Peptide-conjugated phosphorodiamidate morpholino oligomer for exon 51 skipping (DrugBank: -)
1 disease| ID | Disease name (Link within this page) | Number of trials |
|---|---|---|
| 113 | Muscular dystrophy | 5 |
| 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 | EUCTR2019-000601-77-NL (EUCTR) | 10/04/2020 | 25/11/2019 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 24 | Phase 2 | United States;Canada;Belgium;Spain;Ireland;Germany;Netherlands;Italy;United Kingdom | ||
| 2 | EUCTR2019-000601-77-IE (EUCTR) | 11/12/2019 | 26/06/2019 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | 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. | NULL | Not Recruiting | Female: no Male: yes | 24 | Phase 2 | United States;Canada;Spain;Belgium;Ireland;Netherlands;Germany;United Kingdom;Italy | ||
| 3 | EUCTR2019-000601-77-GB (EUCTR) | 04/11/2019 | 12/07/2019 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 70 | Phase 2 | United States;Canada;Spain;Belgium;Ireland;Netherlands;Germany;Italy;United Kingdom | ||
| 4 | EUCTR2019-000601-77-BE (EUCTR) | 01/08/2019 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | 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. | NULL | NA | Female: no Male: yes | 70 | Phase 2 | United States;Canada;Spain;Belgium;Ireland;Netherlands;Germany;United Kingdom;Italy | |||
| 5 | EUCTR2019-000601-77-DE (EUCTR) | 31/03/2020 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | 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. | NULL | NA | Female: no Male: yes | 45 | Phase 2 | United States;Canada;Belgium;Spain;Ireland;Netherlands;Germany;Italy;United Kingdom |