Mitochondrial Diseases Clinical Trials

A listing of Mitochondrial Diseases medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

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Found 34 clinical trials
MABs Therapy m.3243A>G Mutation Carriers

Rationale: Mitochondrial disorders are progressive, often fatal multisystem disorders, in 20-25% of the cases caused by heteroplasmic mutations in the mitochondrial DNA (mtDNA). At this moment

  • 07 Oct, 2021
  • 1 location
Efficacy and Safety Study of Vatiquinone for the Treatment of Mitochondrial Disease Subjects With Refractory Epilepsy (MIT-E)

This is a parallel-arm, double-blind, placebo-controlled study with a screening phase that includes a 28-day run-in phase to establish baseline seizure frequency, followed by a 24-week, randomized, placebo-controlled phase. After completion of the randomized, placebo-controlled phase, participants may enter a 48-week, long-term, extension phase during which they will receive open-label …

lactic acidosis
leigh's disease
electroencephalogram (eeg)
  • 10 Oct, 2022
  • 28 locations
Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD) (NuPower)

SPIMD-301 is a 48-week, randomized, double-blind, parallel-group, placebo-controlled trial to assess efficacy and safety of single daily subcutaneous (SC) administration of elamipretide as a treatment for subjects with primary mitochondrial myopathy associated with nuclear DNA mutations (nPMD).

  • 07 Oct, 2022
  • 16 locations
Clinical Study of Hydroxytyrosol (HT) in Mitochondrial Diseases

Mitochondrial diseases (MDs) are the commonest group of inborn errors resulting from primary dysfunction of mitochondrial respiratory chain (MRC). High phenolics-containing extra-virgin olive

  • 04 Oct, 2022
  • 1 location
The Development of Minimally Invasive Nanosensor Technology to Quantify Mitochondrial Function in Human Muscle

Past mitochondrial disease treatment studies have been unsuccessful in determining treatment efficacy, and a major factor has been the lack of validated biomarkers in mitochondrial myopathy (MM

Accepts healthy volunteers
  • 23 Oct, 2022
  • 1 location

confirmed mitochondrial disease of which the gene defect is known to decrease one or more oxidative phosphorylation system enzymes and who suffer from motor symptoms ("KHENERGYC").

  • 23 Mar, 2022
  • 1 location
Mitochondrial Stress, Brain Imaging, and Epigenetics (MiSBIE)

The MiSBIE study collects biological, behavioral, psychosocial, neuropsychological, and brain imaging data in participants with either: normal mitochondrial function, individuals with the m.3243A>G mitochondrial DNA (mtDNA) mutation, and individuals a single large-scale mtDNA deletion. These defects induce mitochondrial allostatic load (MAL). The 2-day protocol, plus home-based data collection, will provide …

Accepts healthy volunteers
  • 04 Oct, 2022
  • 1 location
Impact of Statin Therapy on Muscle Mitochondrial Function and Aerobic Capacity

The purpose of this study is to test how different doses of a statin, Lipitor, affect muscle health and function, and cardiovascular fitness.

body mass index
Accepts healthy volunteers
lipitor 20mg tablet
muscle biopsy
  • 11 May, 2022
  • 2 locations
Study of N-acetylcysteine in the Treatment of Patients With the m.3243A>G Mutation and Low Brain Glutathione Levels

N-Acetylcysteine (NAC), an anti-oxidant, will be studied to investigate the effects on brain glutathione levels, cognitive skills, motor skills, and quality of life. A group of 18 patients will take either 1800, 3600 or 5400 mg per day of NAC (for 3 months) for a dose escalation study; this means …

  • 08 Jun, 2022
  • 1 location
L-Citrulline Dose Finding Safety Study in MELAS

The main purpose of this study is to determine the safest maximum dose of an amino acid, citrulline, which will be used as potential treatment for adult patients with a disorder of energy metabolism called Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like episodes (MELAS). Once established, this dose will be used …

  • 06 May, 2022
  • 1 location