Trial reportMitochondrion2012
Design and implementation of the first randomized controlled trial of coenzyme CoQ₁₀ in children with primary mitochondrial diseases.
Trial report in Mitochondrion, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. It is linked to trial NCT02616484 (Phase 3 Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency), which is not on this map. Cited by 15 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Phase 3 Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency:
Who cites it
15 citing papers in PubMed.
- Evaluating a Coenzyme Q10-Based Food for Special Medical Purpose, for Mitochondrial Diseases Management: An Open-Label, Pilot Trial.International journal of molecular sciences · 2026Trial
- Development of a novel observer reported outcome tool as the primary efficacy outcome measure for a rare disease randomized controlled trial.Mitochondrion · 2018Trial
- Boosting Mitochondrial Potential: An Imperative Therapeutic Intervention in Amyotrophic Lateral Sclerosis.Current neuropharmacology · 2023Review
- Therapeutic Approaches to Treat Mitochondrial Diseases: "One-Size-Fits-All" and "Precision Medicine" Strategies.Pharmaceutics · 2020Review
- Diagnosis and Treatment of Mitochondrial Myopathies.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2018Review
- Risk factors for poor bone health in primary mitochondrial disease.Journal of inherited metabolic disease · 2017Observational
- Succinyl-CoA synthetase (SUCLA2) deficiency in two siblings with impaired activity of other mitochondrial oxidative enzymes in skeletal muscle without mitochondrial DNA depletion.Molecular genetics and metabolism · 2017Article
- Muscle oxidative phosphorylation quantitation using creatine chemical exchange saturation transfer (CrCEST) MRI in mitochondrial disorders.JCI insight · 2016Article
- Mitochondrial disorders in children: toward development of small-molecule treatment strategies.EMBO molecular medicine · 2016Review
- Mitochondrial dysfunction in cardiac aging.Biochimica et biophysica acta · 2015Review
- Sleep disorders associated with primary mitochondrial diseases.Journal of clinical sleep medicine : JCSM : official publication of the American Academy of Sleep Medicine · 2014Review
- Development of pharmacological strategies for mitochondrial disorders.British journal of pharmacology · 2014Review
- Review of clinical trials for mitochondrial disorders: 1997-2012.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2013Review
- Drug development for rare mitochondrial disorders.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2013Review
- Maternally inherited mitochondrial respiratory disorders: from pathogenetic principles to therapeutic implications.Molecular genetics and metabolismReview
Corrections and comments
- Erratum issued
Authors and funding
9 authors.
Funding
Abstract
We report the design and implementation of the first phase 3 trial of CoenzymeQ₁₀ (CoQ₁₀) in children with genetic mitochondrial diseases. A novel, rigorous set of eligibility criteria was established. The trial, which remains open to recruitment, continues to address multiple challenges to the recruitment of patients, including widely condoned empiric use of CoQ₁₀ by individuals with proven or suspected mitochondrial disease and skepticism among professional and lay mitochondrial disease communities about participating in placebo-controlled trials. These attitudes represent significant barriers to the ethical and scientific evaluation--and ultimate approval--of nutritional and pharmacological therapies for patients with life-threatening inborn errors of energy metabolism.
Indexed as
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What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.