113. Muscular dystrophy Clinical trials / Disease details
Clinical trials : 622 / Drugs : 485 - (DrugBank : 99) / Drug target genes : 59 - Drug target pathways : 168
| 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-DE (EUCTR) | 17/08/2021 | 31/03/2020 | 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 | 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 | 60 | Phase 2 | United States;Canada;Spain;Belgium;Ireland;Netherlands;Germany;United Kingdom;Italy | ||
| 2 | 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 | 60 | Phase 2 | United States;Canada;Spain;Belgium;Ireland;Germany;Netherlands;United Kingdom;Italy | ||
| 3 | EUCTR2019-000601-77-ES (EUCTR) | 15/01/2020 | 05/07/2019 | 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 | Not Recruiting | Female: no Male: yes | 24 | Phase 2 | United States;Canada;Belgium;Spain;Ireland;Netherlands;Germany;United Kingdom;Italy | ||
| 4 | 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 | ||
| 5 | 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 | ||
| 6 | NCT04004065 (ClinicalTrials.gov) | June 26, 2019 | 27/6/2019 | Two-Part Study for Dose Determination of SRP-5051 (Part A), Then Dose Expansion (Part B) in Participants 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 | Drug: SRP-5051 | Sarepta Therapeutics, Inc. | NULL | Recruiting | 7 Years | 21 Years | Male | 60 | Phase 2 | United States;Belgium;Canada;Spain;United Kingdom |
| 7 | NCT03675126 (ClinicalTrials.gov) | December 19, 2018 | 6/8/2018 | An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 | An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 | Muscular Dystrophy, Duchenne | Drug: SRP-5051 | Sarepta Therapeutics, Inc. | NULL | Terminated | 4 Years | N/A | Male | 15 | Phase 1/Phase 2 | United States;Canada |
| 8 | NCT03375255 (ClinicalTrials.gov) | February 5, 2018 | 12/12/2017 | A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 in Patients With Duchenne Muscular Dystrophy (DMD) | A Phase 1 Trial to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Treatment | Muscular Dystrophy, Duchenne | Drug: SRP-5051 | Sarepta Therapeutics, Inc. | NULL | Completed | 12 Years | N/A | Male | 15 | Phase 1 | United States;Canada |
| 9 | 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 |