hospital
NOT_YET_RECRUITING
Seoul
Pusan National University Yangsan Hospital
RECRUITING
Yangsan
Turkey
Ankara Bilkent City Hospital
NOT_YET_RECRUITING
Ankara
Istanbul University
NOT_YET_RECRUITING
Istanbul
Yeditepe University Kosuyolu Hospital
NOT_YET_RECRUITING
Istanbul
Contact Information
Primary
Trial Info
trialinfo@nspharma.com
1-866-677-4276
Time Frame
Start Date:2024-09-18
Estimated Completion Date:2027-05
Participants
Target number of participants:20
Treatments
Experimental: Part 1: NS-050/NCNP-03
Participants will be randomized and receive NS-050/NCNP-03 intravenous (IV) infusions once weekly for 2 weeks at each of MAD levels (1.95, 5, 10, 20, 40, and 80 mg/kg).
Placebo_comparator: Part 1: Placebo
Participants will be randomized and receive NS-050/NCNP-03 placebo-matching IV infusions once weekly for 2 weeks at each of MAD levels.
Experimental: Part 2: NS-050/NCNP-03
Participants will receive NS-050/NCNP-03 IV infusions once weekly for 24 weeks at the dosage selected by the Data and Safety Monitoring Board (DSMB) at the conclusion of Part 1.