113. 筋ジストロフィー Muscular dystrophy Clinical trials / Disease details
臨床試験数 : 646 / 薬物数 : 471 - (DrugBank : 105) / 標的遺伝子数 : 59 - 標的パスウェイ数 : 170
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2021-000122-10-GR (EUCTR) | 25/11/2021 | 13/10/2021 | A Phase 3 study of Viltolarsen in boys with Duchenne Muscular Dystrophy (DMD) | A Phase 3, Multi-center, Open-label Extension Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53X | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Viltolarsen Product Code: NS-065/NCNP-01 INN or Proposed INN: VILTOLARSEN | NS Pharma, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 74 | Phase 3 | United States;Hong Kong;Taiwan;Greece;Spain;Ukraine;Turkey;Chile;Russian Federation;United Kingdom;Italy;Mexico;Canada;Australia;Norway;Netherlands;China;Japan;New Zealand;Korea, Republic of | ||
| 2 | EUCTR2019-002076-13-NO (EUCTR) | 08/09/2021 | 18/02/2021 | This is a randomized, double-blind, Placebo-controlled sudy to evaluate the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) | A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53 | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Viltolarsen Product Code: NS-065/NCNP-01 INN or Proposed INN: VILTOLARSEN | NS Pharma, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 74 | Phase 3 | United States;Hong Kong;Taiwan;Greece;Spain;Ukraine;Turkey;Russian Federation;Chile;United Kingdom;Italy;Mexico;Canada;Australia;Norway;Netherlands;China;Japan;New Zealand;Sweden;Korea, Republic of | ||
| 3 | EUCTR2020-003653-30-ES (EUCTR) | 26/07/2021 | 15/12/2021 | A Phase 2 Open-label Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys with Duchenne Muscular Dystrophy (DMD) Compared to Natural History Controls - Galactic-53 | A Phase 2 Open-label Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys with Duchenne Muscular Dystrophy (DMD) Compared to Natural History Controls - Galactic-53 | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Viltolarsen Product Code: NS-065/NCNP-01 INN or Proposed INN: VILTOLARSEN | NS Pharma, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 20 | Phase 2 | United States;Spain;Turkey;Russian Federation;Italy;China | ||
| 4 | EUCTR2021-000122-10-NL (EUCTR) | 30/06/2021 | 08/04/2021 | A Phase 3 study of Viltolarsen in boys with Duchenne Muscular Dystrophy (DMD) | A Phase 3, Multi-center, Open-label Extension Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53X | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Viltolarsen Product Code: NS-065/NCNP-01 INN or Proposed INN: VILTOLARSEN | NS Pharma, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 74 | Phase 3 | United States;Hong Kong;Taiwan;Greece;Spain;Ukraine;Turkey;Chile;Russian Federation;United Kingdom;Italy;Mexico;Canada;Australia;Norway;Netherlands;China;Japan;New Zealand;Korea, Republic of | ||
| 5 | EUCTR2021-000122-10-ES (EUCTR) | 15/06/2021 | 30/07/2021 | A Phase 3 study of Viltolarsen in boys with Duchenne Muscular Dystrophy (DMD) | A Phase 3, Multi-center, Open-label Extension Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53X | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Viltolarsen Product Code: NS-065/NCNP-01 INN or Proposed INN: VILTOLARSEN | NS Pharma, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 74 | Phase 3 | United States;Hong Kong;Taiwan;Greece;Spain;Ukraine;Turkey;Chile;Russian Federation;United Kingdom;Italy;Mexico;Canada;Australia;Norway;Netherlands;China;Japan;New Zealand;Korea, Republic of | ||
| 6 | EUCTR2019-002076-13-GR (EUCTR) | 12/11/2020 | 24/09/2020 | This is a randomized, double-blind, Placebo-controlled sudy to evaluate the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) | A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53 | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Viltolarsen Product Code: NS-065/NCNP-01 INN or Proposed INN: VILTOLARSEN | NS Pharma, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 74 | Phase 3 | United States;Hong Kong;Taiwan;Greece;Spain;Ukraine;Turkey;Russian Federation;Chile;United Kingdom;Italy;Mexico;Canada;Australia;Norway;Netherlands;China;Japan;New Zealand;Sweden;Korea, Republic of | ||
