Safety and efficacy of (+)-epicatechin in subjects with Friedreich's ataxia: A phase II, open-label, prospective study.

Journal of Inherited Metabolic Disease
Muhammad Yasir QureshiRalitza H Gavrilova

Abstract

(+)-Epicatechin (EPI) induces mitochondrial biogenesis and antioxidant metabolism in muscle fibers and neurons. We aimed to evaluate safety and efficacy of (+)-EPI in pediatric subjects with Friedreich's ataxia (FRDA). This was a phase II, open-label, baseline-controlled single-center trial including 10 participants ages 10 to 22 with confirmed FA diagnosis. (+)-EPI was administered orally at 75 mg/d for 24 weeks, with escalation to 150 mg/d at 12 weeks for subjects not showing improvement of neuromuscular, neurological or cardiac endpoints. Neurological endpoints were change from baseline in Friedreich's Ataxia Rating Scale (FARS) and 8-m timed walk. Cardiac endpoints were changes from baseline in left ventricular (LV) structure and function by cardiac magnetic resonance imaging (MRI) and echocardiogram, changes in cardiac electrophysiology, and changes in biomarkers for heart failure and hypertrophy. Mean FARS/modified (m)FARS scores showed nonstatistically significant improvement by both group and individual analysis. FARS/mFARS scores improved in 5/9 subjects (56%), 8-m walk in 3/9 (33%), 9-peg hole test in 6/10 (60%). LV mass index by cardiac MRI was significantly reduced at 12 weeks (P = .045), and was improved in 7/10 (7...Continue Reading

References

May 1, 1991·Cephalalgia : an International Journal of Headache·C M GibbM Sandler
Dec 13, 2000·Neurology·J B SchulzM B Bogdanov
Jun 22, 2002·Current Opinion in Genetics & Development·Hélène Puccio, Michel Koenig
Oct 9, 2002·Neuropediatrics·R ArtuchM Pineda
Mar 25, 2004·Neurochemical Research·J L BradleyJ M Cooper
Mar 5, 2005·Movement Disorders : Official Journal of the Movement Disorder Society·David R LynchLaura J Balcer
Apr 13, 2005·Neurology·S H SubramonyUNKNOWN Cooperative Ataxia Group
Feb 1, 2008·European Journal of Paediatric Neurology : EJPN : Official Journal of the European Paediatric Neurology Society·Mercè PinedaRafael Artuch
Feb 12, 2009·Muscle & Nerve·Louise R Rodino-KlapacJerry R Mendell
Apr 11, 2009·Journal of Neurology·Thomas Meier, Gunnar Buyse
May 6, 2010·Journal of Cerebral Blood Flow and Metabolism : Official Journal of the International Society of Cerebral Blood Flow and Metabolism·Zahoor A ShahSylvain Doré
Jun 29, 2010·Journal of the American College of Cardiology·Katrina Go YamazakiFrancisco J Villarreal
Aug 11, 2010·Archives of Neurology·David R LynchThomas Meier
Apr 8, 2011·Antioxidants & Redox Signaling·David L KatzAther Ali
Jul 27, 2011·The Journal of Physiology·Leonardo NogueiraMoh H Malek
Dec 20, 2011·FASEB Journal : Official Publication of the Federation of American Societies for Experimental Biology·Maik HüttemannMoh H Malek
May 29, 2012·Expert Opinion on Emerging Drugs·Vinod MalikJerry R Mendell
Aug 25, 2012·Molecular and Cellular Neurosciences·Rachael A Vaubel, Grazia Isaya
Dec 10, 2013·The Journal of Nutritional Biochemistry·Gabriela Gutierrez-SalmeanIsrael Ramirez-Sanchez
Aug 22, 2014·The Journals of Gerontology. Series A, Biological Sciences and Medical Sciences·Aldo Moreno-UlloaIsrael Ramirez-Sanchez
Sep 21, 2016·Annals of Clinical and Translational Neurology·Maya PatelDavid R Lynch
Aug 3, 2017·Brain Research·Xiao-Hui LiSheng-Li Huang
Feb 9, 2018·Molecular Aspects of Medicine·Cesar G FragaMonica Galleano
Jul 28, 2018·Neurodegenerative Disease Management·Theresa ZesiewiczMatthew B Klein
Oct 17, 2018·European Journal of Neurology : the Official Journal of the European Federation of Neurological Societies·T J R RezendeM C França
Feb 6, 2020·International Journal of Molecular Sciences·Piergiorgio La RosaFiorella Piemonte
Feb 12, 2020·Neurology. Genetics·Christian RummeyDavid R Lynch

❮ Previous
Next ❯

Citations

Jul 30, 2020·Antioxidants·Laura R RodríguezJuan Antonio Navarro Langa
Mar 26, 2021·Journal of Inherited Metabolic Disease·Patrick F ChinneryShamima Rahman

❮ Previous
Next ❯

Related Concepts

Related Feeds

Ataxias (MDS)

Ataxia is a neurological condition characterized by lack of voluntary coordination of muscle movements including loss of coordination, balance, and speech. Discover the latest research on ataxia here.

Ataxia

Ataxia is a neurological condition characterized by lack of voluntary coordination of muscle movements including loss of coordination, balance, and speech. Discover the latest research on ataxia here.

Ataxias

Ataxia is a neurological condition characterized by lack of voluntary coordination of muscle movements including loss of coordination, balance, and speech. Discover the latest research on different types of ataxias here.

Related Papers

Irish Journal of Medical Science
P A MCNALLY
Annals of Internal Medicine
E J GAUTHIER
Cardiovascular Research
K J OsterzielMichael Bunse
© 2021 Meta ULC. All rights reserved