NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
A Phase 2 Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of NS-089/NCNP-02 in Boys With Duchenne Muscular Dystrophy (DMD)
1 other identifier
interventional
20
7 countries
25
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Feb 2024
25 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
July 18, 2023
CompletedFirst Posted
Study publicly available on registry
August 16, 2023
CompletedStudy Start
First participant enrolled
February 22, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 11, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
September 11, 2026
March 6, 2026
March 1, 2026
2.6 years
July 18, 2023
March 4, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (10)
Adverse Event and Adverse Drug Reaction
through study completion, up to follow-up phone call for Part 2
Plasma pharmacokinetic (PK) parameters
Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Maximum plasma concentration (Cmax) of NS-089/NCNP-02
Plasma pharmacokinetic (PK) parameters
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-02
Plasma pharmacokinetic (PK) parameters
Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Terminal half-life (T1/2) of NS-089/NCNP-02
Plasma pharmacokinetic (PK) parameters
Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Area under the concentration-time curve from time 0 to the last time point (AUC0-t) of NS-089/NCNP-02
Plasma pharmacokinetic (PK) parameters
Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Area under the concentration-time curve from time 0 to infinity (AUC0-∞) of NS-089/NCNP-02
Plasma pharmacokinetic (PK) parameters
[Time Frame: Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Total body clearance (CLtot) of NS-089/NCNP-02
Plasma pharmacokinetic (PK) parameters
Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] The volume in the terminal state (Vz) of NS-089/NCNP-02
Urine pharmacokinetic parameters
Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Urinary excretion of NS-089/NCNP-02
Change from baseline in skeletal muscle dystrophin protein by immunoblot (Western blot).
Baseline, Week25
Secondary Outcomes (11)
Change from baseline in skeletal muscle dystrophin protein by mass spectrometry.
Baseline, Week25
Change from baseline in skeletal muscle dystrophin protein levels by immunofluorescence staining.
Baseline, Week25
Change from baseline in percentage of exon 44-skipped mRNA of skeletal muscle dystrophin
Baseline, Week25
North Star Ambulatory Assessment (NSAA) score
Baseline, Week13, Week25
Time to Run/Walk 10 Meters (TTRW)
Baseline, Week13, Week25
- +6 more secondary outcomes
Study Arms (1)
NS-089/NCNP-02
EXPERIMENTALExperimental: NS-089/NCNP-02 NS-089/NCNP-02 solution for infusion (Cohort 1) NS-089/NCNP-02 solution for infusion (Cohort 2)
Interventions
Cohort 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
Eligibility Criteria
You may qualify if:
- 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
- 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.
You may not qualify if:
- 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
- Surgery within the 3 months prior to the first dose of study drug or planned during the study duration
- Previously treated in an interventional study of NS-089/NCNP-02
- Having received exon skipping oligonucleotide within 1 year prior to the first dose of IP
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- NS Pharma, Inc.lead
- Nippon Shinyaku Co., Ltd.collaborator
Study Sites (25)
Children's Hospital Colorado
Aurora, Colorado, 80045, United States
Rare Disease Research, LLC - FL
Kissimmee, Florida, 34746, United States
Rare Disease Research
Atlanta, Georgia, 30329, United States
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
University of Kansas Medical Center (KUMC)
Kansas City, Kansas, 66160, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
The Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, 19104, United States
University of Pittsburgh School of Medicine
Pittsburgh, Pennsylvania, 15224, United States
UT Southwestern/Children's Health
Dallas, Texas, 75207, United States
Virginia Commonwealth University Health System
Richmond, Virginia, 23298, United States
Perth Children's Hospital
Nedlands, Western Australia, 6009, Australia
Alberta Children's Hospital
Calgary, Alberta, Canada
British Columbia Children's Hospital
Vancouver, British Columbia, Canada
London Health Sciences Centre
London, Ontario, Canada
Fukui Prefectural Hospital
Fukui-shi, Fukui, 910-8526, Japan
National Hospital Organization Nagara Medical Center
Nagara, Gifu-shi, Gifu, 502-8558, Japan
NHO Osaka Toneyama Medical Center
Toyonaka, Osaka, 560-8552, Japan
Shiga General Hospital
Moriyama-shi, Shiga, 524-8524, Japan
National Center of Neurology and Psychiatry
Kodaira, Tokyo, 187-8551, Japan
Starship Children's Hospital
Auckland, 1023, New Zealand
Pusan National University Yangsan Hospital
Yangsan, Gyeongsangnam, South Korea
Seoul National University Hospital
Seoul, South Korea
Ankara Bilkent City Hospital
Ankara, 06800, Turkey (Türkiye)
Yeditepe University Kosuyolu Hospital
Istanbul, 34718, Turkey (Türkiye)
S.B.U. Dr. Behcet uz Pediatric Diseases and Surgery Training and Research Hospital
Izmir, 11794, Turkey (Türkiye)
Related Links
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 18, 2023
First Posted
August 16, 2023
Study Start
February 22, 2024
Primary Completion (Estimated)
September 11, 2026
Study Completion (Estimated)
September 11, 2026
Last Updated
March 6, 2026
Record last verified: 2026-03