Phosphorodiamidate morpholino oligomer for exon 45 skipping (DrugBank: -)
1 disease告示番号 | 疾患名(ページ内リンク) | 臨床試験数 |
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113 | 筋ジストロフィー | 18 |
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 |
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1 | EUCTR2017-004625-32-BG (EUCTR) | 03/12/2020 | 28/10/2020 | A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53Skipping 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] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: CASIMERSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: GOLODIRSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 150 | Phase 3 | United States;Czechia;Spain;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Poland;Australia;Bulgaria;Germany;Sweden | ||
2 | EUCTR2017-004625-32-SE (EUCTR) | 17/06/2020 | 09/08/2018 | A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53Skipping 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] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: CASIMERSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: GOLODIRSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 260 | Phase 3 | United States;Finland;Spain;Ireland;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Poland;Belgium;Australia;Germany;Sweden | ||
3 | EUCTR2015-002069-52-NO (EUCTR) | 07/04/2020 | 30/09/2019 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 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] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 222 | Phase 3 | Serbia;Portugal;United States;Greece;Spain;Ireland;Russian Federation;Israel;Chile;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Poland;Belgium;Australia;Denmark;Bulgaria;Germany;Norway;Sweden | ||
4 | EUCTR2015-002069-52-GR (EUCTR) | 29/11/2019 | 11/09/2019 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 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] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 222 | Phase 3 | Serbia;Portugal;United States;Greece;Spain;Ireland;Russian Federation;Israel;Chile;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Poland;Belgium;Australia;Denmark;Bulgaria;Norway;Germany;Sweden | ||
5 | EUCTR2015-002069-52-PL (EUCTR) | 11/12/2018 | 13/09/2018 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 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] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 222 | Phase 3 | Serbia;Portugal;United States;Greece;Spain;Ireland;Russian Federation;Israel;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Poland;Belgium;Australia;Denmark;Bulgaria;Norway;Germany;Sweden | ||
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 |
6 | EUCTR2017-004625-32-ES (EUCTR) | 07/09/2018 | 10/09/2018 | A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53Skipping 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] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: CASIMERSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: GOLODIRSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 150 | Phase 3 | United States;Finland;Spain;Ireland;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Poland;Belgium;Australia;Germany;Sweden | ||
7 | EUCTR2015-002069-52-BG (EUCTR) | 06/08/2018 | 11/06/2018 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 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] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 222 | Phase 3 | Norway;Germany;Sweden;France;Czech Republic;Hungary;Canada;Poland;Belgium;Australia;Denmark;Bulgaria;United States;Serbia;Portugal;Greece;Spain;Ireland;Russian Federation;Chile;Israel;United Kingdom;Italy | ||
8 | EUCTR2017-004625-32-GB (EUCTR) | 26/07/2018 | 23/01/2019 | A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53Skipping 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] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: CASIMERSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: GOLODIRSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 260 | Phase 3 | United States;Finland;Spain;Ireland;Israel;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Poland;Australia;Germany;Sweden | ||
9 | EUCTR2015-002069-52-CZ (EUCTR) | 11/09/2017 | 14/03/2017 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 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] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 222 | Phase 3 | United States;Serbia;Portugal;Greece;Spain;Ireland;Russian Federation;Chile;Israel;United Kingdom;Italy;France;Czech Republic;Hungary;Canada;Poland;Belgium;Australia;Denmark;Bulgaria;Norway;Germany;Sweden | ||
10 | EUCTR2015-002069-52-SE (EUCTR) | 26/06/2017 | 20/09/2016 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 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] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 222 | Phase 3 | Serbia;Portugal;United States;Greece;Spain;Ukraine;Ireland;Russian Federation;Israel;Chile;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Poland;Belgium;Australia;Denmark;Bulgaria;Norway;Germany;Sweden | ||
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 |
11 | EUCTR2015-002069-52-ES (EUCTR) | 11/05/2017 | 10/03/2017 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 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] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 99 | Phase 3 | United States;Canada;Spain;Israel;Germany;United Kingdom | ||
12 | EUCTR2015-002069-52-BE (EUCTR) | 28/04/2017 | 05/09/2016 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 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] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 222 | Phase 3 | United States;Serbia;Spain;Ukraine;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Poland;Belgium;Denmark;Australia;Bulgaria;Germany;Sweden | ||
13 | EUCTR2015-002069-52-DE (EUCTR) | 07/02/2017 | 24/10/2016 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 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] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 222 | Phase 3 | Serbia;Portugal;United States;Greece;Spain;Ireland;Russian Federation;Israel;Chile;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Poland;Belgium;Australia;Denmark;Bulgaria;Norway;Germany;Sweden | ||
14 | EUCTR2015-002069-52-GB (EUCTR) | 23/01/2017 | 05/09/2016 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 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] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 222 | Phase 3 | Serbia;Portugal;United States;Greece;Spain;Ireland;Russian Federation;Israel;Chile;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Poland;Belgium;Australia;Denmark;Bulgaria;Norway;Germany;Sweden | ||
15 | EUCTR2017-004625-32-DE (EUCTR) | 26/07/2018 | A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53Skipping 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] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: CASIMERSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: GOLODIRSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | NA | Female: no Male: yes | 260 | Phase 3 | United States;Czechia;Finland;Spain;Ireland;Israel;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Poland;Australia;Bulgaria;Germany;Sweden | |||
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 |
16 | EUCTR2015-002069-52-FR (EUCTR) | 12/01/2017 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 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] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | NA | Female: no Male: yes | 99 | Phase 3 | United States;France;Czech Republic;Canada;Spain;Belgium;Germany;United Kingdom | |||
17 | EUCTR2015-002069-52-IE (EUCTR) | 21/05/2019 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 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] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | NA | Female: no Male: yes | 222 | Phase 3 | United States;Serbia;Portugal;Greece;Spain;Ireland;Russian Federation;Chile;Israel;United Kingdom;Italy;France;Czech Republic;Hungary;Canada;Poland;Belgium;Australia;Denmark;Bulgaria;Norway;Germany;Sweden | |||
18 | EUCTR2017-004625-32-PL (EUCTR) | 07/11/2019 | A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53Skipping 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] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: CASIMERSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: GOLODIRSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | NA | Female: no Male: yes | 260 | Phase 3 | United States;Finland;Spain;Ireland;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Poland;Belgium;Australia;Germany;Sweden |