NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexMale
Age4-14
SponsorNS Pharma, Inc.
About this trial
This is a Phase 2, open-label, multi-center, 2-part study of NS-089/NCNP-02 administered by weekly IV infusion to ambulant boys aged ≥4 to \<15 years with DMD due to mutations amenable to exon 44 skipping. Participants will receive a selected dose of NS-089/NCNP-02 administered once weekly.
The study consists of 2 parts: Part 1 and Part 2. Six participants (Cohort 1) will participate in both Part 1 and Part 2, and 14 participants (Cohort 2) will be added for Part 2.
Eligibility criteria
Qualifiers
Male ≥ 4 years and <15 years of age
Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 44 to restore the dystrophin mRNA reading frame
Able to walk independently without assistive devices
Ability to complete the TTSTAND without assistance in <20 seconds
Disqualifiers
Has a body weight of <20 kg at the time of informed consent (applies to participants screening for Part 1 only)
Evidence of symptomatic cardiomyopathy
Current or previous treatment with anabolic steroids (e.g., oxandrolone) or products containing resveratrol or adenosine triphosphate within 3 months prior to first dose of study drug
Current or previous treatment with any other investigational drug within 3 months prior to the first dose of study drug or within 5 times the half-life of a medication, whichever is longer
Trial design
Treatments tested in this trial
- NS-089/NCNP-02
Treatment groups
20 Participants
are divided into 1 treatment groupSponsors and collaborators
NS Pharma, Inc.
Lead sponsor
Nippon Shinyaku Co., Ltd.
Collaborator
Source ClinicalTrials.gov