Optimizing phase II of drug development for disease-modifying compounds

Alzheimer's and Dementia

Volumn 4, Issue 1 SUPPL. 1, 2008, Pages

  Cummings, Jeffrey L ^a  

^a UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

Adaptive design; Alzheimer's disease; Drug development; Neuropsychological test battery; Phase II; Phase III

Indexed keywords

ALZHEIMER DISEASE VACCINE; AMYLOID; AMYLOID ANTIBODY; AMYLOID BETA PROTEIN; BETA SECRETASE; BETA SECRETASE INHIBITOR; BIOLOGICAL MARKER; FLUORODEOXYGLUCOSE; NOOTROPIC AGENT; PLACEBO; PROTEIN ANTIBODY; ROSIGLITAZONE; TAU PROTEIN; UNCLASSIFIED DRUG;

ALZHEIMER DISEASE; BRAIN ATROPHY; CLINICAL RESEARCH; CLINICAL TRIAL; COGNITIVE DEFECT; DEMENTIA; DISEASE SEVERITY; DOSE RESPONSE; DRUG INDUSTRY; DRUG MARKETING; DRUG RESEARCH; DRUG SYNTHESIS; DRUG TREATMENT FAILURE; EDITORIAL; ELECTROENCEPHALOGRAM; HUMAN; MINI MENTAL STATE EXAMINATION; NONHUMAN; NUCLEAR MAGNETIC RESONANCE IMAGING; OPTIMAL DRUG DOSE; POSITRON EMISSION TOMOGRAPHY; PRIORITY JOURNAL; PROTEIN CEREBROSPINAL FLUID LEVEL; PROTEIN PHOSPHORYLATION; PROTEIN TARGETING; RISK FACTOR; SENSITIVITY ANALYSIS; TREATMENT OUTCOME; VALIDATION PROCESS;

ALZHEIMER DISEASE; BIOLOGICAL MARKERS; CLINICAL TRIALS, PHASE II AS TOPIC; HUMANS; NEUROPSYCHOLOGICAL TESTS; RESEARCH DESIGN;

EID: 38049061575     PISSN: 15525260     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.jalz.2007.10.002     Document Type: Editorial

References (32)

