21. Mitochondrial disease Clinical trials / Disease details
Clinical trials : 39 / Drugs : 42 - (DrugBank : 32) / Drug target genes : 47 - Drug target pathways : 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 | JPRN-jRCT2011210075 | 22/03/2022 | 17/03/2022 | 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) | Mitochondrial Disease with Refractory Epilepsy | Vatiquinone: 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 Penematsa | NULL | Recruiting | Not applicable | < 18age old | Both | 4 | Phase 2-3 | USA;UK;Italy;Spain;Japan |
| 3 | 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 | ||
| 4 | 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 | ||
| 5 | NCT04378075 (ClinicalTrials.gov) | September 28, 2020 | 4/5/2020 | 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 | Mitochondrial Diseases;Drug Resistant Epilepsy;Leigh Disease;Leigh Syndrome;Mitochondrial Encephalopathy (MELAS);Pontocerebellar Hypoplasia Type 6 (PCH6);Alpers Disease;Alpers Syndrome | Drug: Vatiquinone;Other: Placebo | PTC Therapeutics | NULL | Active, not recruiting | N/A | 20 Years | All | 60 | Phase 2/Phase 3 | United States;Canada;France;Italy;Japan;Poland;Spain;Sweden;United Kingdom |
| 6 | 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 | |||
| 7 | 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 | |||
| 8 | 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 |