Enrichment of clinical trials in MCI due to AD using markers of amyloid and neurodegeneration

Neurology

Volumn 87, Issue 12, 2016, Pages 1235-1241

  Wolz, Robin ^a,b   Schwarz, Adam J ^c,d,e   Gray, Katherine R ^a,b   Yu, Peng ^c   Hill, Derek L G ^a  

^a IXICO PLC   (United Kingdom)

^b IMPERIAL COLLEGE LONDON   (United Kingdom)

^c ELI LILLY AND COMPANY   (United States)

^d INDIANA UNIVERSITY   (United States)

^e INDIANA UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

AMYLOID BETA PROTEIN[1-42]; APOLIPOPROTEIN E4; FLORBETAPIR F 18; AMYLOID BETA PROTEIN; BIOLOGICAL MARKER;

AGED; ALZHEIMER DISEASE; ALZHEIMER DISEASE ASSESSMENT SCALE; AMNESIA; ARTICLE; BRAIN SIZE; CEREBROSPINAL FLUID ANALYSIS; CLINICAL DEMENTIA RATING; CLINICAL TRIAL (TOPIC); CONTROLLED STUDY; DISEASE COURSE; DISEASE MARKER; FEMALE; FINANCIAL MANAGEMENT; FUNCTIONAL ASSESSMENT INVENTORY; HUMAN; MAJOR CLINICAL STUDY; MALE; METHODOLOGY; MILD COGNITIVE IMPAIRMENT; MINI MENTAL STATE EXAMINATION; NERVE DEGENERATION; NUCLEAR MAGNETIC RESONANCE IMAGING; PATIENT SELECTION; POSITRON EMISSION TOMOGRAPHY; PRIORITY JOURNAL; REY AUDITORY VERBAL LEARNING TEST; SAMPLE SIZE; STATISTICAL ANALYSIS; BRAIN; CEREBROSPINAL FLUID; COGNITIVE DEFECT; COHORT ANALYSIS; DIAGNOSTIC IMAGING; ECONOMICS; MENTAL HEALTH; METABOLISM; PROCEDURES;

AGED; ALZHEIMER DISEASE; AMYLOID BETA-PEPTIDES; BIOMARKERS; BRAIN; CLINICAL TRIALS AS TOPIC; COGNITIVE DYSFUNCTION; COHORT STUDIES; FEMALE; HUMANS; MALE; MENTAL STATUS SCHEDULE; NERVE DEGENERATION; POSITRON-EMISSION TOMOGRAPHY;

EID: 84988457245     PISSN: 00283878     EISSN: 1526632X     Source Type: Journal    
DOI: 10.1212/WNL.0000000000003126     Document Type: Article

References (37)

