Mitochondrial Disease
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Sign up freeTop drugs studied for this disease
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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 …
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CLINICAL_TRIALS trial_result · 2026-03-25Clinical Trial Update: A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy [Phase 2, Phase 3]
A Phase 2/3 study evaluating Vatiquinone for mitochondrial disease in participants with refractory epilepsy has been terminated, and results…
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CLINICAL_TRIALS trial_result · 2026-01-22Clinical Trial Update: OMT-28 in Patients With Primary Mitochondrial Disease (PMD) (PMD-OPTION) [Phase 2]
Phase 2 clinical trial of OMT-28 in Primary Mitochondrial Disease has completed.
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CLINICAL_TRIALS trial_result · 2025-06-22Clinical Trial Update: Human Mitochondrial Stress-driven Obesity Resistance
A completed clinical trial investigating human mitochondrial stress-driven obesity resistance.
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CLINICAL_TRIALS trial_result · 2025-04-15Clinical Trial Update: Mitochondrial Stress, Brain Imaging, and Epigenetics [NA]
Completed clinical trial investigating mitochondrial stress, brain imaging, and epigenetics in mitochondrial disease. Results have been post…
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CLINICAL_TRIALS trial_result · 2024-08-29Clinical Trial Update: Magnetic Resonance Imaging (MRI) Muscle Phenotyping in Mitochondrial Disease
This clinical trial update reports the completion of a study using MRI muscle phenotyping to characterize mitochondrial disease at the Unive…
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CLINICAL_TRIALS trial_result · 2024-07-17Clinical Trial Update: Anesthesia in Patients With Mitochondrial Disease [NA]
A clinical trial studying anesthesia in patients with mitochondrial disease was terminated, and its results have been posted on ClinicalTria…
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CLINICAL_TRIALS trial_result · 2021-12-17Clinical Trial Update: Open-Label Extension Trial to Characterize the Long-term Safety and Tolerability of Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Myopathy (PMM) [Phase 2]
Open-label extension study terminated; aims to characterize long-term safety and tolerability of elamipretide in genetically confirmed prima…
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CLINICAL_TRIALS trial_result · 2021-10-07Clinical Trial Update: Assessment of Small Fiber Neuropathy in Rare Diseases Using Sudoscan [NA]
A completed clinical trial assessing small fiber neuropathy in Fabry disease and related conditions using Sudoscan; results posted in 2021.
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CLINICAL_TRIALS trial_result · 2021-04-14Clinical Trial Update: EPI-743 for Metabolism or Mitochondrial Disorders [Phase 1, Phase 2]
A clinical trial update for EPI-743 in mitochondrial disease and related neurology/myopathy conditions shows completion with results posted …
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CLINICAL_TRIALS trial_result · 2020-11-12Clinical Trial Update: A Dose-escalating Clinical Trial With KH176 [Phase 1]
Phase 1 dose-escalation trial of KH176 in mitochondrial diseases completed with results posted.
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CLINICAL_TRIALS trial_result · 2020-07-07Clinical Trial Update: Safety, Tolerability, Efficacy of MTP-131 for Treatment of Mitochondrial Disease in Subjects From the MMPOWER Study [Phase 2]
Phase 2 trial update on MTP-131 for primary mitochondrial disease reporting safety, tolerability, and efficacy results.
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CLINICAL_TRIALS trial_result · 2017-11-13Clinical Trial Update: Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease [Phase 2]
Phase 2 open-label, dose-escalation study of RP103 to assess safety, tolerability, and efficacy in inherited mitochondrial diseases, includi…
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CLINICAL_TRIALS trial_result · 2017-08-17Clinical Trial Update: Calf Muscle Strength in Mitochondrial Diseases
A clinical trial evaluating calf muscle strength in mitochondrial diseases has completed. The update does not provide results.
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CLINICAL_TRIALS trial_result · 2017-08-17Clinical Trial Update: GDF-15 as a Biomarker for Mitochondrial Disease
The clinical trial evaluating GDF-15 as a biomarker for mitochondrial disease has completed. It studied mitochondrial disease, metabolic myo…
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CLINICAL_TRIALS trial_result · 2017-07-18Clinical Trial Update: Energy Requirements in Mitochondrial Disease
A completed clinical trial from the University of British Columbia studying energy requirements in mitochondrial disease.
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CLINICAL_TRIALS trial_result · 2015-03-09Clinical Trial Update: Anesthetic Effects in Mitochondrial Disease [NA]
A clinical trial evaluating anesthetic effects in individuals with mitochondrial disease has been terminated.
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CLINICAL_TRIALS trial_result · 2012-12-18Clinical Trial Update: Economic and Medical Evaluation of the Whole Mitochondrial DNA Screening by Surveyor and Mitochips Techniques
A completed clinical trial evaluating the economic and medical value of whole mitochondrial DNA screening using Surveyor and Mitochip techni…
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CLINICAL_TRIALS trial_result · 2012-04-30Clinical Trial Update: Defining 31Phosphorous Magnetic Resonance Spectroscopy Characteristics in Patients With Mitochondrial Myopathy
Completed clinical trial evaluating 31P magnetic resonance spectroscopy characteristics in patients with mitochondrial disease, including mi…
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CLINICAL_TRIALS trial_result · 2011-04-22Clinical Trial Update: Safety and Efficacy Study of A0001 in Patients With the A3243G Mitochondrial DNA Point Mutation [Phase 2]
This Phase 2 safety and efficacy trial of A0001 in patients with the A3243G mitochondrial DNA mutation was terminated.