Duchenne Muscular DystrophyPhase 2NCT05996003
NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
A Phase 2 study testing NS-089/NCNP-02 in people with Duchenne Muscular Dystrophy and Exon 44, run by NS Pharma, Inc. at 29 sites in 7 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 4 to 14 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy, Exon 44, DMD
- What is being tested?
- NS-089/NCNP-02
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 29 sitesin 7 countries
- Study dates
- 22 Feb 2024to 26 Feb 2027
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 to 14 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- 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.
- Other inclusion criteria may apply.
Who cannot join
- 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
- Other exclusion criteria may apply.
Trial locations and contacts
29 sites in 7 countries.
29 sites in 7 countries
United States11 sites
Rare Disease Research
Atlanta, Georgia, 30329
Children's Hospital Colorado
Aurora, Colorado, 80045
Boston Children's Hospital
Boston, Massachusetts, 02115
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
UT Southwestern/Children's Health
Dallas, Texas, 75207
University of Kansas Medical Center (KUMC)
Kansas City, Kansas, 66160
Columbia University Pediatric Neuromuscular Center
New York, New York, 10032
The Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, 19104
University of Pittsburgh School of Medicine
Pittsburgh, Pennsylvania, 15224
Virginia Commonwealth University Health System
Richmond, Virginia, 23298
Japan5 sites
Fukui Prefectural Hospital
Fukui-shi, Fukui, 910-8526
National Center of Neurology and Psychiatry
Kodaira, Tokyo, 187-8551
Shiga General Hospital
Moriyama-shi, Shiga, 524-8524
National Hospital Organization Nagara Medical Center
Nagara, Gifu-shi, Gifu, 502-8558
NHO Osaka Toneyama Medical Center
Toyonaka, Osaka, 560-8552
Turkey (Türkiye)4 sites
Ankara Bilkent City Hospital
Ankara, 06800
Istanbul University- Istanbul Faculty of Medicine
Istanbul, 34093
Yeditepe University Kosuyolu Hospital
Istanbul, 34718
S.B.U. Dr. Behcet uz Pediatric Diseases and Surgery Training and Research Hospital
Izmir, 11794
Canada3 sites
Alberta Children's Hospital
Calgary, Alberta
London Health Sciences Centre
London, Ontario
British Columbia Children's Hospital
Vancouver, British Columbia
South Korea3 sites
Seoul National University Bundang Hospital
Seongnam-si, Gyeonggi-do, 13620
Seoul National University Hospital
Seoul
Pusan National University Yangsan Hospital
Yangsan, Gyeongsangnam
Australia2 sites
Perth Children's Hospital
Nedlands, Western Australia, 6009
Queensland Children's Hospital
South Brisbane, Queensland, 4101
About this study
Official title: A Phase 2 Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of NS-089/NCNP-02 in Boys With Duchenne Muscular Dystrophy (DMD)
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.
- Conditions
- Duchenne Muscular Dystrophy, Exon 44, DMD
- Being tested
- NS-089/NCNP-02
- Keywords
- Exon 44 Skipping, DMD, Brogidirsen
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NS-089/NCNP-02Drug
Also called Brogidirsen
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
| Study group | Role | What they receive |
|---|---|---|
| NS-089/NCNP-02 | Experimental | Experimental: NS-089/NCNP-02
NS-089/NCNP-02 solution for infusion (Cohort 1)
NS-089/NCNP-02 solution for infusion (Cohort 2) Receives: Drug: NS-089/NCNP-02 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Event and Adverse Drug Reaction
Measured: through study completion, up to follow-up phone call for Part 2
Plasma pharmacokinetic (PK) parameters
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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).
Measured: Baseline, Week25
Secondary outcomes (11)
Change from baseline in skeletal muscle dystrophin protein by mass spectrometry.
Measured: Baseline, Week25
Change from baseline in skeletal muscle dystrophin protein levels by immunofluorescence staining.
Measured: Baseline, Week25
Change from baseline in percentage of exon 44-skipped mRNA of skeletal muscle dystrophin
Measured: Baseline, Week25
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.
Measured: Baseline, Week13, Week25
Time to Run/Walk 10 Meters (TTRW)
Measured: Baseline, Week13, Week25
Time to Stand (TTSTAND)
Measured: Baseline, Week13, Week25
Total distance of 6 Minute Walk Test (6MWT)
Measured: Baseline, Week13, Week25
Time to Climb 4 Stairs (TTCLIMB)
Measured: Baseline, Week13, Week25
Muscle strength measured by Quantitative Muscle Testing (QMT)
Measured: Baseline, Week13, Week25
Grip and pinch strength
Measured: Baseline, Week13, Week25
Performance of Upper Limb (PUL) 2.0. score
The PUL 2.0 provides both a total score and sub-scores for the 3 domains (shoulder, middle, and distal) that in DMD are progressively involved with a proximal to distal gradient. The PUL includes 22 items with an entry item to define the starting functional level. The 22 items are subdivided into the high-level shoulder dimension (6 items), middle level elbow dimension (9 items), and distal wrist and hand dimension (7 items). For weaker patients, a low score on the entry item (0-2) means high level items do not need to be performed. Scoring options vary across the scale between 0-1 and 0-2 according to performance. Each dimension can be scored separately with a maximum score of 12 for the high-level shoulder dimension, 17 for the middle level elbow dimension, and 13 for the distal wrist and hand dimension. A total score can be achieved by adding the 3 level scores (maximum total score of 42).
Measured: Baseline, Week13, Week25
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Aug 2023
- Study start22 Feb 2024
- Primary completion26 Feb 2027 (estimated)
- Study completion26 Feb 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- NS Pharma, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Nippon Shinyaku Co., Ltd.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05996003
- Sponsor's protocol ID
- NS-089/NCNP-02-201
- Status last verified
- Sep 2026
- First submitted
- 18 Jul 2023
- Last updated
- 15 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No