A two-stage design for a phase II clinical trial of coenzyme Q10 in ALS

Neurology
G LevyJ L P Thompson

Abstract

The combination of a small pool of patients at any given time with the availability of many potential neuroprotective agents to be tested in ALS requires efficient phase II trial designs. To describe the design of the Clinical Trial of High Dose Coenzyme Q10 (CoQ10) in ALS (QALS study)--a phase II, randomized, placebo-controlled, double-blind, multicenter clinical trial. The study design features two stages. The first stage (dose selection) identifies which of two doses of CoQ10 (1800 mg or 2700 mg) is preferred using a selection procedure rather than a formal hypothesis test. The second stage (early efficacy test) compares the preferred dose of CoQ10 against placebo using a non-superiority or futility design. Data from patients assigned to the preferred dose of CoQ10 in the first stage are also used in the second stage. The primary outcome measure is the decline in Amyotrophic Lateral Sclerosis Functional Rating Scale-revised (ALSFRSr) score from baseline to 9 months. The total sample size required is 185 patients, as compared to a much larger sample size estimated to be necessary using a conventional superiority design (total: 852 patients). The authors report a bias correction made necessary by the inclusion of patient data ...Continue Reading

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Citations

Jul 2, 2008·Current Neurology and Neuroscience Reports·Tiffini Voss, Bernard Ravina
Dec 17, 2008·Current Neurology and Neuroscience Reports·Jinsy Andrews
Aug 16, 2008·Archives of Pharmacal Research·Rengarajan BaskaranBong Kyu Yoo
Nov 21, 2008·Neurotherapeutics : the Journal of the American Society for Experimental NeuroTherapeutics·Maryam Oskoui, Petra Kaufmann
Apr 26, 2011·Journal of Biopharmaceutical Statistics·Sue-Jane WangRobert O'Neill
Jan 24, 2012·International Journal of Toxicology·Padmaja Yerramilli-RaoMerit E Cudkowicz
Aug 25, 2012·Trials·John A KairallaKeith E Muller
Dec 8, 2009·Neuropsychiatric Disease and Treatment·Stefano ZoccolellaPaolo Lamberti
Sep 8, 2012·Therapeutics and Clinical Risk Management·Philippe Corcia, Paul H Gordon
Nov 28, 2013·Neurotherapeutics : the Journal of the American Society for Experimental NeuroTherapeutics·Wendy R GalpernH A Jinnah
May 1, 2012·Clinical Investigation·James D Berry, Merit E Cudkowicz
Jun 5, 2013·Stroke; a Journal of Cerebral Circulation·Sharon D Yeatts
Nov 20, 2012·Current Opinion in Neurology·Matthew GladmanLorne Zinman
Nov 22, 2007·Expert Opinion on Therapeutic Targets·Juan C CoronaRicardo Tapia
Nov 27, 2009·Expert Opinion on Investigational Drugs·Veena LankaMerit Cudkowicz
Aug 10, 2007·Expert Opinion on Investigational Drugs·Jaydeep M Bhatt, Paul H Gordon
Jun 25, 2008·Amyotrophic Lateral Sclerosis : Official Publication of the World Federation of Neurology Research Group on Motor Neuron Diseases·Veena Lanka, Merit Cudkowicz
Feb 15, 2008·Amyotrophic Lateral Sclerosis : Official Publication of the World Federation of Neurology Research Group on Motor Neuron Diseases·David A Schoenfeld, Merit Cudkowicz
Nov 20, 2009·Amyotrophic Lateral Sclerosis : Official Publication of the World Federation of Neurology Research Group on Motor Neuron Diseases·Adriano ChiòUNKNOWN Eurals Consortium
Nov 20, 2009·Amyotrophic Lateral Sclerosis : Official Publication of the World Federation of Neurology Research Group on Motor Neuron Diseases·Richard BuchsbaumUNKNOWN QALS Study Group
Dec 8, 2009·Amyotrophic Lateral Sclerosis : Official Publication of the World Federation of Neurology Research Group on Motor Neuron Diseases·Merit E CudkowiczRobert G Miller
Sep 14, 2007·Amyotrophic Lateral Sclerosis : Official Publication of the World Federation of Neurology Research Group on Motor Neuron Diseases·Paul H GordonHiroshi Mitsumoto
Sep 14, 2007·Amyotrophic Lateral Sclerosis : Official Publication of the World Federation of Neurology Research Group on Motor Neuron Diseases·Geert Jan GroeneveldAlbert C Ludolph
Dec 9, 2009·Free Radical Biology & Medicine·Siân C Barber, Pamela J Shaw
Jul 16, 2008·Physical Medicine and Rehabilitation Clinics of North America·Jeremy M Shefner
Jul 16, 2008·Physical Medicine and Rehabilitation Clinics of North America·Jeffrey Rosenfeld, Amy Ellis
May 9, 2007·Mitochondrion·Wendy R Galpern, Merit E Cudkowicz
Feb 16, 2007·Neuromuscular Disorders : NMD·P KaufmannUNKNOWN International Coordinating Committee for SMA Subcommittee on SMA Clinical Trial Design
Jul 1, 2015·Contemporary Clinical Trials·Bruce Levin
Apr 22, 2009·Muscle & Nerve·Zachary Simmons
Sep 11, 2009·Annals of Neurology·Petra KaufmannUNKNOWN QALS Study Group
Feb 1, 2008·Muscle & Nerve·C E JacksonUNKNOWN Muscle Study Group
Nov 2, 2007·Journal of Neurochemistry·Carine ClerenM Flint Beal
Jun 1, 2011·Movement Disorders : Official Journal of the Movement Disorder Society·C Warren OlanowKarl Kieburtz
Oct 29, 2011·Experimental Neurology·Jonathan D Glass
Nov 9, 2017·Frontiers in Neuroscience·Serhiy Forostyak, Eva Sykova
Apr 20, 2012·The Cochrane Database of Systematic Reviews·Reto BaldingerMarkus Weber
Sep 4, 2018·Alzheimer's & Dementia : Translational Research & Clinical Interventions·Guogen ShanJeffrey L Cummings
Oct 15, 2019·Amyotrophic Lateral Sclerosis & Frontotemporal Degeneration·Suma BabuNazem Atassi

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