113. 筋ジストロフィー
[臨床試験数:567,薬物数:442(DrugBank:93),標的遺伝子数:55,標的パスウェイ数:151

Searched query = "Muscular dystrophy", "Dystrophinopathies", "Myotilinopathy", "Laminopathy", "Caveolinopathy", "LGMD1C", "Desminopathy", "Sarcoglycanopathy", "α-dystroglycanopathy", "FCMD", "Walker-Warburg syndrome", "Muscle-eye-brain disease", "Myotonic dystrophy", "Integrin α7 deficient CMD", "Rigid spine syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
5 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2019-000601-77-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
24Phase 2United States;Canada;Belgium;Spain;Ireland;Germany;Netherlands;Italy;United Kingdom
2EUCTR2019-000601-77-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
3EUCTR2019-000601-77-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
4EUCTR2019-000601-77-DE
(EUCTR)
31/03/2020A 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
45Phase 2United States;Canada;Belgium;Spain;Ireland;Netherlands;Germany;Italy;United Kingdom
5EUCTR2019-000601-77-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
70Phase 2United States;Canada;Spain;Belgium;Ireland;Netherlands;Germany;United Kingdom;Italy