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
Showing 1 to 10 of 39 diseases
| 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 | NCT05650229 (ClinicalTrials.gov) | December 13, 2022 | 6/12/2022 | Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease | An Interventional, Randomised, Double-blind, Parallel-group, Placebo-controlled, Flexible-dose, Adaptive Study of the Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease An Interventional, Randomised, Double-blind, Parallel-group, Placebo-controlled, Flexible-dose, Adap ... | Primary Mitochondrial Disease | Drug: KL1333;Drug: Placebo | Abliva AB | NULL | Recruiting | 18 Years | N/A | All | 180 | Phase 2 | Denmark;United Kingdom |
| 2 | NCT05241262 (ClinicalTrials.gov) | December 1, 2022 | 3/2/2022 | Study of N-acetylcysteine in the Treatment of Patients With the m.3243A>G Mutation and Low Brain Glutathione Levels Study of N-acetylcysteine in the Treatment of Patients With the m.3243A>G Mutation and Low Brain Glu ... | A Multiple Ascending Phase 1 Dose Study of N-acetylcysteine in the Treatment of Patients With the m.3243A>G Mutation and Low Brain Glutathione Levels A Multiple Ascending Phase 1 Dose Study of N-acetylcysteine in the Treatment of Patients With the m. ... | Mitochondrial Disease | Drug: N-Acetylcysteine | Darryl C. De Vivo, MD | United States Department of Defense | Recruiting | 18 Years | 80 Years | All | 18 | Phase 1 | United States |
| 3 | NCT05218655 (ClinicalTrials.gov) | June 21, 2022 | 19/1/2022 | A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial ... | An Open-Label, Safety Study for Previously Treated Vatiquinone (PTC743) Subjects With Inherited Mitochondrial Disease An Open-Label, Safety Study for Previously Treated Vatiquinone (PTC743) Subjects With Inherited Mito ... | Inherited Mitochondrial Disease | Drug: Vatiquinone | PTC Therapeutics | NULL | Enrolling by invitation | N/A | N/A | All | 200 | Phase 3 | United States |
| 4 | NCT05162768 (ClinicalTrials.gov) | April 29, 2022 | 8/12/2021 | Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD) Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease ... | A Phase 3 Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects With Primary Mitochondrial Disease Resulting From Pathogenic Nuclear DNA Mutations (nPMD) NuPower A Phase 3 Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Trial to Evaluate the Efficac ... | Mitochondrial Myopathies;Mitochondrial Pathology;Mitochondrial DNA Mutation;Mitochondrial Diseases;Mitochondrial DNA Deletion;Mitochondrial DNA Depletion;Mitochondrial Metabolism Defect;Mitochondrial Complex I Deficiency Mitochondrial Myopathies;Mitochondrial Pathology;Mitochondrial DNA Mutation;Mitochondrial Diseases;M ... | Drug: Elamipretide;Drug: Placebo | Stealth BioTherapeutics Inc. | NULL | Recruiting | 18 Years | 70 Years | All | 130 | Phase 3 | United States;Australia;Germany;Hungary;Italy;Netherlands;New Zealand;Norway;Spain;United Kingdom |
| 5 | EUCTR2021-003907-16-NO (EUCTR) | 11/04/2022 | 11/01/2022 | Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects with Primary Mitochondrial Disease Resulting from Pathogenic Nuclear DNA Mutations (nPMD) Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjec ... | A Phase 3 Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects with Primary Mitochondrial Disease Resulting from Pathogenic Nuclear DNA Mutations (nPMD) A Phase 3 Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Trial to Evaluate the Efficac ... | Primary Mitochondrial Disease Resulting from Pathogenic Nuclear DNA Mutations (nPMD) MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: HLGT;Classification code 10052635;Term: Cytoplasmic disorders congenital;Level: HLT;Classification code 10052637;Term: Genetic mitochondrial abnormalities NEC;Level: PT;Classification code 10027710;Term: Mitochondrial myopathy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] Primary Mitochondrial DiseaseResulting from Pathogenic Nuclear DNA Mutations (nPMD) MedDRA version: ... | Product Name: Elamipretide Product Code: MTP-131 INN or Proposed INN: ELAMIPRETIDE Other descriptive name: N/A Product Name: Elamipretide Product Code: MTP-131 INN or Proposed INN: ELAMIPRETIDE Other descriptive na ... | Stealth BioTherapeutics Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 130 | Phase 3 | United States;Hungary;Finland;Spain;Denmark;Australia;Netherlands;Germany;Norway;United Kingdom;Italy United States;Hungary;Finland;Spain;Denmark;Australia;Netherlands;Germany;Norway;United Kingdom;Ital ... | ||
| 6 | 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 A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Diseasein Particip ... | Efficacy and Safety Study of Vatiquinone for the Treatment of Mitochondrial Disease Subjects With Refractory Epilepsy (MIT-E) Efficacy and Safety Study of Vatiquinone for the Treatment of Mitochondrial DiseaseSubjects With Ref ... | 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. Vatiquinone: 15 milligrams/kilogram (mg/kg) if body weight <13 kg, and 200 mg if body weight >=13 kg, ... | Vinay Penematsa | NULL | Recruiting | Not applicable | < 18age old | Both | 4 | Phase 2-3 | USA;UK;Italy;Spain;Japan |
