21. Mitochondrial disease Clinical trials / Disease details
Clinical trials : 33 / 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 | 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) | 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. | 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 | ||
| 2 | NCT05162768 (ClinicalTrials.gov) | March 2022 | 8/12/2021 | Study to Evaluate the Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease Resulting 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) - NuPower Study | Mitochondrial Myopathies;Mitochondrial Pathology;Mitochondrial DNA Mutation;Mitochondrial Diseases;Mitochondrial DNA Deletion;Mitochondrial DNA Depletion;Mitochondrial Metabolism Defect;Mitochondrial Complex I Deficiency | Drug: Elamipretide;Drug: Placebo | Stealth BioTherapeutics Inc. | NULL | Recruiting | 18 Years | 70 Years | All | 130 | Phase 3 | United States |
| 3 | NCT02976038 (ClinicalTrials.gov) | December 2016 | 18/11/2016 | 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. | NULL | Terminated | 16 Years | N/A | All | 36 | Phase 2 | United States |
| 4 | NCT02805790 (ClinicalTrials.gov) | August 22, 2016 | 13/6/2016 | Safety, Tolerability, Efficacy of MTP-131 for Treatment of Mitochondrial Disease in Subjects From the MMPOWER Study | A 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 Study | Primary Mitochondrial Disease | Drug: Elamipretide;Drug: Placebo | Stealth BioTherapeutics Inc. | NULL | Completed | 16 Years | N/A | All | 30 | Phase 2 | United States |
| 5 | NCT02367014 (ClinicalTrials.gov) | February 2015 | 9/2/2015 | Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial Myopathy | Phase 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 Disease | Mitochondrial Myopathy | Drug: elamipretide (low dose);Drug: elamipretide (intermediate dose);Drug: elamipretide (high dose);Drug: Placebo | Stealth BioTherapeutics Inc. | NULL | Completed | 16 Years | 65 Years | All | 36 | Phase 1/Phase 2 | United States |