VATIQUINONE ( DrugBank: Vatiquinone )
2 diseases
| ID | Disease name (Link within this page) | Number of trials |
|---|---|---|
| 18 | Spinocerebellar degeneration | 7 |
| 21 | Mitochondrial disease | 8 |
18. Spinocerebellar degeneration
Clinical trials : 76 / Drugs : 98 - (DrugBank : 31) / Drug target genes : 44 - Drug target pathways : 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 | NCT05515536 (ClinicalTrials.gov) | December 8, 2022 | 23/8/2022 | A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia | Long-Term Open-Label Study to Assess the Safety and Efficacy of Vatiquinone in Patients With Friedreich Ataxia | Friedreich Ataxia | Drug: Vatiquinone | PTC Therapeutics | NULL | Enrolling by invitation | N/A | N/A | All | 140 | Phase 3 | United States;Australia;Brazil;Canada;France;Germany;Italy;New Zealand;Spain |
| 2 | NCT05485987 (ClinicalTrials.gov) | August 15, 2022 | 1/8/2022 | A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia | An Open-Label Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Vatiquinone in Children With Friedreich Ataxia Younger Than 7 Years of Age | Friedreich Ataxia | Drug: Vatiquinone | PTC Therapeutics | NULL | Recruiting | N/A | 6 Years | All | 5 | Phase 2 | United States |
| 3 | 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 | ||
| 4 | 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 | ||
| 5 | 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 | ||
| 6 | 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 | ||
| 7 | NCT04577352 (ClinicalTrials.gov) | December 17, 2020 | 30/9/2020 | A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With 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) | Friedreich Ataxia | Drug: Vatiquinone;Drug: Placebo | PTC Therapeutics | NULL | Active, not recruiting | 7 Years | N/A | All | 146 | Phase 2/Phase 3 | United States;Australia;Brazil;Canada;France;Germany;Italy;New Zealand;Spain |
21. Mitochondrial disease
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 |