21. ミトコンドリア病 Mitochondrial disease Clinical trials / Disease details


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

  
8 trials found
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PhaseCountries
1NCT05218655
(ClinicalTrials.gov)
June 21, 202219/1/2022A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial DiseaseAn Open-Label, Safety Study for Previously Treated Vatiquinone (PTC743) Subjects With Inherited Mitochondrial DiseaseInherited Mitochondrial DiseaseDrug: VatiquinonePTC TherapeuticsNULLEnrolling by invitationN/AN/AAll200Phase 3United States
2JPRN-jRCT2011210075
22/03/202217/03/2022A 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) Mitochondrial Disease with Refractory EpilepsyVatiquinone:
15 milligrams/kilogram (mg/kg) if body weight <13 kg, and 200 mg if body weight >=13 kg, administered orally, 3 times per day (TID) or up to 72 weeks
Placebo:
Vatiquinone-matching placebo, administered orally, TID for up to 24 weeks followed by vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight >=13 kg, administered orally, TID for up to 48 weeks.
Vinay PenematsaNULLRecruitingNot applicable< 18age oldBoth4Phase 2-3USA;UK;Italy;Spain;Japan
3EUCTR2020-002100-39-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
4EUCTR2020-002100-39-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
5NCT04378075
(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 TherapeuticsNULLActive, not recruitingN/A20 YearsAll60Phase 2/Phase 3United States;Canada;France;Italy;Japan;Poland;Spain;Sweden;United Kingdom
6EUCTR2020-002100-39-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
7EUCTR2020-002100-39-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
8EUCTR2020-002100-39-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