1. Easdale C., and Vose C. Clinical development. In: Rang H. (Ed). Drug discovery and development. 1st ed. (2006), Churchill Livingstone Elsevier, London 255-271
2. Rang H. Drug discovery and development: facts and figures. In: Rang H. (Ed). Drug discovery and development (2006), Churchill Livingstone Elsevier, London 311-327 1st ed.
3. Cummings J.L. Challenges to demonstrating disease-modifying effects in Alzheimer's disease clinical trial. Alzheimer's & Dementia 2 (2006) 263-271
4. Cummings J.L. Alzheimer's disease. N Engl J Med 351 (2004) 56-67
5. Mori E., Hashimoto M., Krishnan K.R., and Doraiswamy P.M. What constitutes clinical evidence for neuroprotection in Alzheimer disease: support for the cholinesterase inhibitors?. Alzheimer Dis Assoc Disord 20 (2006) S19-S26
6. Cummings J., Doody M., and Clark C. Disease-modifying therapies for Alzheimer disease. Neurology (2007) 69
7. Mohs R.C., Knopman D., Petersen R.C., Ferris S.H., Ernesto C., and Grundman M. Development of cognitive instruments for use in clinical trials of antidementia drugs: additions to the Alzheimer's Disease Assessment Scale that broaden its scope-the Alzheimer's Disease Cooperative Study. Alzheimer Dis Assoc Disord 11 Suppl 2 (1997) S13-S21
8. Petersen R.C., Thomas R.G., Grundman M., Bennett D., Doody R., Ferris S., et al. Vitamin E and donepezil for the treatment of mild cognitive impairment. N Engl J Med 352 (2005) 2379-2388
9. Sano M., Ernesto C., Thomas R.G., Klauber M.R., Schafer K., Grundman M., et al. A controlled trial of selegiline, alpha-tocopherol, or both as treatment for Alzheimer's disease: the Alzheimer's Disease Cooperative Study. N Engl J Med 336 (1997) 1216-1222
10. Jack C.R., Slomkowski M., Gracon S., Hoover T.M., Felmlee J.P., Stewart K., et al. MRI as a biomarker of disease progression in a therapeutic trial of milameline for AD. Neurology 60 (2003) 253-260
11. Mueller S.G., Weiner M.W., Thal L.J., Petersen R.C., Jack C.R., Jagust W., et al. Ways toward an early diagnosis in Alzheimer's disease: the Alzheimer's Disease Neuroimaging Initiative (ADNI). Alzheimers & Dementia 1 (2005) 55-66
12. Li G., Sokal I., Quinn J.F., Leverenz J.B., Brodey M., Schellenberg G.D., et al. CSF tau/Abeta42 ratio for increased risk of mild cognitive impairment: a follow-up study. Neurology 7 (2007) 631-639
13. Brys M., Pirraglia E., Rich K., Rolstad S., Mosconi L., Switalski R., et al. Prediction and longitudinal study of CSF biomarkers in mild cognitive impairment. Neurobiol Aging (2007) Sep 21;[Epub ahead of print].
14. De Felice F.G., Vieira M.N., Saraiva L.M., Figueroa-Villar J.D., Garcia-Abreu J., Liu R., et al. Targeting the neurotoxic species in Alzheimer's disease: inhibitors of Abeta oligomerization. FASEB J 18 (2004) 1366-1372
15. Ghosh A.K., Bilcer G., Hong L., Koelsch G., and Tang J. Memapsin 2 (beta-secretase) inhibitor drug, between fantasy and reality. Curr Alzheimer Res 4 (2007) 418-422
16. Zhong Z., Ewers M., Teipel S., Bürger K., Wallin A., Blennow K., et al. Levels of beta-secretase (BACE1) in cerebrospinal fluid as a predictor of risk in mild cognitive impairmen. Arch Gen Psychiatry 6 (2007) 718-726
17. Klunk W.E., Engler H., Nordberg A., Wang Y., Blomqvist G., Holt D.P., et al. Imaging brain amyloid in Alzheimer's disease with Pittsburgh Compound-B. Ann Neurol 55 (2004) 306-319
18. Small G.W., Kepe V., Ercoli L.M., Siddarth P., Bookheimer S.Y., Miller K.J., et al. PET of brain amyloid and tau in mild cognitive impairment. N Engl J Med 355 (2006) 2652-2663
19. Lockhart A., Lamb J.R., Osredkar T., Sue L.I., Joyce J.N., Ye L., et al. PIB is a non-specific imaging marker of amyloid-beta (A{beta}) peptide-related cerebral amyloidosis. Brain 130 (2007) 2607-2615
20. Edison P., Archer H.A., Hinz R., Hammers A., Pavese N., Tai Y.F., et al. Amyloid, hypometabolism, and cognition in Alzheimer disease: an [11C]PIB and [18F]FDG PET study. Neurology 7 (2007) 501-508
21. Mega M.S., Dinov I.D., Porter V., Chow G., Reback E., Davoodi P., et al. Metabolic patterns associated with the clinical response to galantamine therapy: a fludeoxyglucose f 18 positron emission tomographic study. Arch Neurol 5 (2005) 721-728
22. Diamond E.L., Miller S., Dickerson B.C., Atri A., DePeau K., Fenstermacher E., et al. Relationship of fMRI activation to clinical trial memory measures in Alzheimer disease. Neurology 69 (2007) 1331-1341
23. Walsh D., and Selkoe D. A beta oligomers: a decade of discovery. J Neurochem 5 (2007) 1172-1184
24. Banks M., Cacace A., O'Connell J., and Houston J. High-throughput screening: evolution of technology and methods. In: Gad S. (Ed). Drug discovery handbook (2005), Wiley-Interscience, Hoboken, NJ 559-602
25. Hsueh W. Genetic discoveries as the basis of personalized therapy: rosiglitazone treatment of Alzheimer's disease. Pharmacogenomics J 6 (2006) 222-224
26. Gilman S., Koller M., Black R.S., Jenkins L., Griffith S.G., Fox N.C., et al. Clinical effects of Abeta immunization (AN1792) in patients with AD in an interrupted trial. Neurology 9 (2005) 1553-1562
27. Harrison J., Minassian S.L., Jenkins L., Black R.S., Koller M., and Grundman M. A neuropsychological test battery for use in Alzheimer disease clinical trials. Arch Neurol 64 (2007) 1323-1329
28. Dwolatzky T., Whitehead V., Doniger G.M., Simon E.S., Schweiger A., Jaffe D., et al. Validity of the Mindstreams computerized cognitive battery for mild cognitive impairment. J Mol Neurosci 24 (2004) 33-44
29. Kelly P.J., Stallard N., and Todd S. An adaptive group sequential sesign for Phase II/III clinical trials that select a single treatment from several. J Biopharm Stat 15 (2005) 641-658
30. Gould L. How practical are adaptive esigns likely to be for confirmatory trials. Biometrical J 48 (2006) 644-649
31. Lokhnygina Y. Discussion of the "Executive Summary of the PhRMA Working Group on Adaptive Designs in Clinical Drug Development". J Biopharm Stat 16 (2006) 287-288
32. Hung H.M.J., O'Neill R.T., Wang S.-J., and Lawrence J. A regulatory view on adaptive/flexible clinical trial design. Biometrical J 48 (2006) 565-573