Clinical trials in the neurosciences 15 tables

237 Pages·3.115 MB·English

by  Katherine M Woodbury-Harris

Clinical Trials in the Neurosciences
Frontiers of Neurology and
Neuroscience
Vol. 25
Series Editor
J. Bogousslavsky
Montreux
Clinical Trials in the
Neurosciences
Volume Editors
K.M. Woodbury-Harris
Redmond, Oreg.
B.M. Coull
Tucson, Ariz.
11 figures and 15 tables, 2009
Basel · Freiburg · Paris · London · New York · Bangalore ·
Bangkok · Shanghai · Singapore · Tokyo · Sydney
Dr. Katherine M. Woodbury-Harris Prof. Bruce M. Coull
Consultant in Clinical Trials University of Arizona
and Neuroscience Health Science Center
1936 Turnstone Road Department of Neurology
Redmond, OR 97756 (USA) 1501 N. Campbell Avenue
Tucson, AZ 85724 (USA)
Library of Congress Cataloging-in-Publication Data
Clinical trials in the neurosciences / volume editors, K.M. Woodbury-Harris,
B.M. Coull.
p. ; cm. -- (Frontiers of neurology and neuroscience, ISSN 1660-4431
; v. 25)
Includes bibliographical references and index.
ISBN 978-3-8055-9023-5 (hard cover : alk. paper)
1. Neurology--Research. 2. Neurosciences--Research. 3. Clinical trials.
I. Woodbury-Harris, K. M. (Katherine M.) II. Coull, B. M. (Bruce M.) III.
Series: Frontiers of neurology and neuroscience ; v. 25.
[DNLM: 1. Clinical Trials as Topic. 2. Neurology--methods. 3. Nervous
System Diseases. 4. Neurosurgical Procedures. 5. Research Design. W1
MO568C v.25 2009 / WL 100 C6439 2009]
RC337.C63 2009
616.80072’4--dc22
2009003817
Bibliographic Indices. This publication is listed in bibliographic services, including Current Contents® and Index Medicus.
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editor(s) disclaim responsibility for any injury to persons or property resulting from any ideas, methods, instructions or products
referred to in the content or advertisements.
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text are in accord with current recommendations and practice at the time of publication. However, in view of ongoing research,
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retrieval system, without permission in writing from the publisher.
© Copyright 2009 by S. Karger AG, P.O. Box, CH–4009 Basel (Switzerland)
www.karger.com
Printed in Switzerland on acid-free and non-aging paper (ISO 9706) by Reinhardt Druck, Basel
ISSN 1660–4431
ISBN 978–3–8055–9023–5
e-ISBN 978–3–8055–9024–2
To Henry Barnett who started it all
John Marler who made it happen
The editors would like to thank and acknowledge KAI Research, especially
Selma Kunitz, Kristy Miller, and Patti Shugarts. They would like to
acknowledge the special efforts of Paula Lundquist as well.
Contents
X List of Contributors
1 Introduction
Coull, B.M. (Tucson, Ariz.); Woodbury-Harris, K.M. (Redmond, Oreg.)
4 Evolution of Clinical Trials in Neurology
Stapf, C. (Paris)
Preclinical Trials
Translation
10 Role of Animal Studies in the Design of Clinical Trials
Hall, E.D. (Lexington, Ky.); Traystman, R.J. (Aurora, Colo.)
STAIR
34 Translating Interventions from Ischemic Stroke Models
to Patients: The View in 2009
del Zoppo, G.J. (Seattle, Wash.); Moskowitz, M.A. (Boston, Mass.)
