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

Phase 2Active Not RecruitingNCT06053814

NS Pharma, Inc.20 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Duchenne Muscular Dystrophy

Interventions

NS-050/NCNP-03DRUG

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

PlaceboDRUG

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

Eligibility

Sex: MALEMin age: 4 YearsMax age: 15 Years

Medical Language ↔ Plain English

Inclusion Criteria: * 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. Other inclusion criteria may 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.

Locations (19)

Children's Hospital Los Angeles

Los Angeles, California, United States

Children's Hospital Colorado

Aurora, Colorado, United States

Outcomes

Primary Outcomes

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.

Time frame: 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.

Time frame: 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.

Time frame: Day 1 (1st dose) for each dose level

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

Time frame: Baseline, Week25

Secondary Outcomes

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

Time frame: Baseline, Week25

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

Time frame: Baseline, Week25

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

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.

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

Chicago, Illinois, United States

University of Kansas Medical Center

Kansas City, Kansas, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, United States

Alberta Children's Hospital

Calgary, Alberta, Canada

British Columbia Children's Hospital

Vancouver, British Columbia, Canada

London Health Sciences Centre

London, Ontario, Canada

National Hospital Organization Nagara Medical Center

Nagara, Gifu, Japan

...and 9 more locations

Time frame: Baseline, Week25

Part 2: North Star Ambulatory Assessment (NSAA) score

The NSAA is a functional scale devised for use in ambulant children with Duchenne muscular dystrophy (DMD). It consists of 17 activities graded 0 (unable to perform), 1 (performs with modifications), 2 (normal movement). It assesses abilities necessary to remain ambulant that have been found to progressively deteriorate in untreated DMD patients, as well as in other muscular dystrophies such as Becker Muscular Dystrophy. NSAA Total Score ranges from 0 to 34, with a score of 34 implying normal function.