🏛️ Verified NIH Record • Updated 2026-06-12

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)

🔵 Active, Not Recruiting Phase1 / Phase2 💊 Investigational Drug / Biologic Sponsor: NS Pharma, Inc.

🩺 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: To join this study, participants generally need to meet the listed inclusion criteria (5 items) and avoid the listed exclusions (6 items).

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 Checklist

Check every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).

Usually NOT eligible if any of the following apply:

  • Exclusion Criteria:
  • Evidence of symptomatic cardiomyopathy;
  • Current or previous treatment with anabolic steroids (e.g., oxendolone, oxandrolone) or products containing resveratrol or adenosine triphosphate within 3 months prior to first dose of study drug;
  • Currently taking another investigational drug or has taken another investigational drug within 3 months prior to the first dose of study drug;
  • Surgery within the 3 months prior to the first dose of study drug or planned during the study duration;
  • Having taken any gene therapy. Other exclusion criteria may apply.

🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.

📍 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.

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💡 How to Participate

  1. Check the checklist above to see if you may qualify.
  2. Review the official NIH record below to find the coordinating site.
  3. Ask about the visit schedule, what is covered at no cost, and any travel support.
  4. Confirm with your own doctor before making a decision.
🏛️ View Official NIH Record ↗