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

  
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PhaseCountries
1NCT05650229
(ClinicalTrials.gov)
December 13, 20226/12/2022Efficacy of KL1333 in Adult Patients With Primary Mitochondrial DiseaseAn Interventional, Randomised, Double-blind, Parallel-group, Placebo-controlled, Flexible-dose, Adaptive Study of the Efficacy of KL1333 in Adult Patients With Primary Mitochondrial DiseasePrimary Mitochondrial DiseaseDrug: KL1333;Drug: PlaceboAbliva ABNULLRecruiting18 YearsN/AAll180Phase 2Denmark;United Kingdom
2NCT05241262
(ClinicalTrials.gov)
December 1, 20223/2/2022Study of N-acetylcysteine in the Treatment of Patients With the m.3243A>G Mutation and Low Brain Glutathione LevelsA Multiple Ascending Phase 1 Dose Study of N-acetylcysteine in the Treatment of Patients With the m.3243A>G Mutation and Low Brain Glutathione LevelsMitochondrial DiseaseDrug: N-AcetylcysteineDarryl C. De Vivo, MDUnited States Department of DefenseRecruiting18 Years80 YearsAll18Phase 1United States
3NCT05218655
(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
4NCT05162768
(ClinicalTrials.gov)
April 29, 20228/12/2021Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD)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) NuPowerMitochondrial Myopathies;Mitochondrial Pathology;Mitochondrial DNA Mutation;Mitochondrial Diseases;Mitochondrial DNA Deletion;Mitochondrial DNA Depletion;Mitochondrial Metabolism Defect;Mitochondrial Complex I DeficiencyDrug: Elamipretide;Drug: PlaceboStealth BioTherapeutics Inc.NULLRecruiting18 Years70 YearsAll130Phase 3United States;Australia;Germany;Hungary;Italy;Netherlands;New Zealand;Norway;Spain;United Kingdom
5EUCTR2021-003907-16-NO
(EUCTR)
11/04/202211/01/2022Trial 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 Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects with Primary Mitochondrial Disease Resulting from Pathogenic Nuclear DNA Mutations (nPMD) 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]
Product Name: Elamipretide
Product Code: MTP-131
INN or Proposed INN: ELAMIPRETIDE
Other descriptive name: N/A
Stealth BioTherapeutics Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
130Phase 3United States;Hungary;Finland;Spain;Denmark;Australia;Netherlands;Germany;Norway;United Kingdom;Italy
6JPRN-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
7EUCTR2021-003907-16-ES
(EUCTR)
18/03/202225/01/2022Trial 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 Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects with Primary Mitochondrial Disease Resulting from Pathogenic Nuclear DNA Mutations (nPMD) 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]
Product Name: Elamipretide
Product Code: MTP-131
INN or Proposed INN: ELAMIPRETIDE
Other descriptive name: N/A
Stealth BioTherapeutics Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
130Phase 3United States;Hungary;Finland;Spain;Denmark;Australia;Norway;Netherlands;Germany;United Kingdom;Italy
8EUCTR2021-003907-16-HU
(EUCTR)
02/03/202204/01/2022Trial 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 Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects with Primary Mitochondrial Disease Resulting from Pathogenic Nuclear DNA Mutations (nPMD) 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]
Product Name: Elamipretide
Product Code: MTP-131
INN or Proposed INN: ELAMIPRETIDE
Other descriptive name: N/A
Stealth BioTherapeutics Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
130Phase 3United States;Hungary;Finland;Spain;Denmark;Australia;Norway;Netherlands;Germany;United Kingdom;Italy
9EUCTR2020-002100-39-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
10EUCTR2021-001268-58-HU
(EUCTR)
06/07/202121/03/2021Investigation of effects of cariprazine in patients with mitochondrial schizophreniaInvestigation 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]
Trade Name: Reagila
Product Name: Reagila
Semmelweis University, STIA-POC-2020NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
10Phase 4Hungary
11EUCTR2020-002100-39-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
12NCT04846036
(ClinicalTrials.gov)
February 1, 20215/2/2021The KHENERGYC StudyA Randomized Placebo Controlled, Double-blind Phase II Study to Explore the Safety, Efficacy and Pharmacokinetics of Sonlicromanol in Children With Genetically Confirmed Mitochondrial DiseaseMitochondrial Diseases;Mitochondrial DNA tRNALeu(UUR) m.3243ADrug: Sonlicromanol;Drug: PlaceboKhondrion BVJulius Clinical;ProPharma Group;Europees Fonds voor Regionale Ontwikkeling (EFRO);Ardena;CertaraRecruiting0 Years17 YearsAll24Phase 2Netherlands
