Advances in Clinical Trials For Movement Disorders

被引:6
作者
Kieburtz, Karl [1 ]
Olanow, C. Warren [2 ]
机构
[1] Univ Rochester, Sch Med, Rochester, NY 14642 USA
[2] Mt Sinai Sch Med, New York, NY USA
关键词
Parkinson; Huntington; Clinical Trials; VIRTUAL HOUSE CALLS; PARKINSONS-DISEASE; DOUBLE-BLIND; OPEN-LABEL; DELAYED-START; HUNTINGTONS-DISEASE; MOTOR FLUCTUATIONS; SUBSTANTIA-NIGRA; GENE DELIVERY; LEVODOPA;
D O I
10.1002/mds.26371
中图分类号
R74 [神经病学与精神病学];
学科分类号
100204 [神经病学];
摘要
In the past several years, there have been several innovations in the design of clinical trials assessing new therapies for patients with movement disorders. These include attempts to address difficulties in conducting clinical trials in treated patients in the advanced stages of their illness, demonstrating disease-modifying effects or a reduction in the development of cumulative disability, and assessing the effects of interventions in patients in the premanifest state of their disease. In addition, there have been advances in clinical trial methodologies and changes in regulatory guidelines that permit the performance of more efficient studies, with a reduction in the cost and duration of the development period. These will be reviewed in the present article. (c) 2015 International Parkinson and Movement Disorder Society
引用
收藏
页码:1580 / 1587
页数:8
相关论文
共 51 条
[1]
Virtual house calls for Parkinson disease (Connect.Parkinson): study protocol for a randomized, controlled trial [J].
Achey, Meredith A. ;
Beck, Christopher A. ;
Beran, Denise B. ;
Boyd, Cynthia M. ;
Schmidt, Peter N. ;
Willis, Allison W. ;
Riggare, Sara S. ;
Simone, Richard B. ;
Biglan, Kevin M. ;
Dorsey, E. Ray .
TRIALS, 2014, 15
[2]
Open-Label Surgical Trials for Parkinson Disease: Time for Reconsideration [J].
Alterman, Ron L. ;
Tagliati, Michele ;
Olanow, C. Warren .
ANNALS OF NEUROLOGY, 2011, 70 (01) :5-8
[3]
[Anonymous], 2014, Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics
[4]
[Anonymous], GUID IND ALZH DIS DE
[5]
Detecting and monitoring the symptoms of Parkinson's disease using smartphones: A pilot study [J].
Arora, S. ;
Venkataraman, V. ;
Zhan, A. ;
Donohue, S. ;
Biglan, K. M. ;
Dorsey, E. R. ;
Little, M. A. .
PARKINSONISM & RELATED DISORDERS, 2015, 21 (06) :650-653
[6]
Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients [J].
Bartus, Raymond T. ;
Baumann, Tiffany L. ;
Siffert, Joao ;
Herzog, Christopher D. ;
Alterman, Ron ;
Boulis, Nicholas ;
Turner, Dennis A. ;
Stacy, Mark ;
Lang, Anthony E. ;
Lozano, Andres M. ;
Olanow, C. Warren .
NEUROLOGY, 2013, 80 (18) :1698-1701
[7]
Bioactivity of AAV2-Neurturin Gene Therapy (CERE-120): Differences Between Parkinson's Disease and Nonhuman Primate Brains [J].
Bartus, Raymond T. ;
Herzog, Christopher D. ;
Chu, Yaping ;
Wilson, Alistair ;
Brown, Lamar ;
Siffert, Joao ;
Johnson, Eugene M., Jr. ;
Olanow, C. Warren ;
Mufson, Elliott J. ;
Kordower, Jeffrey H. .
MOVEMENT DISORDERS, 2011, 26 (01) :27-36
[8]
Deuschl G, 2013, NEW ENGL J MED, V368, P2038, DOI [10.1056/NEJMc1303485, 10.1056/NEJMoa1205158]
[9]
Novel Methods and Technologies for 21st-Century Clinical Trials A Review [J].
Dorsey, E. Ray ;
Venuto, Charles ;
Venkataraman, Vinayak ;
Harris, Denzil A. ;
Kieburtz, Karl .
JAMA NEUROLOGY, 2015, 72 (05) :582-588
[10]
Natural History of Huntington Disease [J].
Dorsey, E. Ray ;
Beck, Christopher A. ;
Darwin, Kristin ;
Nichols, Paige ;
Brocht, Alicia F. D. ;
Biglan, Kevin M. ;
Shoulson, Ira .
JAMA NEUROLOGY, 2013, 70 (12) :1520-1530