Pharmacology
39 Scaling Up from Animal to Human Studies
Hemmen, T.M. (San Diego, Calif.); Zivin, J. (La Jolla, Calif.)
46 FDA Requirements for Preclinical Studies
McElvany, K.D. (Chapel Hill, N.C.)
Clinical Trials
Pilot Trials
52 Phase I/II – Design and Analysis
Coleman, W.P. (Buffalo, N.Y.); Woodbury-Harris, K.M. (Redmond, Oreg.);
Coull, B.M. (Tucson, Ariz.)
VII
55 Biomarkers in Neurology
Silva, G.; Furie, K. (Boston, Mass.)
The Randomized, Blinded Trial
62 FDA Investigational New Drug Requirements
McElvany, K.D. (Chapel Hill, N.C.)
66 European Union Regulatory Requirements
Radziszewska, B. (Bethesda, Md.)
71 Adaptive Clinical Trials
Gilbert, P.R. (Bethesda, Md.)
76 Design and Analysis Plans
Palesch, Y.Y. (Charleston, S.C.)
78 Behavioral/Neuropsychological Outcomes and Quality of Life Endpoints
Levin, B.E. (Miami, Fla.)
93 Clinical Endpoints
Sonni, S.; Furie, K. (Boston, Mass.)
98 Data Management and Quality Assurance
Richardson, D. (Lake Forest, Ill.); Kunitz, S.C. (Redmond, Oreg.)
Multicentered Trials
106 Multidisciplinary Trial Design
Stapf, C. (Paris)
114 Design and Analysis Issues
Coleman, W.P. (Buffalo, N.Y.)
118 Strategies for Recruiting and Retaining Minorities
DeBaun, M.R. (St. Louis, Mo.); Gorelick, P.B. (Chicago, Ill.); Som, S. (St. Louis, Mo.)
121 Informed Consent and HIPAA
Woodbury-Harris, K.M. (Redmond, Oreg.)
124 Committees for Multicenter Clinical Trials
Adams, H.P., Jr. (Iowa City, Iowa)
127 Interim Monitoring of Clinical Trials
Richardson, D. (Lake Forest, Ill.)
131 Medical Monitoring
Adams, H.P., Jr. (Iowa City, Iowa)
133 Budgets for Clinical Trials
Kelly, L.C.; Hart, R.G. (San Antonio, Tex.)
136 Institutional Review Boards and Ethical Issues in Randomized Clinical Trials
Shinnar, S. (Bronx, N.Y.)
141 Financial Conflict of Interest
Fureman, B.E. (Rockville, Md.)
VIII Contents
Special Populations and Genetic Studies
144 Clinical Trials in Progressive Neurological Diseases.
Recruitment, Enrollment, Retention and Compliance
Bedlack, R.S. (Durham, N.C.); Cudkowicz, M.E. (Charlestown, Mass.)
152 Genetics in Clinical Trials
Meschia, J.F. (Jacksonville, Fla.); Gwinn, K. (Houston, Tex.)
158 Randomized Clinical Trials in Children
Ment, L.R. (New Haven, Conn.); Shinnar, S. (Bronx, N.Y.)
163 Neurological Emergencies
Silbergleit, R.; Barsan, W. (Ann Arbor, Mich.)
174 Clinical Trials of Surgical Devices for Neurological Disorders
Kaplitt, M.G. (New York, N.Y.); Barbaro, N. (San Francisco, Calif.)
180 Gene Therapy Clinical Trials in the Human Brain.
Protocol Development and Review of Current Applications
Kaplitt, M.G. ( New York, N.Y.)
Imaging
189 Magnetic Resonance Imaging for Surrogate Outcomes and Patient Selection
Hsia, A.W.; Kidwell, C.S. ( Washington, D.C.)
193 Computed Tomography
Lima, F.; Goldmakher, G.; Lev, M.; Furie, K. (Boston, Mass.)
197 Positron Emission Tomography Imaging
Dhawan, V.; Eidelberg, D. (Manhasset, N.Y.)
Training and Education
203 Clinical Research Training Opportunities and Elements
Franz, D. (Honolulu, Hawaii); Coull, B.M. (Tucson, Ariz.)
206 Appendix
208 Author Index
209 Subject Index
Contents IX