13EUCTR2020-003124-16-NL
(EUCTR)
12/01/202120/10/2020The KHENERGYC study: a placebo controlled, double-blind study to explore the safety, efficacy and pharmacokinetics of sonlicromanol in children with a mitochondrial disease.A randomized placebo controlled, double-blind phase II study to explore the safety, efficacy and pharmacokinetics of sonlicromanol in children with genetically confirmed mitochondrial disease. Genetically confirmed mitochondrial disease;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]Product Name: Sonlicromanol
Product Code: KH176
INN or Proposed INN: sonlicromanol
Other descriptive name: KH176
Khondrion B.V.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
24Phase 2Netherlands
14NCT04643249
(ClinicalTrials.gov)
November 10, 202013/11/2020Drug-drug Interaction Study of KL1333 in Healthy SubjectsA Phase I, Open-label, Fixed-sequence, Crossover, Drug-drug Interaction Study to Investigate the Inhibition Potential of KL1333 on CYP1A2, CYP2B6, CYP2C8, CYP2C9, CYP2C19, CYP2D6, and CYP3A4 in Healthy SubjectsMitochondrial DiseaseDrug: KL1333;Drug: Flurbiprofen;Drug: Dextromethorphan;Drug: Bupropion;Drug: Midazolam injection;Drug: Omeprazole;Drug: Caffeine;Drug: RepaglinideAbliva ABNULLCompleted18 Years65 YearsAll14Phase 1United Kingdom
15NCT04378075
(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
16EUCTR2018-001094-25-IT
(EUCTR)
12/09/201920/05/2021Clinical trial with Phenilbutirrate to reduce lactic acid in patients affected with Melas Syndrome and PHD deficency encephalopathyPhenylbutyrate Therapy in Mitochondrial Disease with lactic acidosis: an opel label clinical trial in MELAS and PHD deficiency patients - PHEMI Melas Syndrome and PDH deficency enchephalopathy
MedDRA version: 20.0;Level: PT;Classification code 10053872;Term: MELAS syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Level: LLT;Classification code 10062950;Term: Leigh syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: Feburane
Product Name: pheburane
Product Code: [A042917017]
FONDAZIONE IRCCS ISTITUTO NEUROLOGICO CARLO BESTANULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
9Phase 2Italy
17NCT03888716
(ClinicalTrials.gov)
March 18, 201919/3/2019A Phase Ia/Ib, SAD and MAD Study of of KL1333 in Healthy Subjects and Patients With Primary Mitochondrial DiseaseA Phase Ia/Ib, Multiple-site Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of KL1333 After a Single and Multiple Ascending Oral Doses in Healthy Subjects and Patients With Primary Mitochondrial DiseaseMitochondrial Diseases;Mitochondrial Respiratory Chain Deficiencies;MELAS Syndrome;Mitochondrial MyopathiesDrug: KL1333;Drug: Placebo Oral TabletAbliva ABNULLCompleted18 Years75 YearsAll72Phase 1United Kingdom
18JPRN-JMA-IIA00406
01/02/201918/01/2019Long-term, multicenter trial of SPP-004 in mitochondrial diseases(after confirmation trial)Long-term, multicenter trial of SPP-004 in mitochondrial diseases(after confirmation trial) Mitochondrial disease, involved mainly central nervous systemIntervention type:DRUG. Intervention1:SPP-004, Dose form:CAPSULE, Route of administration:ORAL, intended dose regimen:5-ALA HCl and SFC capsules are administered orally or by a feeding tube in accordance with doses and frequency of administration based on body weight at screening and each observation time during the period of treatment.If capsules cannot be swollen, the content should be dissolved at the time of administration.. Control intervention1:-, Intended dose regimen:-.Saitama Medical University HospitalNULLCompleted>=9 MONTHSNo LimitBOTH55Phase 3Japan
19JPRN-JMA-IIA00358
30/06/201822/06/2018Multicenter trial of SPP-004 in mitochondrial diseases (confirmatory trial)Multicenter trial of SPP-004 in mitochondrial diseases (confirmatory trial) Mitochondrial disease, involved mainly central nervous systemIntervention type:DRUG. Intervention1:SPP-004, Dose form:CAPSULE, Route of administration:ORAL, intended dose regimen:Open period
A capsule of each investigational drug (5-ALA HCl capsule (25 mg) and SFC capsule (39.22 mg)) is administered orally or by a feeding tube. If capsules cannot be swollen, the content should be dissolved at the time of administration.
