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

  
7 trials found
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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PhaseCountries
1EUCTR2019-000601-77-DE
(EUCTR)
17/08/202131/03/2020A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment Duchenne Muscular Dystrophy
MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-5051
Product Code: SRP-5051
INN or Proposed INN: SRP-5051
Other descriptive name: peptide-conjugated phosphorodiamidate morpholino oligomer for exon 51 skipping
Sarepta Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
60Phase 2United States;Canada;Spain;Belgium;Ireland;Netherlands;Germany;United Kingdom;Italy
2EUCTR2019-000601-77-NL
(EUCTR)
10/04/202025/11/2019A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsA Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment Duchenne Muscular Dystrophy
MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-5051
Product Code: SRP-5051
INN or Proposed INN: SRP-5051
Other descriptive name: peptide-conjugated phosphorodiamidate morpholino oligomer for exon 51 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
60Phase 2United States;Canada;Spain;Belgium;Ireland;Germany;Netherlands;United Kingdom;Italy
3EUCTR2019-000601-77-ES
(EUCTR)
15/01/202005/07/2019A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsA Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment Duchenne Muscular Dystrophy
MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-5051
Product Code: SRP-5051
INN or Proposed INN: SRP-5051
Other descriptive name: peptide-conjugated phosphorodiamidate morpholino oligomer for exon 51 skipping
Sarepta Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
24Phase 2United States;Canada;Belgium;Spain;Ireland;Netherlands;Germany;United Kingdom;Italy
4EUCTR2019-000601-77-IE
(EUCTR)
11/12/201926/06/2019A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsA Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment Duchenne Muscular Dystrophy
MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-5051
Product Code: SRP-5051
INN or Proposed INN: SRP-5051
Other descriptive name: peptide-conjugated phosphorodiamidate morpholino oligomer for exon 51 skipping
Sarepta Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
24Phase 2United States;Canada;Spain;Belgium;Ireland;Netherlands;Germany;United Kingdom;Italy
5EUCTR2019-000601-77-GB
(EUCTR)
04/11/201912/07/2019A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsA Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment Duchenne Muscular Dystrophy
MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-5051
Product Code: SRP-5051
INN or Proposed INN: SRP-5051
Other descriptive name: peptide-conjugated phosphorodiamidate morpholino oligomer for exon 51 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
70Phase 2United States;Canada;Spain;Belgium;Ireland;Netherlands;Germany;Italy;United Kingdom
6NCT04004065
(ClinicalTrials.gov)
June 26, 201927/6/2019Two-Part Study for Dose Determination of SRP-5051 (Vesleteplirsen) (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping TreatmentA Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, Then Dose Expansion, in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping TreatmentDuchenne Muscular DystrophyDrug: SRP-5051Sarepta Therapeutics, Inc.NULLRecruiting7 Years21 YearsMale60Phase 2United States;Belgium;Canada;Germany;Italy;Netherlands;Spain;United Kingdom
7EUCTR2019-000601-77-BE
(EUCTR)
01/08/2019A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsA Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment Duchenne Muscular Dystrophy
MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-5051
Product Code: SRP-5051
INN or Proposed INN: SRP-5051
Other descriptive name: peptide-conjugated phosphorodiamidate morpholino oligomer for exon 51 skipping
Sarepta Therapeutics, Inc.NULLNAFemale: no
Male: yes
60Phase 2United States;Canada;Spain;Belgium;Ireland;Germany;Netherlands;United Kingdom;Italy