Alpha-tocotrienol quinone ( DrugBank: Tocotrienol )


2 diseases
告示番号疾患名(ページ内リンク)臨床試験数
21ミトコンドリア病4
156レット症候群1

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.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-PL
(EUCTR)
02/02/2022A 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 3France;United States;Spain;Poland;Germany;United Kingdom;Italy;Sweden
3EUCTR2020-002100-39-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
4EUCTR2020-002100-39-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
88Phase 2;Phase 3France;United States;Canada;Spain;Poland;Australia;Russian Federation;Germany;United Kingdom;Italy;Japan;Sweden

156. レット症候群


臨床試験数 : 44 薬物数 : 61 - (DrugBank : 23) / 標的遺伝子数 : 57 - 標的パスウェイ数 : 83
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2012-005021-76-IT
(EUCTR)
31/01/201311/12/2012Phase 2A clinical trial conducted on children affected by Rett syndrome and who are casually administered with the experimental treatment, EPI-743, compared versus the se of placebo.A Phase 2A Randomized, Placebo Controlled Trial of EPI-743 in Children with Rett Syndrome Rett Syndrome
MedDRA version: 14.1;Level: HLGT;Classification code 10021605;Term: Inborn errors of metabolism;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: EPI-743
Product Code: NA
Other descriptive name: Alpha-tocotrienol quinone
EDISON PHARMACEUTICALS INC.NULLNot RecruitingFemale: yes
Male: no
Phase 2Italy