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
| 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 | EUCTR2016-005001-39-Outside-EU/EEA (EUCTR) | 16/03/2017 | A research study of a new investigational medicinal product for the treatment of patients with Duchenne Muscular Dystrophy. | Open-Label, Multiple-Dose, Efficacy, Safety, and Tolerability Study of Eteplirsen in Subjects with Duchenne Muscular Dystrophy who Participated in Study 4658-us-201 | Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping MedDRA version: 19.1;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] | Trade Name: Exondys 51 Product Name: Eteplirsen Injection Product Code: AVI-4658 INN or Proposed INN: AVI-4658 Other descriptive name: ETEPLIRSEN | Sarepta Therapeutics, Inc. | NULL | NA | Female: no Male: yes | 12 | Phase 2 | United States | |||
| 2 | EUCTR2016-005000-26-Outside-EU/EEA (EUCTR) | 16/03/2017 | A research study of a new investigational medicinal product for the treatment of patients with Duchenne Muscular Dystrophy. | A Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Efficacy, Safety, Tolerability, and Pharmacokinetics Study of AVI-4658 (Eteplirsen), a Phosphorodiamidate Morpholino Oligomer, Administered Over 28 Weeks in the Treatment of Ambulant Subjects with Duchenne Muscular Dystrophy | Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping MedDRA version: 19.1;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] | Trade Name: Exondys 51 Product Name: Eteplirsen Injection Product Code: AVI-4658 INN or Proposed INN: AVI-4658 Other descriptive name: ETEPLIRSEN | Sarepta Therapeutics, Inc. | NULL | NA | Female: no Male: yes | 12 | Phase 2 | United States | |||
| 3 | EUCTR2016-005024-28-Outside-EU/EEA (EUCTR) | 28/03/2017 | Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy | An Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Eteplirsen in Patients With Advanced Stage Duchenne Muscular Dystrophy | Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping MedDRA version: 19.1;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] | Trade Name: EXONDYS 51™ Product Name: Eteplirsen Injection Product Code: AVI-4658 INN or Proposed INN: AVI-4658 Other descriptive name: ETEPLIRSEN | Sarepta Therapeutics, Inc. | NULL | NA | Female: no Male: yes | 24 | Phase 2 | United States | |||
| 4 | EUCTR2016-005002-19-Outside-EU/EEA (EUCTR) | 28/03/2017 | Confirmatory Study of Eteplirsen in DMD Patients (PROMOVI) | An Open-Label, Multi-Center, Study With a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy | Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping MedDRA version: 19.1;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] | Trade Name: EXONDYS 51™ Product Name: Eteplirsen Injection Product Code: AVI-4658 INN or Proposed INN: AVI-4658 Other descriptive name: ETEPLIRSEN | Sarepta Therapeutics, Inc. | NULL | NA | Female: no Male: yes | 160 | Phase 3 | United States | |||
| 5 | EUCTR2016-005023-92-Outside-EU/EEA (EUCTR) | 28/03/2017 | Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy | An Open-Label, Multi-Center Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early Stage Duchenne Muscular Dystrophy | Patients with Duchenne Muscular Dystropy Amenable to Exon 51 Skipping MedDRA version: 19.1;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] | Trade Name: EXONDYS 51™ Product Name: Eteplirsen Injection Product Code: AVI-4658 INN or Proposed INN: AVI-4658 Other descriptive name: ETEPLIRSEN | Sarepta Therapeutics, Inc. | NULL | NA | Female: no Male: yes | 40 | Phase 2 | United States |