| 7 | EUCTR2019-002076-13-NL (EUCTR) | 03/03/2020 | 25/11/2019 | This is a randomized, double-blind, Placebo-controlled sudy to evaluate the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) | A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53 | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Viltolarsen Product Code: NS-065/NCNP-01 INN or Proposed INN: VILTOLARSEN | NS Pharma, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 74 | Phase 3 | United States;Hong Kong;Taiwan;Greece;Spain;Ukraine;Turkey;Chile;Russian Federation;United Kingdom;Italy;Mexico;Canada;Australia;Norway;Netherlands;China;Japan;New Zealand;Sweden;Korea, Republic of | ||
| 8 | EUCTR2019-002076-13-GB (EUCTR) | 02/03/2020 | 16/09/2019 | This is a randomized, double-blind, Placebo-controlled sudy to evaluate the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) | A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53 | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Viltolarsen Product Code: NS-065/NCNP-01 INN or Proposed INN: VILTOLARSEN | NS Pharma, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 74 | Phase 3 | United States;Taiwan;Spain;Turkey;Chile;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Brazil;Poland;Australia;Netherlands;Japan;Sweden;Korea, Republic of | ||
| 9 | EUCTR2019-002076-13-ES (EUCTR) | 16/12/2019 | 06/11/2019 | This is a randomized, double-blind, Placebo-controlled sudy to evaluate the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) | A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53 | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Viltolarsen Product Code: NS-065/NCNP-01 INN or Proposed INN: VILTOLARSEN | NS Pharma, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 74 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Taiwan;Spain;Turkey;Russian Federation;Chile;United Kingdom;Italy;France;Canada;Poland;Belgium;Brazil;Australia;Netherlands;Japan;Sweden;Korea, Republic of | ||
| 10 | NCT03167255 (ClinicalTrials.gov) | July 6, 2017 | 22/5/2017 | Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) | A Phase II, Open-Label, Extension Study to Assess the Safety and Efficacy of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy | Drug: NS-065/NCNP-01 | NS Pharma, Inc. | Nippon Shinyaku Co., Ltd.;Cooperative International Neuromuscular Research Group;Therapeutic Research in Neuromuscular Disorders Solutions (TRiNDS) | Completed | 4 Years | 10 Years | Male | 16 | Phase 2 | United States;Canada |
| 11 | NCT02740972 (ClinicalTrials.gov) | December 2016 | 23/3/2016 | Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy | Drug: NS-065/NCNP-01;Drug: Placebo | NS Pharma, Inc. | Nippon Shinyaku Co., Ltd.;Cooperative International Neuromuscular Research Group;Therapeutic Research in Neuromuscular Disorders Solutions | Completed | 4 Years | 9 Years | Male | 16 | Phase 2 | United States;Canada | |
| 12 | NCT02081625 (ClinicalTrials.gov) | June 2013 | 5/3/2014 | Exploratory Study of NS-065/NCNP-01 in DMD | Exploratory Study of NS-065/NCNP-01 in Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: NS-065/NCNP-01 | National Center of Neurology and Psychiatry, Japan | Nippon Shinyaku Co., Ltd. | Completed | 5 Years | 18 Years | Male | 10 | Phase 1 | Japan |
| 13 | EUCTR2021-000122-10-NO (EUCTR) | 24/02/2022 | A Phase 3 study of Viltolarsen in boys with Duchenne Muscular Dystrophy (DMD) | A Phase 3, Multi-center, Open-label Extension Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53X | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Viltolarsen Product Code: NS-065/NCNP-01 INN or Proposed INN: VILTOLARSEN | NS Pharma, Inc. | NULL | NA | Female: no Male: yes | 74 | Phase 3 | United States;Taiwan;Hong Kong;Greece;Spain;Ukraine;Turkey;Russian Federation;Chile;United Kingdom;Italy;Mexico;Canada;Australia;Norway;Netherlands;China;Japan;New Zealand;Korea, Republic of | |||
| 14 | EUCTR2019-002076-13-SE (EUCTR) | 17/10/2019 | This is a randomized, double-blind, Placebo-controlled sudy to evaluate the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) | A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53 | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Viltolarsen Product Code: NS-065/NCNP-01 INN or Proposed INN: VILTOLARSEN | NS Pharma, Inc. | NULL | Not Recruiting | Female: no Male: yes | 74 | Phase 3 | United States;Taiwan;Spain;Turkey;Chile;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Brazil;Poland;Australia;Netherlands;Japan;Sweden;Korea, Republic of |