Double-blind period.
Same dose in open period is administered orally or by a feeding tube. If capsules cannot be swollen, the content should be dissolved at the time of administration.
Through the study treatment (open period and double-blind period), doses are based on the body weight at observation time during the treatment period.. Control intervention1:Placebo, Dose form:CAPSULE, Route of administration:ORAL, Intended dose regimen:Open period
A capsule of each investigational drug (5-ALA HCl capsule (25 mg) and SFC capsule (39.22 mg)) is administered orally or by a feeding tube. If capsules cannot be swollen, the content should be dissolved at the time of administration.
Double-blind period.
Same dose in open period is administered orally or by a feeding tube. If capsules cannot be swollen, the content should be dissolved at the time of administration.
Through the study treatment (open period and double-blind period), doses are based on the body weight at observation time during the treatment period..
Saitama Medical University HospitalNULLCompleted>=3 MONTHSNo LimitBOTH40Phase 3Japan
20NCT03388528
(ClinicalTrials.gov)
September 8, 201727/10/2017Low Residue Diet Study in Mitochondrial DiseasePhase II Feasibility Study of the Efficacy and Acceptability of a Low Residue Diet in Adult Patients With Mitochondrial DiseaseMitochondrial DiseasesDietary Supplement: Low Residue Diet InterventionNewcastle UniversityNewcastle-upon-Tyne Hospitals NHS TrustCompleted18 YearsN/AAll36Phase 2United Kingdom
21NCT02976038
(ClinicalTrials.gov)
December 201618/11/2016Open-Label Extension Trial to Characterize the Long-term Safety and Tolerability of Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Myopathy (PMM)A Multicenter, Open-Label Phase 2 Extension Trial to Characterize the Long-term Safety and Tolerability of Subcutaneous Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Myopathy (PMM)Primary Mitochondrial DiseaseDrug: elamipretideStealth BioTherapeutics Inc.NULLTerminated16 YearsN/AAll36Phase 2United States
22EUCTR2016-001696-79-NL
(EUCTR)
06/09/201618/08/2016A Phase II study with KH176 in patients with mitochondrial diseaseA double-blind, randomized, placebo-controlled, single-center, two-way cross-over study with KH176 in patients with the mitochondrial DNA tRNALeu(UUR) m.3243A>G mutation and clinical signs of mitochondrial disease - The KHENERGY study Inherited mitochondrial disease, including MELAS (mitochondrial Encephalopathy Lactic Acidosis and Stroke like episodes) and MIDD (Maternally Inherited Diabetes and Deafness);Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]Product Name: KH176
Product Code: KH176
Khondrion BVNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Phase 2Netherlands
23NCT02909400
(ClinicalTrials.gov)
September 201613/9/2016The KHENERGY StudyAn Exploratory, Double-blind, Randomized, Placebo-controlled, Single-center, Two-way Cross-over Study With KH176 in Patients With the Mitochondrial DNA tRNALeu(UUR) m.3243A>G Mutation and Clinical Signs of Mitochondrial DiseaseMitochondrial Diseases;Mitochondrial Myopathies;Mitochondrial Encephalomyopathies;MELAS;MIDDDrug: KH176;Drug: placeboKhondrion BVRadboud UniversityCompleted18 YearsN/AAll20Phase 2Netherlands
24NCT02805790
(ClinicalTrials.gov)
