21. Mitochondrial disease
[
20 clinical trials,
16 drugs（DrugBank:
6 drugs),
19 target genes / 8 target pathways ]
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.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03888716 | March 18, 2019 | 8 April 2019 | A Phase Ia/Ib, SAD and MAD Study of of KL1333 in Healthy Subjects and Patients With Primary Mitochondrial Disease | A 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 Disease | Mitochondrial Diseases;Mitochondrial Respiratory Chain Deficiencies;MELAS Syndrome;Mitochondrial Myopathies | Drug: KL1333;Drug: Placebo Oral Tablet | NeuroVive Pharmaceutical AB | Recruiting | 18 Years | 75 Years | All | 72 | Phase 1 | United Kingdom | |
| 2 | JPRN-JMA-IIA00406 | 01/02/2019 | 2 April 2019 | Long-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 system | Intervention 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 Hospital | Recruiting | >=9 MONTHS | No Limit | BOTH | 55 | Phase 3 | Japan | |
| 3 | JPRN-JMA-IIA00358 | 30/06/2018 | 22 July 2019 | Multicenter 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 system | Intervention 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 Hospital | Not Recruiting | >=3 MONTHS | No Limit | BOTH | 40 | Phase 3 | Japan | |
| 4 | NCT03388528 | September 8, 2017 | 22 October 2019 | Low Residue Diet Study in Mitochondrial Disease | Phase II Feasibility Study of the Efficacy and Acceptability of a Low Residue Diet in Adult Patients With Mitochondrial Disease | Mitochondrial Diseases | Dietary Supplement: Low Residue Diet Intervention | Newcastle University | Newcastle-upon-Tyne Hospitals NHS Trust | Not recruiting | 18 Years | N/A | All | 36 | N/A | United Kingdom |
| 5 | NCT02976038 | December 2016 | 23 April 2019 | Open-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 Disease | Drug: elamipretide | Stealth BioTherapeutics Inc. | Not recruiting | 16 Years | N/A | All | 36 | Phase 2 | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | EUCTR2016-001696-79-NL | 06/09/2016 | 12 September 2016 | A Phase II study with KH176 in patients with mitochondrial disease | A 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 Pharmaceutical Form: Oral liquid Pharmaceutical form of the placebo: Oral liquid Route of administration of the placebo: Oral use | Khondrion BV | Authorised | Female: yes Male: yes | Phase 2 | Netherlands | ||||
| 7 | NCT02909400 | September 2016 | 5 March 2018 | The KHENERGY Study | An 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 Disease | Mitochondrial Diseases;Mitochondrial Myopathies;Mitochondrial Encephalomyopathies;MELAS;MIDD | Drug: placebo;Drug: KH176 | Khondrion BV | Radboud Center for Mitochondrial Medicine (RCMM);Radboud University | Not recruiting | 18 Years | N/A | All | 20 | Phase 2 | Netherlands |
| 8 | NCT02805790 | August 22, 2016 | 11 June 2018 | A 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 Study | A 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 Study | Primary Mitochondrial Disease | Drug: Elamipretide;Drug: Placebo | Stealth BioTherapeutics Inc. | Not recruiting | 16 Years | N/A | All | 30 | Phase 2 | United States | |
| 9 | JPRN-JMA-IIA00214 | 02/06/2015 | 23 April 2019 | Long-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 symptoms | Intervention 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 Hospital | Not Recruiting | >=9 MONTHS | <30 MONTHS | BOTH | 10 | Phase 2 | Japan | |
| 10 | NCT02473445 | May 19, 2015 | 11 June 2018 | A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease | A 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 Disease | Mitochondrial Diseases | Drug: Cysteamine Bitartrate | Horizon Pharma USA, Inc. | Not recruiting | 6 Years | 17 Years | All | 22 | Phase 2 | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 11 | NCT02544217 | May 2015 | 15 February 2016 | A Dose-escalating Clinical Trial With KH176 | A Phase I, Randomized, Double Blind, Placebo-controlled, Dose-escalating Clinical Trial With KH176 | MELAS;LHON;Leigh Syndrome;Mitochondrial Disease;Mitochondrial DNA tRNALeu(UUR) m.3243A| Drug: KH176;Drug: placebo | Khondrion BV | Drug Research Unit Ghent, Belgium | Not recruiting | 18 Years | 55 Years | Male | 30 | Phase 1 | Belgium |
