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| Name | Class |
|---|---|
| Nippon Shinyaku Co., Ltd. | INDUSTRY |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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) |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| NS-089/NCNP-02 | Drug | 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Trial info | Contact | 1-866-677-6276 | trialinfo@nspharma.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital Colorado | Recruiting | Aurora | Colorado | 80045 | United States |
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| Label | URL |
|---|---|
| Please follow this link for additional information on how to participate | View source |
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| 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 |
| Change from baseline in percentage of exon 44-skipped mRNA of skeletal muscle dystrophin | 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. | Baseline, Week13, Week25 |
| Time to Run/Walk 10 Meters (TTRW) | Baseline, Week13, Week25 |
| Time to Stand (TTSTAND) | Baseline, Week13, Week25 |
| Total distance of 6 Minute Walk Test (6MWT) | Baseline, Week13, Week25 |
| Time to Climb 4 Stairs (TTCLIMB) | Baseline, Week13, Week25 |
| Muscle strength measured by Quantitative Muscle Testing (QMT) | Baseline, Week13, Week25 |
| Grip and pinch strength | 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). | Baseline, Week13, Week25 |
| Rare Disease Research, LLC - FL | Recruiting | Kissimmee | Florida | 34746 | United States |
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| Rare Disease Research | Recruiting | Atlanta | Georgia | 30329 | United States |
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| Ann and Robert H. Lurie Children's Hospital of Chicago | Recruiting | Chicago | Illinois | 60611 | United States |
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| University of Kansas Medical Center (KUMC) | Recruiting | Kansas City | Kansas | 66160 | United States |
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| Cincinnati Children's Hospital Medical Center | Recruiting | Cincinnati | Ohio | 45229 | United States |
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| The Children's Hospital of Philadelphia (CHOP) | Recruiting | Philadelphia | Pennsylvania | 19104 | United States |
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| University of Pittsburgh School of Medicine | Recruiting | Pittsburgh | Pennsylvania | 15224 | United States |
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| UT Southwestern/Children's Health | Recruiting | Dallas | Texas | 75207 | United States |
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| Virginia Commonwealth University Health System | Recruiting | Richmond | Virginia | 23298 | United States |
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| Perth Children's Hospital | Not yet recruiting | Nedlands | Western Australia | 6009 | Australia |
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| Alberta Children's Hospital | Not yet recruiting | Calgary | Alberta | Canada |
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| British Columbia Children's Hospital | Not yet recruiting | Vancouver | British Columbia | Canada |
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| London Health Sciences Centre | Recruiting | London | Ontario | Canada |
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| Fukui Prefectural Hospital | Not yet recruiting | Fukui-shi | Fukui | 910-8526 | Japan |
| National Hospital Organization Nagara Medical Center | Recruiting | Nagara | Gifu-shi, Gifu | 502-8558 | Japan |
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| NHO Osaka Toneyama Medical Center | Not yet recruiting | Toyonaka | Osaka | 560-8552 | Japan |
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| Shiga General Hospital | Not yet recruiting | Moriyama-shi | Shiga | 524-8524 | Japan |
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| National Center of Neurology and Psychiatry | Not yet recruiting | Kodaira | Tokyo | 187-8551 | Japan |
| Starship Children's Hospital | Not yet recruiting | Auckland | 1023 | New Zealand |
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| Pusan National University Yangsan Hospital | Not yet recruiting | Yangsan | Gyeongsangnam | South Korea |
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| Seoul National University Hospital | Not yet recruiting | Seoul | South Korea |
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| Ankara Bilkent City Hospital | Not yet recruiting | Ankara | 06800 | Turkey (Türkiye) |
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| Yeditepe University Kosuyolu Hospital | Not yet recruiting | Istanbul | 34718 | Turkey (Türkiye) |
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| S.B.U. Dr. Behcet uz Pediatric Diseases and Surgery Training and Research Hospital | Not yet recruiting | Izmir | 11794 | Turkey (Türkiye) |
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| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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