August 22, 201613/6/2016Safety, Tolerability, Efficacy of MTP-131 for Treatment of Mitochondrial Disease in Subjects From the MMPOWER StudyA Phase 2 Randomized, Double-Blind, Placebo-Controlled Crossover Study to Evaluate Safety, Tolerability, and Efficacy of Subcutaneous Injections of MTP-131 in Subjects With Mitochondrial Myopathy Previously Treated in the SPIMM-201 StudyPrimary Mitochondrial DiseaseDrug: Elamipretide;Drug: PlaceboStealth BioTherapeutics Inc.NULLCompleted16 YearsN/AAll30Phase 2United States
25JPRN-JMA-IIA00214
02/06/201526/05/2015Long-term, multicenter trial of SPP-004 in mitochondrial diseases.Long-term, multicenter trial of SPP-004 in mitochondrial diseases. Mitochondrial disease, mainly to cranial nerve symptomsIntervention type:DRUG. Intervention1:SPP-004, Dose form:CAPSULE, Route of administration:ORAL, intended dose regimen:5-ALA HCl and SFC capsules are administered orally or by a feeding tube in accordance with doses and frequency of administration based on body weight at each observation time during the period of treatment. If capsules cannot be swollen, the content should be dissolved at the time of administration..Saitama Medical University HospitalNULLCompleted>=9 MONTHS<30 MONTHSBOTH10Phase 2Japan
26NCT02473445
(ClinicalTrials.gov)
May 19, 201510/6/2015A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial DiseaseA Long-Term Open-Label Extension Study of RP103-MITO-001 to Assess the Safety, Tolerability and Efficacy of Cysteamine Bitartrate Delayed-release Capsules (RP103) for Treatment of Children With Inherited Mitochondrial DiseaseMitochondrial DiseasesDrug: Cysteamine BitartrateHorizon Pharma USA, Inc.NULLTerminated6 Years17 YearsAll22Phase 2United States
27NCT02544217
(ClinicalTrials.gov)
May 201519/4/2015A Dose-escalating Clinical Trial With KH176A Phase I, Randomized, Double Blind, Placebo-controlled, Dose-escalating Clinical Trial With KH176MELAS;LHON;Leigh Syndrome;Mitochondrial Disease;Mitochondrial DNA tRNALeu(UUR) m.3243ADrug: KH176;Drug: placeboKhondrion BVDrug Research Unit Ghent, BelgiumCompleted18 Years55 YearsMale32Phase 1Belgium
28NCT02367014
(ClinicalTrials.gov)
February 20159/2/2015Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial MyopathyPhase 1/2 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending-Dose Clinical Study for the Safety, Tolerability, and Efficacy of IV MTP-131 for Mitochondrial Myopathy in Genetically Confirmed Mitochondrial DiseaseMitochondrial MyopathyDrug: elamipretide (low dose);Drug: elamipretide (intermediate dose);Drug: elamipretide (high dose);Drug: PlaceboStealth BioTherapeutics Inc.NULLCompleted16 Years65 YearsAll36Phase 1/Phase 2United States
29JPRN-JMA-IIA00200
10/12/201409/12/2014Multicenter trial of SPP-004 in mitochondrial diseasesMulticenter trial of SPP-004 in mitochondrial diseases Mitochondrial disease, mainly to cranial nerve symptomsIntervention type:DRUG. Intervention1:SPP-004, Dose form:CAPSULE, Route of administration:ORAL, intended dose regimen:A capsule of each investigational drug [5-ALA HCl capsule (25 mg) and SFC capsule (39.22 mg)] is administered twice a day orally or by a feeding tube . After Week 13, a capsule of each investigational drug (5-ALA HCl capsule and SFC capsule) is administered twice a day orally and by a feeding tube. If capsules cannot be swollen, the content should be dissolved at the time of administration.. Control intervention1:Placebo, Dose form:CAPSULE, Route of administration:ORAL, Intended dose regimen:A capsule of each investigational drug [5-ALA HCl placebo capsule (25 mg) and SFC capsule (39.22 mg)] is administered twice a day orally or by a feeding tube.
