NS-050/NCNP-03 in Boys With DMD (Meteor50)

NCT06053814 | Active Not Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: NS-050/NCNP-03-101
• Study Type: interventional
• Target: 20
• Locations: 5 countries
• Sites: 19

Brief 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.

Conditions

Duchenne Muscular Dystrophy

Outcome Measures

Primary Outcomes (4)

• Part 1: Overall Summary of Treatment-emergent Adverse Events (TEAEs)

  TEAEs will be summarized both at the patient level for number of TEAEs, highest severity, relationship, action, and outcome, and at the TEAE level (summarizing events) by system organ class (SOC) and preferred term (PT) as well as severity, relationship, action, and outcome. The most recent version of the Medical Dictionary for Regulatory Activities (MedDRA) will be used for coding TEAEs.

  Baseline up to Week 24

• Part 1: Area Under the Plasma Concentration Versus Time Curve (AUC) of NS-050/NCNP-03

  Blood samples will be collected at the designated time frame. Pharmacokinetic (PK) parameters of NS-050/NCNP-03 will be calculated using non-compartmental methods.

  Day 1 (1st dose) for each dose level

• Part 1: Amount of Drug Excreted in Urine of NS-050/NCNP-03

  Urine samples will be collected at the designated time frame. PK parameters of NS-050/NCNP-03 will be calculated using non-compartmental methods.

  Day 1 (1st dose) for each dose level

• Part 2: Change from baseline in skeletal muscle dystrophin protein by immunoblot (Western blot)

  Baseline, Week25

Secondary Outcomes (11)

• Part 2: Change from baseline in skeletal muscle dystrophin protein by mass spectrometry

  Baseline, Week25

• Part 2: Change from baseline in skeletal muscle dystrophin protein levels by immunofluorescence staining

  Baseline, Week25

• Part 2: Change from baseline in percentage of exon 50-skipped mRNA of skeletal muscle dystrophin

  Baseline, Week25

• Part 2: North Star Ambulatory Assessment (NSAA) score

  Baseline, Week13, Week25

• Part 2: Time to Stand (TTSTAND)

  Baseline, Week13, Week25

Study Arms (3)

Part 1: NS-050/NCNP-03

EXPERIMENTAL

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).

Drug: NS-050/NCNP-03

Part 1: Placebo

PLACEBO COMPARATOR

Participants will be randomized and receive NS-050/NCNP-03 placebo-matching IV infusions once weekly for 2 weeks at each of MAD levels.

Drug: Placebo

Part 2: NS-050/NCNP-03

EXPERIMENTAL

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.

Drug: NS-050/NCNP-03

Interventions

NS-050/NCNP-03 DRUG

NS-050/NCNP-03 solution for IV infusion.

Part 1: NS-050/NCNP-03; Part 2: NS-050/NCNP-03

Placebo DRUG

NS-050/NCNP-03 placebo-matching solution for IV infusion.

Part 1: Placebo

Eligibility Criteria

• Age: 4 Years - 15 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Male ≥ 4 years and \<16 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• NS Pharma, Inc.: lead
• Nippon Shinyaku Co., Ltd.: collaborator

Study Sites (19)

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80011, United States

Location

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66103, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15213, United States

Location

Alberta Children's Hospital

Calgary, Alberta, Canada

Location

British Columbia Children's Hospital

Vancouver, British Columbia, Canada

Location

London Health Sciences Centre

London, Ontario, Canada

Location

National Hospital Organization Nagara Medical Center

Nagara, Gifu, 502-8558, Japan

Location

Hyogo Medical University Hospital

Nishinomiya, Hyōgo, 663-8501, Japan

Location

Miyagi Children's Hospital

Sendai, Miyagi, 989-3126, Japan

Location

NHO Osaka Toneyama Medical Center

Toyonaka, Osaka, 560-8552, Japan

Location

National Center of Neurology and Psychiatry

Kodaira, Tokyo, 187-8551, Japan

Location

Pusan National University Yangsan Hospital

Yangsan, Gyeongsangnam-do, South Korea

Location

Seoul National University Hospital

Seoul, South Korea

Location

Ankara Bilkent City Hospital

Ankara, Turkey (Türkiye)

Location

Istanbul University

Istanbul, Turkey (Türkiye)

Location

Yeditepe University Kosuyolu Hospital

Istanbul, Turkey (Türkiye)

Location

Related Links

• Please follow this link for additional information on how to participate

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 19, 2023
• First Posted: September 26, 2023
• Study Start: September 18, 2024
• Primary Completion (Estimated): May 1, 2027
• Study Completion (Estimated): March 1, 2028
• Last Updated: April 23, 2026

Record last verified: 2026-04

Locations

TU (3); JP (5); US (6); CA (3); KR (2)