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

Duchenne Muscular Dystrophy Clinical Trial

Official title:

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)

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06053814
• Other study ID #: NS-050/NCNP-03-101
• Status: Not yet recruiting
• Phase: Phase 1/Phase 2
• Start date: July 2024
• Est. completion date: May 2027

Study information

• Verified date: February 2024
• Source: NS Pharma, Inc.
• Contact: Trial Info
• Phone: 1-866-677-4276
• Email: trialinfo[@]nspharma.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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 mutation amenable to exon 50 skipping.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 20
• Est. completion date: May 2027
• Est. primary completion date: May 2027
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 4 Years to 14 Years

Eligibility

Inclusion Criteria:

• Male = 4 years and <15 years of age;
• Confirmed DMD mutation(s) 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 <7 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.

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• NS-050/NCNP-03
NS-050/NCNP-03 solution for IV infusion.
• Placebo
NS-050/NCNP-03 placebo-matching solution for IV infusion.

Locations

Country	Name	City	State
United States	Shriners Hospital for Children	Portland	Oregon

Sponsors (2)

Lead Sponsor	Collaborator
NS Pharma, Inc.	Nippon Shinyaku Co., Ltd.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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
Primary	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
Primary	Part 2: Change from baseline in skeletal muscle dystrophin protein by immunoblot (Western blot)		Baseline, Week25
Secondary	Part 2: Change from baseline in skeletal muscle dystrophin protein by mass spectrometry		Baseline, Week25
Secondary	Part 2: Change from baseline in skeletal muscle dystrophin protein levels by immunofluorescence staining		Baseline, Week25
Secondary	Part 2: Change from baseline in percentage of exon 50-skipped mRNA of skeletal muscle dystrophin		Baseline, Week25
Secondary	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.	Baseline, Week13, Week25
Secondary	Part 2: Time to Stand (TTSTAND)		Baseline, Week13, Week25
Secondary	Part 2: Time to Run/Walk 10 Meters (TTRW)		Baseline, Week13, Week25
Secondary	Part 2: Time to Climb 4 Stairs (TTCLIMB)		Baseline, Week13, Week25
Secondary	Part 2: Total distance of 6 Minute Walk Test (6MWT)		Baseline, Week13, Week25
Secondary	Part 2: Muscle strength measured by Quantitative Muscle Testing (QMT)		Baseline, Week13, Week25
Secondary	Part 2: Grip/Pinch Strength		Baseline, Week13, Week25
Secondary	Part 2: Performance of Upper Limb (PUL) 2.0. score	The PUL 2.0 provides both a total score and sub-scores for the 3 domains (shoulder, middle, and distal) that in DMD are progressively involved with a proximal to distal gradient. The PUL includes 22 items with an entry item to define the starting functional level. The 22 items are subdivided into the high level shoulder dimension (6 items), middle level elbow dimension (9 items), and distal wrist and hand dimension (7 items). For weaker patients, a low score on the entry item (0 2) means high level items do not need to be performed. Scoring options vary across the scale between 0-1 and 0-2 according to performance. Each dimension can be scored separately with a maximum score of 12 for the high level shoulder dimension, 17 for the middle level elbow dimension, and 13 for the distal wrist and hand dimension. A total score can be achieved by adding the 3 level scores (maximum total score of 42).	Baseline, Week13, Week25