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

Purpose

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.

Condition

  • Duchenne Muscular Dystrophy

Eligibility

Eligible Ages
Between 4 Years and 14 Years
Eligible Genders
Male
Accepts Healthy Volunteers
No

Inclusion Criteria

  • Male ≥ 4 years and <15 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.

Study Design

Phase
Phase 1/Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Part 1: NS-050/NCNP-03 		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
    NS-050/NCNP-03 solution for IV infusion.
Placebo Comparator
Part 1: Placebo 		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
    NS-050/NCNP-03 placebo-matching solution for IV infusion.
Experimental
Part 2: NS-050/NCNP-03 		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
    NS-050/NCNP-03 solution for IV infusion.

Recruiting Locations

University of Kansas Medical Center
Kansas City, Kansas 66103
Contact:
Alex McNeil
913-945-9943
asteinkamp@kumc.edu

More Details

Status
Recruiting
Sponsor
NS Pharma, Inc.

Study Contact

Trial Info
1-866-677-4276
trialinfo@nspharma.com