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 | EUCTR2018-001762-42-DK (EUCTR) | 18/12/2019 | 30/10/2019 | A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophy | A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping | Duchenne Muscular Dystrophy MedDRA version: 20.0;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 Product Code: AVI-4658 INN or Proposed INN: ETEPLIRSEN | Sarepta Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 154 | Phase 3 | United States;Serbia;Taiwan;Greece;Spain;Ukraine;Ireland;Russian Federation;Colombia;Italy;Switzerland;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;Slovenia;Turkey;United Kingdom;Hungary;Mexico;Canada;Belgium;Poland;Romania;Germany;Norway;New Zealand;Sweden | ||
| 2 | EUCTR2018-001762-42-ES (EUCTR) | 29/10/2019 | 09/08/2019 | A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophy | A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping | 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] | Trade Name: Exondys 51 Product Name: Eteplirsen Product Code: AVI-4658 INN or Proposed INN: ETEPLIRSEN | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 152 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | Taiwan;Hong Kong;Spain;Ireland;Turkey;Russian Federation;Chile;Colombia;Italy;United Kingdom;France;Canada;Argentina;Belgium;Peru;Australia;Netherlands;Germany;Korea, Republic of | ||
| 3 | EUCTR2019-000337-39-BE (EUCTR) | 03/06/2019 | 08/04/2019 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients who have completed study 4658-102 | An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients with Duchenne Muscular Dystrophy Who Have Completed Study 4658-102 | Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping MedDRA version: 20.0;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 Product Code: AVI-4658 INN or Proposed INN: ETEPLIRSEN | Sarepta Therapeutics, Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 15 | Phase 2 | France;Belgium;United Kingdom | ||
| 4 | EUCTR2016-000951-29-IT (EUCTR) | 13/06/2017 | 07/02/2018 | A research study of a new investigational medicinal product for the treatment of young Duchenne Muscular Dystrophy patients | An Open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping - N/A | Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping MedDRA version: 20.0;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 Product Code: AVI-4658 INN or Proposed INN: eteplirsen Other descriptive name: ETEPLIRSEN | SAREPTA THERAPEUTICS, INC. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 12 | Phase 2 | France;Belgium;Germany;United Kingdom;Italy | ||
| 5 | EUCTR2016-000951-29-FR (EUCTR) | 01/06/2017 | 17/07/2017 | A research study of a new investigational medicinal product for the treatment of young Duchenne Muscular Dystrophy patients | An Open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping | Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping MedDRA version: 20.0;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 Product Code: AVI-4658 INN or Proposed INN: eteplirsen Other descriptive name: ETEPLIRSEN | Sarepta Therapeutics, Inc | NULL | Not Recruiting | Female: no Male: yes | 12 | Phase 1 | France;Belgium;Germany;Italy;United Kingdom | ||
| 6 | 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 | |||
| 7 | 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 | |||
| 8 | EUCTR2018-001762-42-FR (EUCTR) | 08/07/2019 | A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophy | A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping | 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] | Trade Name: Exondys 51 Product Name: Eteplirsen Product Code: AVI-4658 INN or Proposed INN: ETEPLIRSEN | Sarepta Therapeutics, Inc. | NULL | NA | Female: no Male: yes | 152 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | Taiwan;Hong Kong;Spain;Ireland;Turkey;Russian Federation;Chile;Colombia;Italy;United Kingdom;France;Canada;Argentina;Belgium;Peru;Australia;Netherlands;Germany;New Zealand;Korea, Republic of | |||
| 9 | 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 | |||
| 10 | 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 | |||
| 11 | EUCTR2019-000337-39-FR (EUCTR) | 05/04/2019 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients who have completed study 4658-102 | An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients with Duchenne Muscular Dystrophy Who Have Completed Study 4658-102 | Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping MedDRA version: 20.0;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 Product Code: AVI-4658 INN or Proposed INN: ETEPLIRSEN | Sarepta Therapeutics, Inc | NULL | NA | Female: no Male: yes | 15 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;Belgium;United Kingdom | |||
| 12 | 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 |