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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

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

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.