| 7 | EUCTR2021-003907-16-ES (EUCTR) | 18/03/2022 | 25/01/2022 | Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects with Primary Mitochondrial Disease Resulting from Pathogenic Nuclear DNA Mutations (nPMD) Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjec ... | A Phase 3 Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects with Primary Mitochondrial Disease Resulting from Pathogenic Nuclear DNA Mutations (nPMD) A Phase 3 Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Trial to Evaluate the Efficac ... | Primary Mitochondrial Disease Resulting from Pathogenic Nuclear DNA Mutations (nPMD) MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: HLGT;Classification code 10052635;Term: Cytoplasmic disorders congenital;Level: HLT;Classification code 10052637;Term: Genetic mitochondrial abnormalities NEC;Level: PT;Classification code 10027710;Term: Mitochondrial myopathy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] Primary Mitochondrial DiseaseResulting from Pathogenic Nuclear DNA Mutations (nPMD) MedDRA version: ... | Product Name: Elamipretide Product Code: MTP-131 INN or Proposed INN: ELAMIPRETIDE Other descriptive name: N/A Product Name: Elamipretide Product Code: MTP-131 INN or Proposed INN: ELAMIPRETIDE Other descriptive na ... | Stealth BioTherapeutics Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 130 | Phase 3 | United States;Hungary;Finland;Spain;Denmark;Australia;Norway;Netherlands;Germany;United Kingdom;Italy United States;Hungary;Finland;Spain;Denmark;Australia;Norway;Netherlands;Germany;United Kingdom;Ital ... | ||
| 8 | EUCTR2021-003907-16-HU (EUCTR) | 02/03/2022 | 04/01/2022 | Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects with Primary Mitochondrial Disease Resulting from Pathogenic Nuclear DNA Mutations (nPMD) Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjec ... | A Phase 3 Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects with Primary Mitochondrial Disease Resulting from Pathogenic Nuclear DNA Mutations (nPMD) A Phase 3 Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Trial to Evaluate the Efficac ... | Primary Mitochondrial Disease Resulting from Pathogenic Nuclear DNA Mutations (nPMD) MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: HLGT;Classification code 10052635;Term: Cytoplasmic disorders congenital;Level: HLT;Classification code 10052637;Term: Genetic mitochondrial abnormalities NEC;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] Primary Mitochondrial DiseaseResulting from Pathogenic Nuclear DNA Mutations (nPMD) MedDRA version: ... | Product Name: Elamipretide Product Code: MTP-131 INN or Proposed INN: ELAMIPRETIDE Other descriptive name: N/A Product Name: Elamipretide Product Code: MTP-131 INN or Proposed INN: ELAMIPRETIDE Other descriptive na ... | Stealth BioTherapeutics Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 130 | Phase 3 | United States;Hungary;Finland;Spain;Denmark;Australia;Norway;Netherlands;Germany;United Kingdom;Italy United States;Hungary;Finland;Spain;Denmark;Australia;Norway;Netherlands;Germany;United Kingdom;Ital ... | ||
| 9 | 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 A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Diseasein Particip ... | Efficacy and Safety Study of Vatiquinone for the Treatment of Mitochondrial Disease Subjects With Refractory Epilepsy (MIT-E) - MIT-E Efficacy and Safety Study of Vatiquinone for the Treatment of Mitochondrial DiseaseSubjects With Ref ... | 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] Genetically determined mitochondrial diseaseand associated refractory epilepsy MedDRA version: 20.0; ... | Product Name: Vatiquinone Product Code: PTC743 INN or Proposed INN: VATIQUINONE Other descriptive name: EPI743; alpha-tocotrienol quinone Product Name: Vatiquinone Product Code: PTC743 INN or Proposed INN: VATIQUINONE Other descriptive name: ... | 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 | ||
| 10 | EUCTR2021-001268-58-HU (EUCTR) | 06/07/2021 | 21/03/2021 | Investigation of effects of cariprazine in patients with mitochondrial schizophrenia | Investigation of cellbiological effects of cariprazine in patients with mitochondrial schizophrenia Investigation of cellbiological effects of cariprazine in patients with mitochondrial schizophrenia ... | Mitochondrial disease associated schizophrenia MedDRA version: 20.0;Level: PT;Classification code 10039626;Term: Schizophrenia;System Organ Class: 10037175 - Psychiatric disorders;Therapeutic area: Psychiatry and Psychology [F] - Behavioral Disciplines and Activities [F04] Mitochondrial diseaseassociated schizophrenia MedDRA version: 20.0;Level: PT;Classification code 100 ... | Trade Name: Reagila Product Name: Reagila | Semmelweis University, STIA-POC-2020 | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 10 | Phase 4 | Hungary |