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| 12 | NCT02367014 | February 2015 | 13 June 2016 | A Study Investigating the Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial Myopathy | A 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 Disease | Mitochondrial Myopathy | Drug: MTP-131;Drug: Placebo | Stealth BioTherapeutics Inc. | Not recruiting | 16 Years | 65 Years | Both | 36 | Phase 1/Phase 2 | United States | |
| 13 | JPRN-JMA-IIA00200 | 10/12/2014 | 2 April 2019 | Multicenter trial of SPP-004 in mitochondrial diseases | Multicenter trial of SPP-004 in mitochondrial diseases | Mitochondrial disease, mainly to cranial nerve symptoms | Intervention 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 Hospital | Not Recruiting | >=3 YEARS | <2 YEARS | BOTH | 10 | Phase 2 | Japan | |
| 14 | NCT02023866 | May 2014 | 16 December 2017 | Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease | An 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 Disease | Inherited Mitochondrial Disease, Including Leigh Syndrome | Drug: Cysteamine Bitartrate | Horizon Pharma USA, Inc. | Not recruiting | 6 Years | 17 Years | All | 36 | Phase 2 | United States | |
| 15 | NCT02053766 | January 2014 | 10 December 2018 | Anesthesia in Patients With Mitochondrial Disease | Anesthesia in Patients With Mitochondrial Disease | Mitochondrial Diseases | Drug: Sevoflurane;Drug: Dexmedetomidine;Drug: Propofol | The University of Texas Health Science Center, Houston | Recruiting | N/A | 17 Years | All | 60 | N/A | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 16 | NCT01642056 | September 1, 2012 | 7 October 2019 | EPI-743 for Metabolism or Mitochondrial Disorders | Therapeutic Trial of EPI -743 In Patients With Disorders of Energy Utilization or Oxidation-Reduction | Mitochondrial Disease;Neurology;Myoptahy | Drug: EPI-743;Drug: Placebo | National Human Genome Research Institute (NHGRI) | Not recruiting | 2 Years | 11 Years | All | 20 | Phase 1/Phase 2 | United States | |
| 17 | NCT01831934 | September 2010 | 16 December 2017 | Responses to Influenza Vaccine in Patients With Mitochondrial Disorders (MELAS) | Metabolic and Immune Responses to TIV in Patients With Mitochondrial Disease | MELAS Syndrome | Biological: Fluzone® | Stanford University | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Not recruiting | 13 Years | 65 Years | All | 22 | Phase 4 | United States |
| 18 | NCT00432744 | January 2007 | 16 December 2017 | Phase III Trial of Coenzyme Q10 in Mitochondrial Disease | Phase 3 Trial of Coenzyme Q10 in Mitochondrial Disease | Mitochondrial Diseases | Drug: CoenzymeQ10;Drug: Placebo | University of Florida | FDA Office of Orphan Products Development;Food and Drug Administration | Not recruiting | 12 Months | 17 Years | All | 24 | Phase 3 | United States;Canada |
| 19 | NCT01001585 | September 2006 | 16 March 2015 | Anesthetic Effects in Mitochondrial Disease | Anesthetic Effects in Mitochondrial Disease | Mitochondrial Disease | Drug: sevoflurane | d sessler | Not recruiting | 12 Months | 16 Years | Both | 55 | N/A | United States | |
| 20 | NCT00060515 | April 2003 | 19 February 2015 | RG2133 (2',3',5'-Tri-O-Acetyluridine) in Mitochondrial Disease | An 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 Diseases | Mitochondrial Diseases | Drug: RG2133 (2',3',5'-tri-O-acetyluridine) | Repligen Corporation | Not recruiting | 3 Years | N/A | Both | 12 | Phase 1 | United States |