Indication
Mitochondrial Diseases
15 clinical trials
14 products
1 drug
Clinical trial
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) NuPowerStatus: Active (not recruiting), Estimated PCD: 2024-09-01
Product
PlaceboProduct
ElamipretideClinical trial
A Phase IIb Open-label, Multi-centre, Extension Study to Explore the Long-term Safety and Efficacy of KH176 in Subjects With a Genetically Confirmed Mitochondrial DNA tRNALeu(UUR) m.3243A>G Mutation Who Have Completed the KHENERGYZE Study KH176-202.Status: Completed, Estimated PCD: 2023-06-01
Product
KH176Clinical trial
Emergency Use Protocol for EPI-743 in Acutely Ill Patients With Inherited Mitochondrial Respiratory Chain Disease Within 90 Days of End-of-Life CareStatus:
Product
EPI-743Product
VatiquinoneClinical trial
Efficacy and Safety Study of Vatiquinone for the Treatment of Mitochondrial Disease Subjects With Refractory EpilepsyStatus: Completed, Estimated PCD: 2023-03-18
Product
elamipretideClinical trial
An Intermediate Size Expanded Access Protocol of Elamipretide for Subcutaneous Injection in Patients With Genetically Confirmed Rare Diseases With Known Mitochondrial DysfunctionStatus:
Clinical trial
A Randomized Placebo Controlled, Double-blind Phase II Study to Explore the Safety, Efficacy and Pharmacokinetics of Sonlicromanol in Children With Genetically Confirmed Mitochondrial DiseaseStatus: Recruiting, Estimated PCD: 2024-12-01
Product
SonlicromanolClinical trial
Genetisk Diagnostik Vid medfödda Metabola SjukdomarStatus: , Estimated PCD: 2030-12-31
Product
IEM-EPClinical trial
Impact of Statin Therapy on Muscle Mitochondrial Function and Aerobic CapacityStatus: Completed, Estimated PCD: 2023-07-07
Product
LipitorClinical trial
IMPLEMENTING A SINGLE MUSCLE FIBER DNA QUANTIFICATION TECHNIQUE AS A INTERPRETATION TOOL FOR THE VARIANTS OF UNKNOWN SERVICE IN MITOCHONDRIAL DISEASESStatus: Completed, Estimated PCD: 2020-09-30
Product
samplesClinical trial
Interest of Multi-omics (WES / RNA-Seq) Approach to Fight Against the Diagnostic Deadlock in Mitochondrial DiseasesStatus: Recruiting, Estimated PCD: 2022-03-07
Product
Mitochondrial MyopathyClinical trial
A Natural History Study of Neurodegeneration and Optic Atrophy Caused by Ferredoxin Reductase Mutations in Pediatric and Adult PatientsStatus: Completed, Estimated PCD: 2023-08-17
Product
Mutation analysisClinical trial
A Natural History Study of Neurodegeneration and Optic Atrophy Caused by SLC25A46 Mutations in Pediatric and Adult PatientsStatus: Completed, Estimated PCD: 2023-08-17
Clinical trial
The Safety, Tolerability, Pharmacodynamics, and Efficacy of Erythrocyte Encapsulated Thymidine Phosphorylase (EE-TP) in Patients With MNGIEStatus: Withdrawn, Estimated PCD: 2027-09-01
Product
EE-TPClinical trial
A Phase II, Monocenter, Single Arm Study To Assess The Safety and Efficacy Of Combination Deoxycytidine and Deoxythymidine For Mitochondrial Depletion DisordersStatus: Recruiting, Estimated PCD: 2025-12-31
Clinical trial
A Phase III, Randomised, Double-blind, Placebo-controlled, Parallel-group, Pivotal Trial to Assess the Efficacy and Safety of Sonlicromanol in Adult Subjects With a Genetically Confirmed Mitochondrial DNA tRNALeu(UUR) m.3243A>G VariantStatus: Not yet recruiting, Estimated PCD: 2027-07-02
Drug
Varlilumab