Phosphorodiamidate Morpholino Oligomer ( DrugBank: - )
1 disease
| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 113 | 筋ジストロフィー | 34 |
113. 筋ジストロフィー
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 | EUCTR2015-002069-52-NL (EUCTR) | 01/07/2022 | 09/06/2017 | 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 | 99 | Phase 3 | United States;Greece;Spain;Ireland;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Poland;Denmark;Bulgaria;Norway;Netherlands;Germany;Sweden | ||
| 2 | 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 | ||
| 3 | EUCTR2015-002069-52-IE (EUCTR) | 11/12/2020 | 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: 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 | 222 | Phase 3 | Serbia;United States;Taiwan;Greece;Spain;Ukraine;Ireland;Israel;Russian Federation;Italy;France;Denmark;Australia;Netherlands;Korea, Republic of;Czechia;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Argentina;Belgium;Poland;Bulgaria;Norway;Germany;Sweden | ||
| 4 | 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;Bulgaria;Germany;Sweden | ||
| 5 | 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;Czechia;Spain;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Poland;Bulgaria;Germany;Sweden | ||
| 6 | 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 | ||
| 7 | 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 | ||
| 8 | 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 | ||
| 9 | 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 | ||
| 10 | 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 | United States;Serbia;Taiwan;Greece;Spain;Ukraine;Ireland;Russian Federation;Israel;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Argentina;Belgium;Poland;Bulgaria;Norway;Germany;Sweden | ||
| 11 | EUCTR2015-002069-52-DK (EUCTR) | 26/11/2019 | 25/07/2019 | 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: 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 | 222 | Phase 3 | Serbia;United States;Taiwan;Greece;Spain;Ukraine;Ireland;Israel;Russian Federation;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Argentina;Belgium;Poland;Bulgaria;Norway;Germany;Sweden | ||
| 12 | 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 | ||
| 13 | EUCTR2017-004625-32-FR (EUCTR) | 05/02/2019 | 23/07/2018 | 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 | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 260 | Phase 3 | United States;United Kingdom;Spain;Canada;Czech Republic;Sweden;Belgium;Ireland;Finland;Poland;Italy;Israel;France;Australia;Bulgaria;Germany | ||
| 14 | EUCTR2017-004625-32-BE (EUCTR) | 20/12/2018 | 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 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: 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 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Finland;Spain;Ireland;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Poland;Belgium;Australia;Germany;Sweden | ||
| 15 | 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 | Russian Federation;Hungary;United States;United Kingdom;Portugal;Spain;Greece;Canada;Czech Republic;Sweden;Netherlands;Belgium;Norway;Ireland;Poland;Denmark;Italy;Israel;Australia;Bulgaria;France;Serbia;Germany | ||
| 16 | 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 | ||
| 17 | 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: 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 | 222 | Phase 3 | United States;Serbia;Taiwan;Greece;Spain;Ukraine;Ireland;Israel;Russian Federation;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Bulgaria;Norway;Germany;Sweden | ||
| 18 | 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 | ||
| 19 | EUCTR2015-002069-52-CZ (EUCTR) | 11/09/2017 | 14/03/2017 | 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 | 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: 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 | 222 | Phase 3 | United States;Serbia;Taiwan;Greece;Spain;Ukraine;Ireland;Israel;Russian Federation;Italy;France;Denmark;Australia;Netherlands;Korea, Republic of;Czechia;United Kingdom;Hungary;Czech Republic;Mexico;Canada;Argentina;Belgium;Poland;Bulgaria;Norway;Germany;Sweden | ||
| 20 | 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 | Not Recruiting | Female: no Male: yes | 222 | Phase 3 | Portugal;Serbia;United States;Greece;Spain;Ukraine;Ireland;Chile;Israel;Russian Federation;Italy;France;Denmark;Australia;Netherlands;United Kingdom;Hungary;Czech Republic;Canada;Belgium;Poland;Bulgaria;Germany;Norway;Sweden | ||
| 21 | 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 | ||
| 22 | 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: 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 | 222 | Phase 3 | United States;Serbia;Greece;Spain;Ukraine;Ireland;Russian Federation;Israel;Italy;France;Denmark;Australia;Czechia;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Bulgaria;Georgia;Norway;Germany;Sweden | ||
| 23 | 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: 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 | 222 | Phase 3 | United States;Serbia;Taiwan;Greece;Spain;Ukraine;Ireland;Israel;Russian Federation;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Bulgaria;Norway;Germany;Sweden | ||
| 24 | 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 | ||
| 25 | EUCTR2014-002008-25-FR (EUCTR) | 06/07/2016 | 11/01/2017 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A 2-Part, Randomized, Double-Blind, Placebo-Controlled, Dose Titration, Safety, Tolerability, and Pharmacokinetics Study (Part 1) Followed by an Open-Label Efficacy and Safety Evaluation (Part 2) of SRP-4053 in Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 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-4053 Product Code: SRP-4053 INN or Proposed INN: Not Available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 48 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;Italy;United Kingdom | ||
| 26 | EUCTR2014-002008-25-IT (EUCTR) | 13/11/2014 | 10/09/2014 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A 2-Part, Randomized, Double-Blind, Placebo-Controlled, Dose Titration, Safety, Tolerability, and Pharmacokinetics Study (Part 1) Followed by an Open-Label Efficacy and Safety Evaluation (Part 2) of SRP-4053 in Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping MedDRA version: 17.0;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-4053 Product Code: SRP-4053 INN or Proposed INN: Not Available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 48 | Phase 1;Phase 2 | France;United Kingdom;Italy | ||
| 27 | EUCTR2014-002008-25-GB (EUCTR) | 10/09/2014 | 08/09/2014 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A 2-Part, Randomized, Double-Blind, Placebo-Controlled, Dose Titration, Safety, Tolerability, and Pharmacokinetics Study (Part 1) Followed by an Open-Label Efficacy and Safety Evaluation (Part 2) of SRP-4053 in Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 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-4053 Product Code: SRP-4053 INN or Proposed INN: Not Available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 48 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;United States;Italy;United Kingdom | ||
| 28 | EUCTR2006-003833-33-GB (EUCTR) | 13/06/2007 | 09/01/2009 | Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: A Phase I/II Clinical Trial Using AVI-4658 | Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: A Phase I/II Clinical Trial Using AVI-4658 | Duchenne Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy | Product Name: AVI-4658 Product Code: AVI-4658 Other descriptive name: Phosphorodiamidate Morpholino Oligomer | Imperial College, London | NULL | Not Recruiting | Female: no Male: yes | 9 | Phase 1;Phase 2 | United Kingdom | ||
| 29 | EUCTR2017-004625-32-CZ (EUCTR) | 09/09/2019 | 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 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: 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 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Finland;Spain;Ireland;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Poland;Belgium;Australia;Germany;Sweden | |||
| 30 | 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 | 60 | Phase 2 | United States;Canada;Spain;Belgium;Ireland;Germany;Netherlands;United Kingdom;Italy | |||
| 31 | 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;United Kingdom;Spain;Canada;Czech Republic;Sweden;Belgium;Ireland;Finland;Poland;Italy;Israel;Australia;France;Bulgaria;Germany | |||
| 32 | 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 | |||
| 33 | 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 | |||
| 34 | 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;Spain;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Poland;Bulgaria;Germany;Sweden |