NS-050/NCNP-03 in Boys With DMD (Meteor50) (NCT06053814)
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)
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: Children's Hospital Los Angeles — Los Angeles, California
Age range: 4 Years to 15 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (19 Sites)
- Children's Hospital Los Angeles — Los Angeles, California
- Children's Hospital Colorado — Aurora, Colorado
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois
- University of Kansas Medical Center — Kansas City, Kansas
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania
- University of Pittsburgh Medical Center — Pittsburgh, Pennsylvania
- Alberta Children's Hospital — Calgary, Alberta
- British Columbia Children's Hospital — Vancouver, British Columbia
- London Health Sciences Centre — London, Ontario
- National Hospital Organization Nagara Medical Center — Nagara, Gifu
- Hyogo Medical University Hospital — Nishinomiya, Hyōgo
- Miyagi Children's Hospital — Sendai, Miyagi
- NHO Osaka Toneyama Medical Center — Toyonaka, Osaka
- National Center of Neurology and Psychiatry — Kodaira, Tokyo
- Pusan National University Yangsan Hospital — Yangsan, Gyeongsangnam-do
- Seoul National University Hospital — Seoul,
- Ankara Bilkent City Hospital — Ankara,
- Istanbul University — Istanbul,
- Yeditepe University Kosuyolu Hospital — Istanbul,
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Duchenne Muscular Dystrophy
- Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy — Little Rock, Arkansas
- Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of DMD and BMD — Columbus, Ohio
- Microdystrophin Gene Transfer Study in Adolescents and Children With DMD — Los Angeles, California
- A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD) — Little Rock, Arkansas
- Pediatric Radio Frequency Coils Generic — Cincinnati, Ohio
Explore by condition: All Duchenne Muscular Dystrophy clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.