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| Name | Class |
|---|---|
| Nippon Shinyaku Co., Ltd. | INDUSTRY |
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This is a Phase 1/2 study of Multiple-Ascending Dose (MAD) levels for 12 weeks of treatment followed by 24 weeks of open-label treatment with a selected dose of NS-050/NCNP-03 administered once weekly to ambulant boys with DMD, who have a DMD exon deletion amenable to exon 50 skipping.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Part 1: NS-050/NCNP-03 | Experimental | Participants will be randomized and receive NS-050/NCNP-03 intravenous (IV) infusions once weekly for 2 weeks at each of MAD levels (1.95, 5, 10, 20, 40, and 80 mg/kg). |
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| Part 1: Placebo | Placebo Comparator | Participants will be randomized and receive NS-050/NCNP-03 placebo-matching IV infusions once weekly for 2 weeks at each of MAD levels. |
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| Part 2: NS-050/NCNP-03 | Experimental | Participants will receive NS-050/NCNP-03 IV infusions once weekly for 24 weeks at the dosage selected by the Data and Safety Monitoring Board (DSMB) at the conclusion of Part 1. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| NS-050/NCNP-03 | Drug | NS-050/NCNP-03 solution for IV infusion. |
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| Measure | Description | Time Frame |
|---|---|---|
| Part 1: Overall Summary of Treatment-emergent Adverse Events (TEAEs) | TEAEs will be summarized both at the patient level for number of TEAEs, highest severity, relationship, action, and outcome, and at the TEAE level (summarizing events) by system organ class (SOC) and preferred term (PT) as well as severity, relationship, action, and outcome. The most recent version of the Medical Dictionary for Regulatory Activities (MedDRA) will be used for coding TEAEs. | Baseline up to Week 24 |
| Part 1: Area Under the Plasma Concentration Versus Time Curve (AUC) of NS-050/NCNP-03 | Blood samples will be collected at the designated time frame. Pharmacokinetic (PK) parameters of NS-050/NCNP-03 will be calculated using non-compartmental methods. | Day 1 (1st dose) for each dose level |
| Part 1: Amount of Drug Excreted in Urine of NS-050/NCNP-03 | Urine samples will be collected at the designated time frame. PK parameters of NS-050/NCNP-03 will be calculated using non-compartmental methods. | Day 1 (1st dose) for each dose level |
| Part 2: Change from baseline in skeletal muscle dystrophin protein by immunoblot (Western blot) | Baseline, Week25 |
| Measure | Description | Time Frame |
|---|---|---|
| Part 2: Change from baseline in skeletal muscle dystrophin protein by mass spectrometry | Baseline, Week25 | |
| Part 2: Change from baseline in skeletal muscle dystrophin protein levels by immunofluorescence staining | Baseline, Week25 |
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Inclusion Criteria:
Other inclusion criteria may apply.
Exclusion Criteria:
Other exclusion criteria may apply.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital Los Angeles | Los Angeles | California | 90027 | United States | ||
| Children's Hospital Colorado |
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| Placebo | Drug | NS-050/NCNP-03 placebo-matching solution for IV infusion. |
|
| Part 2: Change from baseline in percentage of exon 50-skipped mRNA of skeletal muscle dystrophin | Baseline, Week25 |
| Part 2: 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 |
| Part 2: Time to Stand (TTSTAND) | Baseline, Week13, Week25 |
| Part 2: Time to Run/Walk 10 Meters (TTRW) | Baseline, Week13, Week25 |
| Part 2: Time to Climb 4 Stairs (TTCLIMB) | Baseline, Week13, Week25 |
| Part 2: Total distance of 6 Minute Walk Test (6MWT) | Baseline, Week13, Week25 |
| Part 2: Muscle strength measured by Quantitative Muscle Testing (QMT) | Baseline, Week13, Week25 |
| Part 2: Grip/Pinch Strength | Baseline, Week13, Week25 |
| Part 2: 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 |
| Aurora |
| Colorado |
| 80011 |
| United States |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | United States |
| University of Kansas Medical Center | Kansas City | Kansas | 66103 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| University of Pittsburgh Medical Center | Pittsburgh | Pennsylvania | 15213 | United States |
| Alberta Children's Hospital | Calgary | Alberta | Canada |
| British Columbia Children's Hospital | Vancouver | British Columbia | Canada |
| London Health Sciences Centre | London | Ontario | Canada |
| National Hospital Organization Nagara Medical Center | Nagara | Gifu | 502-8558 | Japan |
| Hyogo Medical University Hospital | Nishinomiya | Hyōgo | 663-8501 | Japan |
| Miyagi Children's Hospital | Sendai | Miyagi | 989-3126 | Japan |
| NHO Osaka Toneyama Medical Center | Toyonaka | Osaka | 560-8552 | Japan |
| National Center of Neurology and Psychiatry | Kodaira | Tokyo | 187-8551 | Japan |
| Pusan National University Yangsan Hospital | Yangsan | Gyeongsangnam-do | South Korea |
| Seoul National University Hospital | Seoul | South Korea |
| Ankara Bilkent City Hospital | Ankara | Turkey (Türkiye) |
| Istanbul University | Istanbul | Turkey (Türkiye) |
| Yeditepe University Kosuyolu Hospital | Istanbul | Turkey (Türkiye) |
| 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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