How should we design future mechanistic and/or efficacy clinical trials?

被引：12

作者：

Fava, Maurizio ^{[1
]}

机构：

[1] Massachusetts Gen Hosp, Dept Psychiat, Boston, MA 02114 USA

来源：

NEUROPSYCHOPHARMACOLOGY | 2024年 / 49卷 / 01期

关键词：

MAJOR DEPRESSIVE DISORDER; PLACEBO-RESPONSE; DOUBLE-BLIND; ANTIDEPRESSANT EFFICACY; ADJUNCTIVE TREATMENT; INADEQUATE RESPONSE; THERAPY; BUPRENORPHINE/SAMIDORPHAN; AUGMENTATION; MEDICATIONS;

D O I：

10.1038/s41386-023-01600-9

中图分类号：

Q189 [神经科学];

学科分类号：

071006 ;

摘要：

The emergence of new molecular targets, together with the development of new approaches to neuropsychiatric diseases, involving psychedelics as well as gene and cell therapies, are creating the need to improve the efficiency of mechanistic and/or efficacy clinical trials. This review article will discuss a number of issues that have hampered our ability to detect therapeutic signals, from excessive placebo/sham response rates to the imprecision of diagnostic and outcome assessments. In addition to reviewing the limitations of current efficacy and mechanistic neuropsychiatric clinical trials, this review presents some of the methodological approaches that may improve the overall performance of our neuropsychiatric trials, including the adoption of novel study designs such as the sequential parallel comparison design and independent confirmation of the appropriateness of subjects' enrollment. In addition, this review will discuss several designs that make mechanistic clinical trials more precise.

引用

页码：197 / 204

页数：8

共 46 条

[1] Efficacy Outcomes From 3 Clinical Trials of Edivoxetine as Adjunctive Treatment for Patients With Major Depressive Disorder Who Are Partial Responders to Selective Serotonin Reuptake Inhibitor Treatment [J].

Ball, Susan G. ;

Ferguson, Margaret B. ;

Martinez, James M. ;

Pangallo, Beth A. ;

Nery, E. Serap Monkul ;

Dellva, Mary Anne ;

Sparks, JonDavid ;

Zhang, Qi ;

Liu, Peng ;

Bangs, Mark ;

Goldberger, Celine .

JOURNAL OF CLINICAL PSYCHIATRY, 2016, 77 (05) :E635-E642

[2] Psychometric evaluation of the Major Depression Inventory (MDI) as depression severity scale using the LEAD (Longitudinal Expert Assessment of All Data) as index of validity [J].

Bech, Per ;

Timmerby, N. ;

Martiny, K. ;

Lunde, M. ;

Soendergaard, S. .

BMC PSYCHIATRY, 2015, 15

[3]

BELLAMY N, 1988, J RHEUMATOL, V15, P1833

[4] Evaluation of performance of some enrichment designs dealing with high placebo response in psychiatric clinical trials [J].

Chen, Yeh-Fong ;

Yang, Yang ;

Hung, H. M. James ;

Wang, Sue-Jane .

CONTEMPORARY CLINICAL TRIALS, 2011, 32 (04) :592-604

[5] Effect of Dextromethorphan-Quinidine on Agitation in Patients With Alzheimer Disease Dementia A Randomized Clinical Trial [J].

Cummings, Jeffrey L. ;

Lyketsos, Constantine G. ;

Peskind, Elaine R. ;

Porsteinsson, Anton P. ;

Mintzer, Jacobo E. ;

Scharre, DouglasW. ;

De La Gandara, Jose E. ;

Agronin, Marc ;

Davis, Charles S. ;

Uyen Nguyen ;

Shin, Paul ;

Tariot, Pierre N. ;

Siffert, Joao .

JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION, 2015, 314 (12) :1242-1254

[6]

Daniel DG, 2010, 2010 AUT INT SOC CNS

[7] A repeated measures model for analysis of continuous outcomes in sequential parallel comparison design studies [J].

Doros, Gheorghe ;

Pencina, Michael ;

Rybin, Denis ;

Meisner, Allison ;

Fava, Maurizio .

STATISTICS IN MEDICINE, 2013, 32 (16) :2767-2789

[8]

Dotson S, 2019, ANN CLIN PSYCHIATRY, V31, P249

[9] A Meta-analysis of Factors Impacting Detection of Antidepressant Efficacy in Clinical Trials: The Importance of Academic Sites [J].

Dunlop, Boadie W. ;

Thase, Michael E. ;

Wun, Chuan-Chuan ;

Fayyad, Rana ;

Guico-Pabia, Christine J. ;

Musgnung, Jeff ;

Ninan, Philip T. .

NEUROPSYCHOPHARMACOLOGY, 2012, 37 (13) :2830-2836

[10] The double-blind variable placebo lead-in period: Results from two antidepressant clinical trials [J].

Faries, DE ;

Heiligenstein, JH ;

Tollefson, GD ;

Potter, WZ .

JOURNAL OF CLINICAL PSYCHOPHARMACOLOGY, 2001, 21 (06) :561-568

← 1 2 3 4 5 →