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
This trial does not accept healthy volunteersQualifiers
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
Design model
Sequential
Treatments tested in this trial
NS-089/NCNP-02
DrugCohort 1: Part 1 Dose Level 1-3: a 4-week Treatment Phase at each treatment dose level Part 2 Single Dose Level: a 24-week Treatment Phase at the MTD of Part 1 Cohort 2: Part 2 Single Dose Level: a 24-week Treatment Phase at the MTD of Part 1
Treatment groups
20 Participants
are divided into 1 treatment groupGroup A: NS-089/NCNP-02Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Adverse Event and Adverse Drug Reaction
Time frame
through study completion, up to follow-up phone call for Part 22
Plasma pharmacokinetic (PK) parameters
Time frame
Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Maximum plasma concentration (Cmax) of NS-089/NCNP-023
Plasma pharmacokinetic (PK) parameters
Time frame
Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Time of the maximum plasma concentration (Tmax) of NS-089/NCNP-024
Plasma pharmacokinetic (PK) parameters
Time frame
Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Terminal half-life (T1/2) of NS-089/NCNP-02Secondary outcomes
1
Change from baseline in skeletal muscle dystrophin protein by mass spectrometry.
Time frame
Baseline, Week252
Change from baseline in skeletal muscle dystrophin protein levels by immunofluorescence staining.
Time frame
Baseline, Week253
Change from baseline in percentage of exon 44-skipped mRNA of skeletal muscle dystrophin
Time frame
Baseline, Week254
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.
Time frame
Baseline, Week13, Week25Sponsors and contacts
Click on the lead sponsor to view all of their trials.
NS Pharma, Inc.
Lead sponsor
Nippon Shinyaku Co., Ltd.
Collaborator
Source ClinicalTrials.gov