Mitochondrial Diseases
📧 Sign up to get email alerts when something new happens for Mitochondrial Diseases.
Sign up freeTop drugs studied for this disease
-
CLINICAL_TRIALS trial_result · 2026-04-22Clinical Trial Update: Effect Of The System In The Enriched Fat Metabolism Of The Child Reached Mitochondrial Cytopathy [NA]
A completed clinical trial evaluating a system for enriched fat metabolism in children with mitochondrial diseases.
-
CLINICAL_TRIALS trial_result · 2026-04-20Clinical Trial Update: Digital Health Technology for People With Mitochondrial Disease [NA]
The clinical trial update indicates that the study evaluating digital health technology for people with mitochondrial diseases has completed…
-
CLINICAL_TRIALS trial_result · 2026-04-07Clinical Trial Update: Open Label Extension to Assess Long Term Safety and Efficacy of KL1333 in Patients With Primary Mitochondrial Disease [Phase 2]
Open-label extension study to evaluate long-term safety and efficacy of KL1333 in patients with primary mitochondrial disease; Phase 2, not …
-
CLINICAL_TRIALS trial_result · 2025-11-28Clinical Trial Update: Reference Values of Intraepidermal Nerve Fiber Density in Children and Small Fiber Neuropathy in Neurodevelopmental Disorders
This clinical trial update provides reference values for intraepidermal nerve fiber density in children. It also investigates small fiber ne…
-
CLINICAL_TRIALS trial_result · 2025-10-24Clinical Trial Update: Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD) [Phase 3]
Phase 3 study evaluating the efficacy and safety of elamipretide in subjects with primary mitochondrial disease from nuclear DNA mutations h…
- Want full summaries, importance scores, and drug timelines? Upgrade to Plus or Pro.
-
CLINICAL_TRIALS trial_result · 2025-06-22Clinical Trial Update: Exercise-mediated Rescue of Mitochondrial Dysfunctions Driving Insulin Resistance [NA]
A completed clinical trial evaluated whether exercise can rescue mitochondrial dysfunction that drives insulin resistance.
-
CLINICAL_TRIALS trial_result · 2025-03-30Clinical Trial Update: Mitochondrial Diseases - Long-read Genome and Transcriptome Sequencing in Cases Unresolved After Short-read Genomics [NA]
A completed clinical trial evaluating long-read genome and transcriptome sequencing to resolve cases unresolved by short-read genomic method…
-
CLINICAL_TRIALS trial_result · 2025-01-13Clinical Trial Update: MItochondrial Diabetes LOw Carb- Diet Study [NA]
A clinical trial evaluating a low-carbohydrate diet for mitochondrial diabetes has completed.
-
CLINICAL_TRIALS trial_result · 2025-01-10Clinical Trial Update: The KHENERGYC Study [Phase 2]
Phase 2 KHENERGYC study for mitochondrial diseases is currently suspended.
-
CLINICAL_TRIALS trial_result · 2024-12-18Clinical Trial Update: Impact of Statin Therapy on Muscle Mitochondrial Function and Aerobic Capacity [Phase 4]
A Phase 4 clinical trial evaluating the effect of statin therapy on muscle mitochondrial function and aerobic capacity in patients with mito…
-
CLINICAL_TRIALS trial_result · 2024-11-29Clinical Trial Update: Promoting Resilience in Stress Management (PRISM) and Clinical-focused Narrative (CFN) Pilot in Adults With Primary Mitochondrial Disease (PMD). [Phase 1, Phase 2]
A Phase 1/2 clinical trial evaluating resilience-based interventions (PRISM) and narrative-focused care (CFN) in adults with primary mitocho…
-
CLINICAL_TRIALS trial_result · 2024-11-06Clinical Trial Update: Clinical Study of Hydroxytyrosol (HT) in Mitochondrial Diseases [NA]
A clinical study evaluating hydroxytyrosol for mitochondrial diseases has completed.
-
CLINICAL_TRIALS trial_result · 2024-09-03Clinical Trial Update: Muscle OXPHOS and Nutrient Homeostasis
This entry reports the completion of a clinical trial studying muscle oxidative phosphorylation and nutrient homeostasis.
-
CLINICAL_TRIALS trial_result · 2024-06-12Clinical Trial Update: Genomic Profiling of Mitochondrial Disease - Imaging Analysis for Precise Mitochondrial Medicine
A completed Mayo Clinic study reports on genomic profiling and imaging analysis to advance precision mitochondrial medicine.
-
CLINICAL_TRIALS trial_result · 2024-03-06Clinical Trial Update: The KHENEREXT Study [Phase 2]
Phase 2 clinical trial update for KHNEREXT study investigating a treatment for mitochondrial diseases, including the m.3243A>G mutation-rela…
-
CLINICAL_TRIALS trial_result · 2022-08-31Clinical Trial Update: The KHENERGYZE Study [Phase 2]
Phase 2 trial update for the KHENERGYZE study investigating a treatment for mitochondrial diseases and mitochondrial myopathies (including M…
-
CLINICAL_TRIALS trial_result · 2021-08-31Clinical Trial Update: A Study to Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-BM-BLD in Pediatric Patients With Pearson Syndrome [Phase 1, Phase 2]
A phase 1/2 clinical trial evaluating safety and therapeutic effects of MNV-BM-BLD transplantation in pediatric patients with Pearson syndro…
-
CLINICAL_TRIALS trial_result · 2019-10-29Clinical Trial Update: Mitochondria and Chronic Kidney Disease [Phase 2]
Phase 2 clinical trial on mitochondria-related interventions for symptoms in patients with mitochondrial diseases and those on hemodialysis;…
-
CLINICAL_TRIALS trial_result · 2018-06-28Clinical Trial Update: Imaging Neuromelanin and Iron in Dystonia/Parkinsonism
University College London provides an update on a clinical trial investigating imaging of neuromelanin and brain iron in dystonia and parkin…
-
CLINICAL_TRIALS trial_result · 2018-05-11Clinical Trial Update: A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease [Phase 2]
This update describes a long-term extension of a Phase 2 trial of cysteamine bitartrate delayed-release capsules (RP103) in children with in…
-
CLINICAL_TRIALS trial_result · 2018-02-23Clinical Trial Update: The KHENERGY Study [Phase 2]
Phase 2 trial update for the KHENERGY study in mitochondrial diseases has completed.
-
CLINICAL_TRIALS trial_result · 2018-01-03Clinical Trial Update: Heart Rate Variability in Response to Metformin Challenge [Phase 2]
Phase 2 trial update on heart rate variability in response to a metformin challenge; completed with posted results.
-
CLINICAL_TRIALS trial_result · 2017-09-21Clinical Trial Update: A Study of Bezafibrate in Mitochondrial Myopathy [Phase 2]
Phase 2 clinical trial of bezafibrate in mitochondrial diseases has completed.
-
CLINICAL_TRIALS trial_result · 2017-08-08Clinical Trial Update: Dichloroacetate Treatment of Congenital Lactic Acidosis Phase B: Expanded Access
Phase B expanded-access trial update on dichloroacetate for congenital lactic acidosis; the trial is no longer available.
-
CLINICAL_TRIALS trial_result · 2016-04-06Clinical Trial Update: Nutritional Assessment in Mitochondrial Cytopathy
A completed clinical trial assessing nutritional status in mitochondrial cytopathy, conducted by Insel Gruppe AG and University Hospital Ber…