+ Site Statistics
+ Search Articles
+ PDF Full Text Service
How our service works
Request PDF Full Text
+ Follow Us
Follow on Facebook
Follow on Twitter
Follow on LinkedIn
+ Subscribe to Site Feeds
Most Shared
PDF Full Text
+ Translate
+ Recently Requested

How clinical trials of myasthenia gravis can inform pre-clinical drug development

How clinical trials of myasthenia gravis can inform pre-clinical drug development

Experimental Neurology 270: 78-81

Pre-clinical evaluations often provide the rationale for therapeutic assessments in humans; however, in many diseases an agent found successful in animal models does not show efficacy in human subjects. Our contention is that the approach of rigorous, clinical trials can be used to inform how preclinical assessments should be performed. Clinical trials in humans are carefully designed investigations executed with consideration of critical methodological issues, such as pre-specified entrance criteria and validated, outcome measures coupled with power analysis to identify sample size. Blinding of evaluators of subjective measures and randomization of subjects are also critical aspects of trial performance. Investigative agents are also tested in subjects with active disease, rather than prior to disease induction as in some pre-clinical assessments. Application of standard procedures, including uniform reporting standards, would likely assist in reproducibility of pre-clinical experiments. Adapting methods of clinical trial performance will likely improve the success rate of therapeutics to ultimately achieve human use.

Please choose payment method:

(PDF emailed within 0-6 h: $19.90)

Accession: 058010601

Download citation: RISBibTeXText

PMID: 25592627

DOI: 10.1016/j.expneurol.2014.12.022

Related references

A randomised clinical trial comparing prednisone and azathioprine in myasthenia gravis. Results of the second interim analysis. Myasthenia Gravis Clinical Study Group. Journal of Neurology, Neurosurgery, and Psychiatry 56(11): 1157-1163, 1993

Clinical trial of plasma exchange and high-dose intravenous immunoglobulin in myasthenia gravis. Myasthenia Gravis Clinical Study Group. Annals of Neurology 41(6): 789-796, 1997

Recommendations for myasthenia gravis clinical trials. Muscle and Nerve 47(1): 144-145, 2013

Controlled clinical trials of anything in myasthenia gravis. Archives of Neurology 54(11): 1323, 1997

Recommendations for myasthenia gravis clinical trials. Muscle and Nerve 45(6): 909-917, 2012

Discussion of "clinical course and prognosis of myasthenia gravis" and "clinical study in myasthenia gravis". Shinkei Kenkyu no Shimpo. Advances in Neurological Sciences 15(4): 903-905, 1971

Clinical trials for myasthenia gravis: a historical perspective. Annals of the new York Academy of Sciences 1413(1): 5, 2018

Recent clinical trials on treatment of myasthenia gravis. Rinsho Shinkeigaku 52(11): 832-835, 2012

VATS thymectomy for nonthymomatous myasthenia gravis: standardized outcome assessment using the myasthenia gravis foundation of America clinical classification. Innovations 6(2): 104-109, 2012

Clinical characteristics of early onset autoimmune myasthenia gravis and persistent neonatal myasthenia gravis. Annals of Neurology 18(3): 401, 1985

Therapy of myasthenia gravis; clinical experiences with the treatment of myasthenia gravis pseudoparalytica with pyridostigmine. Deutsche Medizinische Wochenschrift 78(22): 805-807, 1953

Comparison of clinical manifestations between patients with ocular myasthenia gravis and generalized myasthenia gravis. Korean Journal of Ophthalmology 25(1): 1-7, 2011

Choosing the best animal model for pre-clinical trials of autoimmune myasthenia gravis. Neuromuscular Disorders 25: S244-S245, 2015

Comparative effectiveness clinical trials to advance treatment of myasthenia gravis. Annals of the new York Academy of Sciences 1413(1): 69-75, 2018

The MG composite: an outcome measure for myasthenia gravis for use in clinical trials and everyday practice. Annals of the new York Academy of Sciences 1274: 99, 2012