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

Levy, Gilberto; Kaufmann, Petra; Buchsbaum, Richard; Montes, Jacqueline; Barsdorf, Alexandra I.; Arbing, Rachel; Battista, Vanessa; Zhou, Xiaowen; Mitsumoto, Hiroshi; Levin, B.; Thompson, John L.P.

doi:10.1212/01.wnl.0000201182.60750.66

Cited by 94 publications

(62 citation statements)

References 28 publications

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“…If there are several interventions or dosages of interest, and the objective is to decide which should be investigated further, a selection design can be employed [43]. The "best" agent or dosage to study is selected as the one that has the superior effect (e.g., has the highest group mean or highest percentage of positive responses); formal statistical comparisons are not performed.…”

Section: Designs For Late Exploratory Clinical Trial Designsmentioning

confidence: 99%

Designing Clinical Trials for Dystonia

et al. 2014

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With advances in the understanding of the pathophysiology of dystonia, novel therapeutics are being developed. Such therapies will require clinical investigation ranging from exploratory studies to examine safety, tolerability, dosage selection, and preliminary efficacy to confirmatory studies to evaluate efficacy definitively. As dystonia is a rare and complex disorder with clinical and etiological heterogeneity, clinical trials will require careful consideration of the trial design, including enrollment criteria, concomitant medication use, and outcome measures. Given the complexities of designing and implementing efficient clinical trials, it is important for clinicians and statisticians to collaborate closely throughout the clinical development process and that each has a basic understanding of both the clinical and statistical issues that must be addressed. To facilitate designing appropriate clinical trials in this field, we review important general clinical trial and regulatory principles, and discuss the critical components of trials with an emphasis on considerations specific to dystonia. Additionally, we discuss designs used in early exploratory, late exploratory, and confirmatory phases, including adaptive designs.

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Section: Designs For Late Exploratory Clinical Trial Designsmentioning

confidence: 99%

Designing Clinical Trials for Dystonia

et al. 2014

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“…Likewise, very high doses of coenzyme Q10 are tried for the treatment of various other diseases such as Parkinson's disease, Huntington's disease, and amyotrophic lateral sclerosis. The dosage under clinical trials is currently up to 3,000 mg/day for human use (Ferrante et al, 2005;Levy et al, 2006). For animal study, the dosage was up to 20,000 mg/kg/day as reported by previous researchers (Yang et al, 2005;Smith et al, 2006).…”

Section: Discussionmentioning

confidence: 99%

Relative Bioavailability of Coenzyme Q10 in Emulsion and Liposome Formulations

Choi¹,

Kim²,

Shanmugam³

et al. 2010

Biomolecules and Therapeutics

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-The purpose of this study was to evaluate relative bioavailability of the coenzyme Q10 (CoQ10) in emulsion and three liposome formulations after a single oral administration (60 mg/kg) into rats. Emulsion formulation of CoQ10 was prepared by conventional method using Phospholipon 85G as an emulsifier, and three liposome formulations (neutral, anionic, and cationic) of CoQ10 were prepared by traditional lipid film hydration technique using Phospholipon 85G, cholesterol, and charge carrier lipids (1,2-dioleoyl-

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“…In a phase II clinical trial, it was well tolerated and safe (Ferrante et al, 2005), but showed no efficacy in a phase II futility trial (NCT00243932) (Kaufmann et al, 2009;Levy et al, 2006).…”

Section: Coenzyme Q10mentioning

confidence: 99%

“…The symptoms of motor neuron disease (MND) have first been described by several neurologists by the mid-19 th century. The French neurologist Charcot defined the nosological entity "amyotrophic lateral sclerosis" (ALS) some years later (historic aspects of ALS are reviewed in (Eisen, 2007;Mitsumoto et al, 2006;Oliveira & Pereira, 2009;Rowland, 2001;Wijesekera & Leigh, 2009)). In the present understanding, MND comprehends a spectrum of different neurodegenerative syndromes which show a common neuropathology, i. e. the progressive degeneration of motor neurons.…”

Section: Introductionmentioning

confidence: 99%