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 21. ミトコンドリア病 [臨床試験数:20,薬物数:16(DrugBank:6),標的遺伝子数:19,標的パスウェイ数:8] 

Searched query = "Mitochondrial disease"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/21/2019, 11/20/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"    Synonyms (DrugBank) were also searched for.
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
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Target_
size
PhaseCountries
1NCT03888716March 18, 20198 April 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 TabletNeuroVive Pharmaceutical ABRecruiting18 Years75 YearsAll72Phase 1United Kingdom
2JPRN-JMA-IIA0040601/02/20192 April 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 HospitalRecruiting>=9 MONTHSNo LimitBOTH55Phase 3Japan
3JPRN-JMA-IIA0035830/06/201822 July 2019Multicenter 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 HospitalNot Recruiting>=3 MONTHSNo LimitBOTH40Phase 3Japan
4NCT03388528September 8, 201722 October 2019Low 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 TrustNot recruiting18 YearsN/AAll36N/AUnited Kingdom
5NCT02976038December 201623 April 2019Open-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.Not recruiting16 YearsN/AAll36Phase 2United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2016-001696-79-NL06/09/201612 September 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 studyInherited 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
Pharmaceutical Form: Oral liquid
Pharmaceutical form of the placebo: Oral liquid
Route of administration of the placebo: Oral use
Khondrion BVAuthorisedFemale: yes
Male: yes
Phase 2Netherlands
7NCT02909400September 20165 March 2018The 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: placebo;Drug: KH176Khondrion BVRadboud Center for Mitochondrial Medicine (RCMM);Radboud UniversityNot recruiting18 YearsN/AAll20Phase 2Netherlands
8NCT02805790August 22, 201611 June 2018A Study to Evaluate the Safety, Tolerability, and Efficacy of Subcutaneous Injections of Elamipretide (MTP-131) in Subjects With Genetically Confirmed Mitochondrial Disease Previously Treated in the Stealth BioTherapeutics SPIMM-201 StudyA Phase 2 Randomized, Double-Blind, Placebo-Controlled Crossover Study to Evaluate the Safety, Tolerability, and Efficacy of Subcutaneous Injections of Elamipretide (MTP-131) in Subjects With Genetically Confirmed Mitochondrial Disease Previously Treated in the Stealth BioTherapeutics SPIMM-201 StudyPrimary Mitochondrial DiseaseDrug: Elamipretide;Drug: PlaceboStealth BioTherapeutics Inc.Not recruiting16 YearsN/AAll30Phase 2United States
9JPRN-JMA-IIA0021402/06/201523 April 2019Long-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 HospitalNot Recruiting>=9 MONTHS<30 MONTHSBOTH10Phase 2Japan
10NCT02473445May 19, 201511 June 2018A 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.Not recruiting6 Years17 YearsAll22Phase 2United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11NCT02544217May 201515 February 2016A 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, BelgiumNot recruiting18 Years55 YearsMale30Phase 1Belgium
12NCT02367014February 201513 June 2016A Study Investigating the Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial MyopathyA Phase 1/2 Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending-Dose Clinical Study Investigating the Safety, Tolerability, and Efficacy of Intravenous MTP-131 for the Treatment of Mitochondrial Myopathy in Subjects With Genetically Confirmed Mitochondrial DiseaseMitochondrial MyopathyDrug: MTP-131;Drug: PlaceboStealth BioTherapeutics Inc.Not recruiting16 Years65 YearsBoth36Phase 1/Phase 2United States
13JPRN-JMA-IIA0020010/12/20142 April 2019Multicenter trial of SPP-004 in mitochondrial diseasesMulticenter trial of SPP-004 in mitochondrial diseasesMitochondrial 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 HospitalNot Recruiting>=3 YEARS<2 YEARSBOTH10Phase 2Japan
14NCT02023866May 201416 December 2017Open-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.Not recruiting6 Years17 YearsAll36Phase 2United States
15NCT02053766January 201410 December 2018Anesthesia in Patients With Mitochondrial DiseaseAnesthesia in Patients With Mitochondrial DiseaseMitochondrial DiseasesDrug: Sevoflurane;Drug: Dexmedetomidine;Drug: PropofolThe University of Texas Health Science Center, HoustonRecruitingN/A17 YearsAll60N/AUnited States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16NCT01642056September 1, 20127 October 2019EPI-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)Not recruiting2 Years11 YearsAll20Phase 1/Phase 2United States
17NCT01831934September 201016 December 2017Responses 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)Not recruiting13 Years65 YearsAll22Phase 4United States
18NCT00432744January 200716 December 2017Phase 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 AdministrationNot recruiting12 Months17 YearsAll24Phase 3United States;Canada
19NCT01001585September 200616 March 2015Anesthetic Effects in Mitochondrial DiseaseAnesthetic Effects in Mitochondrial DiseaseMitochondrial DiseaseDrug: sevofluraned sesslerNot recruiting12 Months16 YearsBoth55N/AUnited States
20NCT00060515April 200319 February 2015RG2133 (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 CorporationNot recruiting3 YearsN/ABoth12Phase 1United States

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