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Found 57 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

  • 0 views
  • 07 Oct, 2021
  • 1 location
A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy

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 …

melas
epilepsy
electroencephalogram (eeg)
leigh's disease
ketogenic diet
  • 216 views
  • 10 Nov, 2021
  • 16 locations
A Study of the Efficacy and Safety of 24 Week Treatment With REN001 in Patients With Primary Mitochondrial Myopathy

This is a randomized, double-blind, placebo-controlled, parallel group, multi-centre, study designed to investigate the efficacy and safety of REN001 administered once daily over a 24-week period to patients with PMM.

  • 0 views
  • 19 Jan, 2022
  • 37 locations
The KHENERGYZE Study

Mitochondrial diseases, estimated prevalence 1 in 4,300 adults, is caused by pathogenic mutations in genes finally encoding for mitochondrial proteins of the various enzyme complexes of the

chromosomal aberration
phonophobia
leucine
nausea
vomiting
  • 44 views
  • 04 Jan, 2022
  • 4 locations
The KHENEREXT Study

This is an open-label, multi-centre study in subjects with a genetically confirmed mitochondrial deoxyribonucleic acid (DNA) transfer ribonucleic acid (tRNA)Leu(UUR) m.3243A>G mutation who completed study KH176-202. In the KH176-203 study subjects will be receiving KH176 100 mg BID or KH176 50 mg bid in die (BID) (as determined by the …

  • 0 views
  • 03 Nov, 2021
  • 4 locations
Anesthesia in Patients With Mitochondrial Disease

This pilot study is a prospective, randomized clinical trial to evaluate the effect of anesthesia in the mitochondrial dysfunction patient.

  • 26 views
  • 08 Sep, 2021
  • 1 location
Mitochondrial Diseases - Long-read Genome and Transcriptome Sequencing in Cases Unresolved After Short-read Genomics

The MiDiSeq project will enroll 20 unresolved index patients with suspected mitochondrial disease prioritized for genomic analysis.

  • 0 views
  • 08 Sep, 2021
  • 1 location
A Phase Ia/Ib SAD and MAD Study of of KL1333 in Healthy Subjects and Patients With Primary Mitochondrial Disease

only and will be completed before Part C including patients with primary mitochondrial disease will be initiated. The starting dose in the first cohort of Part A will be 25 mg. The dose level in the

body mass index
  • 0 views
  • 09 Sep, 2021
  • 2 locations
Drug-drug Interaction Study of KL1333 in Healthy Subjects

A Phase I, Open-label, Fixed-sequence, Crossover, Drug-drug Interaction Study to Investigate the Inhibition Potential of KL1333 on CYP1A2, CYP2B6, CYP2C8, CYP2C9, CYP2C19, CYP2D6, and CYP3A4 in Healthy Subjects

  • 0 views
  • 09 Sep, 2021
  • 1 location
Mitochondrial Stress Brain Imaging and Epigenetics

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 …

  • 0 views
  • 08 Sep, 2021
  • 1 location