PTC743 ( DrugBank: - )
2 diseases
| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 18 | 脊髄小脳変性症（多系統萎縮症を除く。） | 4 |
| 21 | ミトコンドリア病 | 6 |
18. 脊髄小脳変性症（多系統萎縮症を除く。）
臨床試験数 : 76 / 薬物数 : 98 - (DrugBank : 31) / 標的遺伝子数 : 44 - 標的パスウェイ数 : 65
| 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 | EUCTR2020-002812-36-ES (EUCTR) | 02/06/2021 | 19/11/2020 | A study to determine if Vatiquinone, the study drug, is safe and effective to treat a neurological condition called Friedreich ataxia | A Randomized, Parallel-Arm, Double-Blind, Placebo-Controlled Study with Open-Label Extension to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Friedreich ataxia (MOVE-FA) - MOVE-FA | Friedreich Ataxia (FA) MedDRA version: 20.0;Level: PT;Classification code 10017374;Term: Friedreich's ataxia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Vatiquinone Product Code: PTC743 INN or Proposed INN: VATIQUINONE Other descriptive name: alpha-tocotrienolquinone | PTC Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 126 | Phase 2;Phase 3 | United States;France;Canada;Brazil;Spain;Australia;Germany;Italy | ||
| 2 | EUCTR2020-002812-36-IT (EUCTR) | 14/04/2021 | 04/06/2021 | A study to determine if Vatiquinone, the study drug, is safe and effective to treat a neurological condition called Friedreich ataxia | A Randomized, Parallel-Arm, Double-Blind, Placebo-Controlled Study with Open-Label Extension to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Friedreich ataxia (MOVE-FA) - MOVE-FA | Friedreich Ataxia (FA) MedDRA version: 20.0;Level: PT;Classification code 10017374;Term: Friedreich's ataxia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Vatiquinone Product Code: [PTC743] INN or Proposed INN: Vatiquinone | PTC THERAPEUTICS INC. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 126 | Phase 2;Phase 3 | France;United States;Canada;Spain;Brazil;Australia;Germany;Italy | ||
| 3 | EUCTR2020-002812-36-FR (EUCTR) | 18/03/2021 | 15/01/2021 | A study to determine if Vatiquinone, the study drug, is safe and effective to treat a neurological condition called Friedreich ataxia | A Randomized, Parallel-Arm, Double-Blind, Placebo-Controlled Study with Open-Label Extension to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Friedreich ataxia (MOVE-FA) - MOVE-FA | Friedreich Ataxia (FA) MedDRA version: 20.0;Level: PT;Classification code 10017374;Term: Friedreich's ataxia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Vatiquinone Product Code: PTC743 INN or Proposed INN: VATIQUINONE Other descriptive name: alpha-tocotrienolquinone | PTC Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 126 | Phase 2;Phase 3 | United States;France;Canada;Spain;Brazil;Australia;Germany;Italy;New Zealand | ||
| 4 | EUCTR2020-002812-36-DE (EUCTR) | 18/02/2021 | 06/11/2020 | A study to determine if Vatiquinone, the study drug, is safe and effective to treat a neurological condition called Friedreich ataxia | A Randomized, Parallel-Arm, Double-Blind, Placebo-Controlled Study with Open-Label Extension to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Friedreich ataxia (MOVE-FA) - MOVE-FA | Friedreich Ataxia (FA) MedDRA version: 20.0;Level: PT;Classification code 10017374;Term: Friedreich's ataxia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Vatiquinone Product Code: PTC743 INN or Proposed INN: VATIQUINONE Other descriptive name: alpha-tocotrienolquinone | PTC Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 126 | Phase 2;Phase 3 | France;United States;Canada;Spain;Brazil;Australia;Germany;Italy;New Zealand |
21. ミトコンドリア病
臨床試験数 : 39 / 薬物数 : 42 - (DrugBank : 32) / 標的遺伝子数 : 47 - 標的パスウェイ数 : 67
| 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 | NCT05218655 (ClinicalTrials.gov) | June 21, 2022 | 19/1/2022 | A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease | An Open-Label, Safety Study for Previously Treated Vatiquinone (PTC743) Subjects With Inherited Mitochondrial Disease | Inherited Mitochondrial Disease | Drug: Vatiquinone | PTC Therapeutics | NULL | Enrolling by invitation | N/A | N/A | All | 200 | Phase 3 | United States |
