Design and implementation of the first randomized controlled trial of coenzyme Q10 in children with primary mitochondrial diseases |
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| Authors: | Peter W Stacpoole TonJ deGrauw Annette S Feigenbaum Charles Hoppel Douglas S Kerr Shawn E McCandless Michael V Miles Brian H Robinson Peter H Tang |
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| Institution: | 1. University of Florida, USA;2. Cincinnati Children''s Hospital Medical Center, USA;3. The Hospital for Sick Children and University of Toronto, Canada;4. Case Western Reserve University, USA;1. Department of Pediatrics, Johns Hopkins Medical Institutions, Baltimore, MD;2. Laboratory of Infectious Diseases, National Institutes of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD;3. Stanley Division of Developmental Neurovirology, Johns Hopkins University School of Medicine, Baltimore, MD;4. Department of Pediatrics, Feinberg School of Medicine of Northwestern University, Chicago, IL;5. Division of Viral Diseases, Centers for Disease Control and Prevention, Atlanta, GA;6. Department of Biostatistics, University of Michigan School of Public Health, Ann Arbor, MI;1. Department of Obstetrics and Gynaecology, Zhongda Hospital, School of Medicine, Southeast University, Nanjing, China;2. School of Public Health, Southeast University, Nanjing, China;3. Ministry of Education Key Laboratory of Environmental Medicine Engineering, Southeast University, Nanjing, China;1. Department of Neurological Surgery, Oregon Health & Science University, Portland, OR;2. Doernbecher Children''s Hospital, Portland, OR;3. Department of Neurological Surgery, Boston Children''s Hospital, Boston, MA;4. Harvard Medical School, Boston, MA;5. Departments of Neurology and Pediatrics, The Children''s Hospital of Philadelphia, Philadelphia, PA;6. Perelman School of Medicine of the University of Pennsylvania, Philadelphia, PA;7. Division of Gastroenterology, Hepatology, and Nutrition, The Children''s Hospital of Philadelphia, Philadelphia, PA;8. Children''s Hospital Colorado, Aurora, CO;9. University of Colorado, Aurora, CO;10. Division of Genomic Diagnostics, The Children''s Hospital of Philadelphia, Philadelphia, PA;11. Division of Gastroenterology, Hepatology, and Nutrition, The Hospital for Sick Children, Toronto, Ontario, Canada;12. University of Toronto, Toronto, Ontario, Canada;1. Department of Cardiology and Internal Medication, Military Hospital of Ouakam, Dakar, Senegal;2. Department of Cardiology, Principal Hospital of Dakar, Dakar, Senegal;3. Department of Cardiology, University Cheickh Anta Diop, Dakar, Senegal |
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| 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. |
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