VILTOLARSEN ( DrugBank: Viltolarsen )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
113筋ジストロフィー18

113. 筋ジストロフィー


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.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2021-000122-10-GR
(EUCTR)
25/11/202113/10/2021A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
74Phase 3United 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
2EUCTR2019-002076-13-NO
(EUCTR)
08/09/202118/02/2021This 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
74Phase 3United 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
3EUCTR2020-003653-30-ES
(EUCTR)
26/07/202115/12/2021A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
20Phase 2United States;Spain;Turkey;Russian Federation;Italy;China
4EUCTR2021-000122-10-NL
(EUCTR)
30/06/202108/04/2021A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
74Phase 3United 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
5EUCTR2021-000122-10-ES
(EUCTR)
15/06/202130/07/2021A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
74Phase 3United 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
6NCT04687020
(ClinicalTrials.gov)
June 10, 202116/12/2020Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)Duchenne Muscular DystrophyDrug: ViltolarsenNS Pharma, Inc.NULLActive, not recruitingN/AN/AMale9Phase 4United States;Canada
7EUCTR2020-003653-30-IT
(EUCTR)
08/06/202108/06/2021This is an Open-label Study to evaluate the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys with Duchenne Muscular Dystrophy (DMD)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, IncNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
20Phase 2United States;Spain;Turkey;Russian Federation;China;Italy
8EUCTR2021-000122-10-IT
(EUCTR)
26/04/202108/06/2021A 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, IncNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
74Phase 3United 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
9NCT04768062
(ClinicalTrials.gov)
April 13, 202119/2/2021Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X)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)Duchenne Muscular DystrophyDrug: ViltolarsenNS Pharma, Inc.Nippon Shinyaku Co., Ltd.Enrolling by invitationN/AN/AMale74Phase 3Australia;Greece;Italy;Japan;Korea, Republic of;Netherlands;New Zealand;Russian Federation;Spain;Turkey
10EUCTR2019-002076-13-GR
(EUCTR)
12/11/202024/09/2020This 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
74Phase 3United 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
11NCT04060199
(ClinicalTrials.gov)
April 14, 202015/8/2019Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)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)Duchenne Muscular DystrophyDrug: Viltolarsen;Drug: PlaceboNS Pharma, Inc.Nippon Shinyaku Co., Ltd.Recruiting4 Years7 YearsMale74Phase 3United States;Australia;Canada;Chile;Greece;Hong Kong;Italy;Japan;Korea, Republic of;Mexico;Netherlands;New Zealand;Norway;Russian Federation;Spain;Taiwan;Turkey;Ukraine;United Kingdom;Sweden
12EUCTR2019-002076-13-NL
(EUCTR)
03/03/202025/11/2019This 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
74Phase 3United 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
13EUCTR2019-002076-13-GB
(EUCTR)
02/03/202016/09/2019This 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
74Phase 3United States;Taiwan;Spain;Turkey;Chile;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Brazil;Poland;Australia;Netherlands;Japan;Sweden;Korea, Republic of
14EUCTR2019-002076-13-IT
(EUCTR)
27/01/202005/01/2021This 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, IncNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
74Phase 3United States;Taiwan;Greece;Spain;Turkey;Chile;Russian Federation;United Kingdom;Italy;France;Canada;Poland;Brazil;Belgium;Australia;Netherlands;Japan;Sweden;Korea, Republic of
15EUCTR2019-002076-13-ES
(EUCTR)
16/12/201906/11/2019This 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.NULLAuthorised-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): noUnited States;Taiwan;Spain;Turkey;Russian Federation;Chile;United Kingdom;Italy;France;Canada;Poland;Belgium;Brazil;Australia;Netherlands;Japan;Sweden;Korea, Republic of
16NCT04337112
(ClinicalTrials.gov)
January 23, 20082/4/2020The Expanded Access Use of Viltolarsen in Duchenne Muscular Dystrophy With Confirmed Exon 53 Amenable MutationThe Expanded Access Use of Viltolarsen for the Treatment of Duchenne Muscular Dystrophy (DMD) Amenable to Exon 53 SkippingMuscular Dystrophy, Duchenne;DMDDrug: viltolarsenNS Pharma, Inc.NULLApproved for marketing3 Years12 YearsMaleNULL
17EUCTR2019-002076-13-SE
(EUCTR)
17/10/2019This 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.NULLNot RecruitingFemale: no
Male: yes
74Phase 3United States;Taiwan;Spain;Turkey;Chile;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Brazil;Poland;Australia;Netherlands;Japan;Sweden;Korea, Republic of
18EUCTR2021-000122-10-NO
(EUCTR)
24/02/2022A 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.NULLNAFemale: no
Male: yes
74Phase 3United 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