VATIQUINONE ( DrugBank: Vatiquinone )


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。)5
21ミトコンドリア病5

18. 脊髄小脳変性症(多系統萎縮症を除く。)


臨床試験数 : 71 薬物数 : 101 - (DrugBank : 30) / 標的遺伝子数 : 45 - 標的パスウェイ数 : 65
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2020-002812-36-ES
(EUCTR)
02/06/202119/11/2020A study to determine if Vatiquinone, the study drug, is safe and effective to treat a neurological condition called Friedreich ataxiaA 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
126Phase 2;Phase 3United States;France;Canada;Brazil;Spain;Australia;Germany;Italy
2EUCTR2020-002812-36-IT
(EUCTR)
14/04/202104/06/2021A study to determine if Vatiquinone, the study drug, is safe and effective to treat a neurological condition called Friedreich ataxiaA 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
126Phase 2;Phase 3France;United States;Canada;Spain;Brazil;Australia;Germany;Italy
3EUCTR2020-002812-36-FR
(EUCTR)
18/03/202115/01/2021A study to determine if Vatiquinone, the study drug, is safe and effective to treat a neurological condition called Friedreich ataxiaA 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
126Phase 2;Phase 3United States;France;Canada;Spain;Brazil;Australia;Germany;Italy;New Zealand
4EUCTR2020-002812-36-DE
(EUCTR)
18/02/202106/11/2020A study to determine if Vatiquinone, the study drug, is safe and effective to treat a neurological condition called Friedreich ataxiaA 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
126Phase 2;Phase 3France;United States;Canada;Spain;Brazil;Australia;Germany;Italy;New Zealand
5NCT04577352
(ClinicalTrials.gov)
December 17, 202030/9/2020A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich AtaxiaA 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)Friedreich AtaxiaDrug: Vatiquinone;Drug: PlaceboPTC TherapeuticsNULLActive, not recruiting7 YearsN/AAll146Phase 2/Phase 3United States;Australia;Brazil;Canada;France;Germany;Italy;New Zealand;Spain

21. ミトコンドリア病


臨床試験数 : 33 薬物数 : 43 - (DrugBank : 32) / 標的遺伝子数 : 47 - 標的パスウェイ数 : 67
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2020-002100-39-ES
(EUCTR)
26/07/202109/02/2021A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory EpilepsyEfficacy 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
80Phase 2;Phase 3France;United States;Spain;Germany;United Kingdom;Italy
2EUCTR2020-002100-39-IT
(EUCTR)
14/04/202104/06/2021A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory EpilepsyEfficacy 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
80Phase 2;Phase 3France;United States;Spain;Germany;United Kingdom;Italy;Sweden
3NCT04378075
(ClinicalTrials.gov)
September 28, 20204/5/2020A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory EpilepsyEfficacy and Safety Study of Vatiquinone for the Treatment of Mitochondrial Disease Subjects With Refractory EpilepsyMitochondrial Diseases;Drug Resistant Epilepsy;Leigh Disease;Leigh Syndrome;Mitochondrial Encephalopathy (MELAS);Pontocerebellar Hypoplasia Type 6 (PCH6);Alpers Disease;Alpers SyndromeDrug: Vatiquinone;Other: PlaceboPTC TherapeuticsNULLRecruitingN/A20 YearsAll60Phase 2/Phase 3United States;Italy;Spain;United Kingdom
4EUCTR2020-002100-39-FR
(EUCTR)
25/02/2021A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory EpilepsyEfficacy 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.NULLNAFemale: yes
Male: yes
80Phase 2;Phase 3United States;France;Spain;Germany;United Kingdom;Italy
5EUCTR2020-002100-39-SE
(EUCTR)
14/12/2021A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory EpilepsyEfficacy 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.NULLNAFemale: yes
Male: yes
80Phase 2;Phase 3France;United States;Canada;Spain;Poland;Australia;Russian Federation;Germany;United Kingdom;Japan;Italy;Sweden