1. Jack CR Jr, Knopman DS, Jagust WJ, et al. Hypothetical model of dynamic biomarkers of the Alzheimer's pathological cascade. Lancet Neurol 2010;27:685-691.
2. Vinters H. Emerging concepts in Alzheimer's disease. Annu Rev Pathol 2015;10:291-319.
3. Beach T, Schneider J, Sue L, et al. Theoretical impact of florbetapir (18F) amyloid imaging on diagnosis of Alzheimer dementia and detection of preclinical cortical amyloid. J Neuropathol Exp Neurol 2014;73:948-953.
4. McKhann GM, Knopman DS, Chertkow H, et al. The diagnosis of dementia due to Alzheimer's disease: recommendations from the National Institute on Aging and the Alzheimer's Association workgroups on diagnostic guidelines for Alzheimer's disease. Alzheimers Dement 2011;7:263-269.
5. Dubois B, Feldman HH, Jacova C, et al. Advancing research diagnostic criteria for Alzheimer's disease: the IWG-2 criteria. Lancet Neurol 2014;13:614-629.
6. Albert MS, DeKosky ST, Dickson D, et al. The diagnosis of mild cognitive impairment due to Alzheimer's disease: recommendations from the National Institute on Aging-Alzheimer's Association workgroups on diagnostic guidelines for Alzheimer's disease. Alzheimers Dement 2011;7:270-279.
7. Sperling RA, Aisen PS, Beckett LA, et al. Toward defining the preclinical stages of Alzheimer's disease: recommendations from the National Institute on Aging-Alzheimer's Association workgroups on diagnostic guidelines for Alzheimer's disease. Alzheimers Dement 2011;7:280-292.
8. Wharton S, Brayne C, Savva G, et al. Epidemiological neuropathology: the MRC Cognitive Function and Aging Study experience. J Alzheimers Dis 2011;25:359-372.
9. Beach T, Monsell S, Phillips L, Kukull W. Accuracy of the clinical diagnosis of Alzheimer disease at National Institute on Aging Alzheimer Disease Centers, 2005-2010. J Neuropathol Exp Neurol 2012;71:266-273.
10. Jack CR Jr, Wiste HJ, Knopman DS, et al. Rates of b-amyloid accumulation are independent of hippocampal neurodegeneration. Neurology 2014;82:1605-1612.
11. Vellas B, Carrillo MC, Sampaio C, et al; EU/US/CTAD Task Force Members. Designing drug trials for Alzheimer's disease: what we have learned from the release of the phase III antibody trials: a report from the EU/US/CTAD Task Force. Alzheimers Dement 2013;9:438-444.
12. Karran E, Hardy J. A critique of the drug discovery and phase 3 clinical programmes targeting the amyloid hypothesis for Alzheimer's disease. Ann Neurol 2014; 76:185-205.
13. Hampel H, Frank R, Broich K, et al. Biomarkers for Alzheimer's disease: academic, industry and regulatory perspectives. Nat Rev Drug Discov 2010;9:560-574.
14. FDA. Guidance for Industry Alzheimer's Disease: Developing Drugs for the Treatment of Early Stage Disease. Washington, DC: US Food and Drug Administration; 2012.
15. Lasser R, Ostrowitzki S, Scheltens P, et al. Efficacy and safety of gantenerumab from the phase 3 SCarlet RoAD trial, a study of gantenerumab in patients with prodromal AD. Paper presented at: CTAD, November 5-7, 2015; Barcelona, Spain.
16. Sevigny J, Chiao P, Williams L, et al. Randomized, placebocontrolled, phase 1B study of the anti-beta-amyloid antibody aducanumab (BIIB037) in patients with prodromal or mild Alzheimer's disease: interim results. Presented at: CTAD, November 5-7, 2015; Barcelona, Spain.
17. Vos S, van Rossum I, Verhey F, et al. Prediction of Alzheimer disease in subjects with amnestic and nonamnestic MCI. Neurology 2013;80:1124-1132.
18. Samtani M, Raghavan N, Novak G, Nandy P, Narayan V. Disease progression model for Clinical Dementia Rating-Sum of Boxes in mild cognitive impairment and Alzheimer's subjects from the Alzheimer's Disease Neuroimaging Initiative. Neuropsychiatr Dis Treat 2014;10:929-952.
19. Yu P, Sun J, Wolz R, et al. Operationalizing hippocampal volume as an enrichment biomarker for amnestic mild cognitive impairment trials: effect of algorithm, test-retest variability, and cut point on trial cost, duration, and sample size. Neurobiol Aging 2014;35:808-818.
20. Lorenzi M, Donohue M, Paternicò D, et al. Enrichment through biomarkers in clinical trials of Alzheimer's drugs in patients with mild cognitive impairment. Neurobiol Aging 2010;31:1443-1451.
21. Kohannim O, Hua X, Hibar DP, et al. Boosting power for clinical trials using classifiers based on multiple biomarkers. Neurobiol Aging 2010;31:1429-1442.
22. Yu P, Dean RA, Hall SD, et al. Enriching amnestic mild cognitive impairment populations for clinical trials: optimal combination of biomarkers to predict conversion to dementia. J Alzheimers Dis 2012;32:373-385.
23. van Rossum IA, Vos SJ, Burns L, et al. Injury markers predict time to dementia in subjects with MCI and amyloid pathology. Neurology 2012;79:1809-1816.
24. Holland D, McEvoy L, Desikan R, Dale A. Enrichment and stratification for predementia Alzheimer disease clinical trials. PLoS One 2012;7:e47739.
25. Macklin EA, Blacker D, Hyman BT, Betensky RA. Improved design of prodromal Alzheimer's disease trials through cohort enrichment and surrogate endpoints. J Alzheimers Dis 2013;36:475-486.
26. Hill D, Schwarz A, Isaac M, et al. CAMD/EMA biomarker qualification of hippocampal volume for enrichment of clinical trials in pre-dementia stages of Alzheimer's disease. Alzheimers Dement 2014;10:421-429.
27. Delor I, Charoin JE, Gieschke JE, Retout S, Jacqmin P. Modeling Alzheimer's disease progression using disease onset time and disease trajectory concepts applied to CDR-SOB scores from ADNI. CPT Pharmacometrics Syst Pharmacol 2013;2:e78.
28. Jack CR, Knopman DS, Weigand SD, et al. An operational approach to NIA-AA criteria for preclinical Alzheimer's disease. Ann Neurol 2012;71:765-775.
29. Shaw L, Vanderstichele H, Knapik-Czajka M, et al. Cerebrospinal fluid biomarker signature in Alzheimer's Disease Neuroimaging Initiative subjects. Ann Neurol 2009;65:403-413.
30. Weigand S, Vemuri P, Wiste H, et al. Transforming CSF Ab42 measures into calculated Pittsburgh Compound B (PIBcalc) units of brain Ab amyloid. Alzheimers Dement 2011;7:133-141.
31. Mueller S, Weiner M, Thal L, et al. The Alzheimer's Disease Neuroimaging Initiative. Neuroimaging Clin N Am 2005;15:869-877.
32. Wolz R, Schwarz AJ, Yu P, et al. Robustness of automated hippocampal volumetry across magnetic resonance field strengths and repeat images. Alzheimers Dement 2014; 10:430-438.
33. Blennow K, Mattsson N, Schoell M, Hansson O, Zetterberg H. Amyloid biomarkers in Alzheimer's disease. Trends Pharmacol Sci 2015;36:297-309.
34. Wolz R, Aljabar P, Hajnal J, Hammers A, Rueckert D. LEAP: learning embeddings for atlas propagation. Neuroimage 2010;49:1316-1325.
35. Ittner LM, Götz J. Amyloid-b and tau: a toxic pas de deux in Alzheimer's disease. Nat Rev Neurosci 2011;12: 67-72.
36. Palmqvist S, Zetterberg H, Mattsson N, et al. Detailed comparison of amyloid PET and CSF biomarkers for identifying early Alzheimer disease. Neurology 2015; 85:1240-1249.
37. Wessels A, Siemers E, Yu P, et al. A combined measure of cognition and function for clinical trials: the integrated Alzheimer's disease rating scale (iADRS). J Prev Alzheimers Dis 2015;2:227-241.