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| Name | Class |
|---|---|
| Nippon Shinyaku Co., Ltd. | INDUSTRY |
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This is a Phase 3, multi-center, open-label extension study in ambulant boys with DMD who have completed the 48-week treatment period of either viltolarsen or placebo in Study NS-065/NCNP-01-301.
This Phase 3 study is a multi-center, open-label extension study in ambulant boys with DMD who have completed the 48-week treatment period of either viltolarsen or placebo in Study NS-065/NCNP-01-301. Patients will receive viltolarsen administered IV at weekly doses of 80 mg/kg.
Study NS-065/NCNP-01-302 will be comprised of a 96-week treatment period.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Viltolarsen | Experimental | Patients amenable to exon 53 skipping will receive viltolarsen intravenous (IV) infusions, weekly, at 80 mg/kg for up to 96 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Viltolarsen | Drug | Received during weekly intravenous infusions |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of participants with treatment related Adverse Events as assessed by CTCAE v4.03 | baseline to up to 96 weeks of treatment |
| Measure | Description | Time Frame |
|---|---|---|
| Time to Stand Test (TTSTAND) | Change in Time to Stand | baseline to 96 weeks of treatment |
| Time to Run/Walk 10 Meters Test (TTRW) | Change in Time to Run/Walk 10 meters |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Queensland Children's Hospital | Brisbane | Australia | ||||
| The Childrens Hospital at Westmead |
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| Label | URL |
|---|---|
| Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53) | View source |
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| baseline to 96 weeks of treatment |
| Six-minute Walk Test (6MWT) | Change in Six-minute Walk | baseline to 96 weeks of treatment |
| North Star Ambulatory Assessment (NSAA) | Change in North Star Ambulatory Assessment | baseline to 96 weeks of treatment |
| Time to Climb 4 Stairs Test (TTCLIMB) | Change in Time to Climb 4 Stairs | baseline to 96 weeks of treatment |
| Muscle Strength Measured by Hand-Held Dynamometer | Change in Muscle Strength Measured by Hand-Held Dynamometer | baseline to 96 weeks of treatment |
| Westmead |
| Australia |
| CHU de Quebec Research Centre | Québec | Canada |
| Hospital de Niños Roberto del Rio | Santiago | Chile |
| Pontificia Universidad Católica de Chile | Santiago | Chile |
| Chinese PLA General Hospital | Beijing | China |
| Hunan Children's Hospital | Changsha | China |
| Children's Hospital of Fudan University | Shanghai | China |
| Shenzhen Children's Hospital | Shenzhen | China |
| Fakultni nemocnice Hradec Kralove | Nový Hradec Králové | Czechia |
| Agia Sofia Children's Hospital | Athens | Greece |
| Hippokration General Hospital of Thessaloniki | Thessaloniki | Greece |
| Fondazione Policlinico Universitario A. Gemelli - Universita Cattolica del Sacro Cuore | Rome | Italy |
| National Center of Neurology and Psychiatry | Tokyo | Japan |
| Instituto Nacional de Pediatría | Mexico City | Mexico |
| Leids Universitair Medisch Centrum | Leiden | Netherlands |
| Radboud Universitair Medisch Centrum | Nijmegen | Netherlands |
| New Zealand Clinical Research Ltd. | Auckland | New Zealand |
| Rikshospitalet | Oslo | Norway |
| Russian National Research Medical University | Moscow | Russia |
| "Saint Petersburg State Paediatric Medical University" based at Consultative and Diagnostic Centre | Saint Petersburg | Russia |
| Tomsk National Research Medical Center of Russian Academy of Sciences | Tomsk | Russia |
| Pusan National University Yangsan Hospital | Pusan | South Korea |
| Seoul National University Hospital | Seoul | South Korea |
| Hospital Sant Joan de Deu | Barcelona | Spain |
| Hospital Universitario La Paz | Madrid | Spain |
| Yeditepe University Kosuyolu Hospital | Istanbul | Turkey (Türkiye) |
| Birmingham Heartlands Hospital | Birmingham | United Kingdom |
| Royal Hospital for Children | Glasgow | United Kingdom |
| Royal Manchester Children's Hospital | Glasgow | United Kingdom |
| University College London Institute of Child Health | London | United Kingdom |
| 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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| ID | Term |
|---|---|
| C000654848 | viltolarsen |
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