If capsules cannot be swollen, the content should be dissolved at the time of administration.
.
Saitama Medical University HospitalNULLCompleted>=3 YEARS<2 YEARSBOTH10Phase 2Japan
30NCT02023866
(ClinicalTrials.gov)
May 201417/12/2013Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial DiseaseAn Open-Label, Dose-Escalating Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamics of Cysteamine Bitartrate Delayed-release Capsules (RP103) for Treatment of Children With Inherited Mitochondrial DiseaseInherited Mitochondrial Disease, Including Leigh SyndromeDrug: Cysteamine BitartrateHorizon Pharma USA, Inc.NULLCompleted6 Years17 YearsAll36Phase 2United States
31NCT02053766
(ClinicalTrials.gov)
January 10, 201431/1/2014Anesthesia in Patients With Mitochondrial DiseaseAnesthesia in Patients With Mitochondrial DiseaseMitochondrial DiseasesDrug: Sevoflurane;Drug: Dexmedetomidine;Drug: PropofolThe University of Texas Health Science Center, HoustonNULLRecruitingN/A17 YearsAll60N/AUnited States
32NCT01642056
(ClinicalTrials.gov)
September 1, 201214/7/2012EPI-743 for Metabolism or Mitochondrial DisordersTherapeutic Trial of EPI -743 In Patients With Disorders of Energy Utilization or Oxidation-ReductionMitochondrial Disease;Neurology;MyoptahyDrug: EPI-743;Drug: PlaceboNational Human Genome Research Institute (NHGRI)NULLCompleted2 Years11 YearsAll20Phase 1/Phase 2United States
33NCT01831934
(ClinicalTrials.gov)
September 201019/3/2013Responses to Influenza Vaccine in Patients With Mitochondrial Disorders (MELAS)Metabolic and Immune Responses to TIV in Patients With Mitochondrial DiseaseMELAS SyndromeBiological: Fluzone®Stanford UniversityEunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)Completed13 Years65 YearsAll22Phase 4United States
34NCT00432744
(ClinicalTrials.gov)
January 20076/2/2007Phase III Trial of Coenzyme Q10 in Mitochondrial DiseasePhase 3 Trial of Coenzyme Q10 in Mitochondrial DiseaseMitochondrial DiseasesDrug: CoenzymeQ10;Drug: PlaceboUniversity of FloridaFDA Office of Orphan Products Development;Food and Drug AdministrationCompleted12 Months17 YearsAll24Phase 3United States;Canada
35NCT01001585
(ClinicalTrials.gov)
September 200621/10/2009Anesthetic Effects in Mitochondrial DiseaseAnesthetic Effects in Mitochondrial DiseaseMitochondrial DiseaseDrug: sevofluraned sesslerNULLTerminated12 Months16 YearsBoth55N/AUnited States
36NCT00060515
(ClinicalTrials.gov)
April 20037/5/2003RG2133 (2',3',5'-Tri-O-Acetyluridine) in Mitochondrial DiseaseAn Open-Label Dose-Escalation Phase I Study to Asses the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of RG2133 (2',3',5'-Tri-O-Acetyluridine) in the Treatment of Inherited Mitochondrial DiseasesMitochondrial DiseasesDrug: RG2133 (2',3',5'-tri-O-acetyluridine)Repligen CorporationNULLTerminated3 YearsN/ABoth12Phase 1United States
37EUCTR2020-002100-39-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
38EUCTR2020-002100-39-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
39EUCTR2020-002100-39-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