| 2 | EUCTR2020-002100-39-ES (EUCTR) | 26/07/2021 | 09/02/2021 | A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy | Efficacy and Safety Study of Vatiquinone for the Treatment of Mitochondrial Disease Subjects With Refractory Epilepsy (MIT-E) - MIT-E | Genetically determined mitochondrial disease and associated refractory epilepsy MedDRA version: 20.0;Level: LLT;Classification code 10077953;Term: Refractory epilepsy;System Organ Class: 100000004852 MedDRA version: 20.0;Level: HLT;Classification code 10052637;Term: Genetic mitochondrial abnormalities NEC;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Vatiquinone Product Code: PTC743 INN or Proposed INN: VATIQUINONE Other descriptive name: EPI743; alpha-tocotrienol quinone | PTC THERAPEUTICS, INC. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 80 | Phase 2;Phase 3 | France;United States;Spain;Germany;United Kingdom;Italy | ||
| 3 | EUCTR2020-002100-39-IT (EUCTR) | 14/04/2021 | 04/06/2021 | A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy | Efficacy and Safety Study of Vatiquinone for the Treatment of Mitochondrial Disease Subjects With Refractory Epilepsy (MIT-E) - MIT-E | Genetically determined mitochondrial disease and associated refractory epilepsy MedDRA version: 20.0;Level: LLT;Classification code 10077953;Term: Refractory epilepsy;System Organ Class: 100000004852 MedDRA version: 20.0;Level: HLT;Classification code 10052637;Term: Genetic mitochondrial abnormalities NEC;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Vatiquinone Product Code: [PTC743] INN or Proposed INN: VATIQUINONE | PTC THERAPEUTICS INC. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 80 | Phase 2;Phase 3 | France;United States;Spain;Germany;United Kingdom;Italy;Sweden | ||
| 4 | EUCTR2020-002100-39-FR (EUCTR) | 25/02/2021 | A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy | Efficacy and Safety Study of Vatiquinone for the Treatment of Mitochondrial Disease Subjects With Refractory Epilepsy (MIT-E) - MIT-E | Genetically determined mitochondrial disease and associated refractory epilepsy MedDRA version: 20.0;Level: LLT;Classification code 10077953;Term: Refractory epilepsy;System Organ Class: 100000004852 MedDRA version: 20.0;Level: HLT;Classification code 10052637;Term: Genetic mitochondrial abnormalities NEC;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Vatiquinone Product Code: PTC743 INN or Proposed INN: VATIQUINONE Other descriptive name: EPI743; alpha-tocotrienol quinone | PTC THERAPEUTICS, INC. | NULL | NA | Female: yes Male: yes | 80 | Phase 2;Phase 3 | United States;France;Spain;Germany;United Kingdom;Italy | |||
| 5 | EUCTR2020-002100-39-SE (EUCTR) | 14/12/2021 | A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy | Efficacy and Safety Study of Vatiquinone for the Treatment of Mitochondrial Disease Subjects With Refractory Epilepsy (MIT-E) - MIT-E | Genetically determined mitochondrial disease and associated refractory epilepsy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Vatiquinone Product Code: PTC743 INN or Proposed INN: VATIQUINONE Other descriptive name: EPI743; alpha-tocotrienol quinone | PTC THERAPEUTICS, INC. | NULL | NA | Female: yes Male: yes | 88 | Phase 2;Phase 3 | France;United States;Canada;Spain;Poland;Australia;Russian Federation;Germany;United Kingdom;Italy;Japan;Sweden | |||
| 6 | EUCTR2020-002100-39-PL (EUCTR) | 02/02/2022 | A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy | Efficacy and Safety Study of Vatiquinone for the Treatment of Mitochondrial Disease Subjects With Refractory Epilepsy (MIT-E) - MIT-E | Genetically determined mitochondrial disease and associated refractory epilepsy MedDRA version: 20.0;Level: LLT;Classification code 10077953;Term: Refractory epilepsy;System Organ Class: 100000004852 MedDRA version: 20.0;Level: HLT;Classification code 10052637;Term: Genetic mitochondrial abnormalities NEC;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Vatiquinone Product Code: PTC743 INN or Proposed INN: VATIQUINONE Other descriptive name: EPI743; alpha-tocotrienol quinone | PTC THERAPEUTICS, INC. | NULL | NA | Female: yes Male: yes | 80 | Phase 2;Phase 3 | France;United States;Spain;Poland;Germany;United Kingdom;Italy;Sweden |