Clinical trials of antitumor agents: Experimental design and timeline considerations

Cancer Journal from Scientific American

Volumn 3, Issue 3, 1997, Pages 127-136

  Frei III, Emil ^a  

^a DANA FARBER CANCER INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANTINEOPLASTIC AGENT; BRYOSTATIN; FUMAGILLOL CHLOROACETYLCARBAMATE; NEW DRUG; RETINOIC ACID;

BONE MARROW TOXICITY; CANCER RESEARCH; CEREBELLAR ATAXIA; CLINICAL ARTICLE; CLINICAL TRIAL; DRUG RESEARCH; DRUG SAFETY; FOOD AND DRUG ADMINISTRATION; HUMAN; MAXIMUM ALLOWABLE CONCENTRATION; MYALGIA; NEUROTOXICITY; PHASE 1 CLINICAL TRIAL; PRIORITY JOURNAL; REVIEW;

ANTINEOPLASTIC AGENTS; CLINICAL TRIALS; HUMANS; RESEARCH DESIGN; TIME FACTORS;

EID: 0030921468     PISSN: 10814442     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Review

References (61)

1. DiMasi JA. A new look at United States drug development and approval times. American Journal of Therapeutics 1996;3:1-11.
2. Kaetin KI. Pharmaceutical innovation in an era of reform. American Journal of Therapeutics 1995;2:7630-7634.
3. National Committee to Review Current Procedures for Approval of New Drugs for Cancer and AIDS. Final Report 1990. Washington, DC.
4. Lasagna L, Frei E III. Report and recommendations resulting from the Washington Clinical Trials Committee. Am J Clin Oncol; in press.
5. Kessler D. The FDA, AIDs, and cancer. Scientific American 1995;264:134-141.
6. Clinton W. New drugs for cancer and AIDS. New York Times, April 4, 1996.
7. Gibbs JB, Oliff A. Pharmaceutical research in molecular oncology. Cell 1994;79:193-198.
8. Schipper H, Goh CR, Wang TL. Shifting the cancer paradigm: must we kill to cure? J Clin Oncol 1995;13:901-905.
9. Weinberg RA. How cancer arises. Scientific American 1996; 275:62-71.
10. Toppmeyer DL. Phase I trial design and methodology. In Teicher BA, ed. Anticancer Drug Development. Totowa, NJ: Humana Press, 1997:227-247.
11. Collins JM, Grieshaber CK, Chabner BA. Pharmacologically guided phase I clinical trial based upon preclinical drug development. J Natl Cancer Inst 1990;82:1321-1326.
12. Carter SK, Selawry O, Slavik M. Phase I clinical trials. Monogr Natl Cancer Inst 1977;45:75-80.
13. Rozencweig M, Dodion P, Nicaise C et al. Approach to phase I trials in cancer patients. In: Cortes-Funes H, Rozencweig M, eds. New Approaches in Cancer Therapy. New York: Raven Press, 1982:1-13.
14. Christian MC, Korn EL. The limited precision of clinical trials. J Natl Cancer Inst 1994;86:1662-1668.
15. Daugherty C, Ratain MJ, Grochowski E et al. Perceptions of cancer patients and their physicians involved in phase I trials. J Clin Oncol 1995;13:1062-1072.
16. Daugherty C, Ratain MJ, Mick R. A proposal for a new phase I clinical trial design: patient choice dose cohort. Clin Res 1993;41:52-59.
17. Decoster G, Stein G, Holdener EE. Responses and toxic deaths in phase I clinical trials. Ann Oncol 1994;2:175-181.
18. Emanuel EJ. A phase I trial on the ethics of phase I trials. J Clin Oncol 1995;13:1049-1051.
19. Estey E, Hoth D, Simon R et al. Therapeutic response in phase I trials of antineoplastic agents. Cancer Treatment Reports 1986;70: 1105-1115.
20. Von Hoff DD, Turner J. Response rates, duration of response, and dose response effects in phase I studies of antineoplastics. Invest New Drugs 1991;9:115-122.
21. Slavin ML, Stubbe L, Plant HJ et al. Attitudes to chemotherapy: comparing views of patients with cancer with those of doctors, nurses and the general public. BMJ 1990;300:1458.
22. Zelen M. The theory and practice of clinical trials. In: Holland JF, Frei E III, Bast RC Jr. et al, eds. Cancer Medicine (3rd ed). Philadelphia: Williams & Wilkins, 1993:340-361.
23. Hansen HH, Selawy OS, Muggia FM et al. Clinical studies with 1-(2-chloroethyl)-3-cyclohexyl-1-nitrosourea (CCNU) (NSC 79037). Cancer Res 1971;31:223-227.
24. Ratain MJ, Schilsky RL, Conley BA et al. Pharmacodynamics in cancer therapy. J Clin Oncol 1990;8:1739-1753.
25. Bernal SD, Lampidis PJ, McIsaac RM et al. Anticarcinoma activity in vivo of rhodamine 123, a mitochondrial specific dye. Science 1983;222:169,172.
26. Chen LB. Mitochondrial potential in living cells. Annual Review of Cell Biology 1988;4:155-185.
27. Ratain MJ, Mick R, Schilsky RL et al. Statistical and ethical issues in the design and conduct of phase I and II clinical trials of new anticancer agents. J Natl Cancer Inst 1993;85:1637-1643.
28. O'Quigley J, Pepe M, Fisher L. Continual reassessment method: a practical design for phase I clinical trials in cancer. Biometrics 1990; 46:33-48.
29. Berry DA. A case for Bayesian use in clinical trials. Stat Med 1993; 12:1377-1393.
30. European Organization for the Research and Treatment of Cancer. Pharmacokinetically guided dose escalation in phase I clinical trials: commentary and proposed guidelines. Eur J Cancer 1987; 23:1083-1087.
31. Faries D. Practical modifications of the continual reassessment method for phase I cancer clinical trials. Journal of Biopharmaceutical Statistics 1994;4:147-164.
32. Gatsonis C, Greenhouse JB. Bayesian methods for phase I clinical trials. Stat Med 1992;11:1377-1389.
33. Graham MA, Workman P. The impact of pharmacokinetically guided dose escalation strategies in phase I clinical trials: critical evaluation and recommendations for future studies. Ann Oncol 1992;3:339-347.
34. Lipsett MB. On the nature and ethics of phase I clinical trials. JAMA 1972;248:941-942.
35. Penta JS, Rosner GL, Trump DL. Choice of starting dose and escalation for phase I studies of antitumor agents. Cancer Chemother Pharmacol 1992;31:247-250.
36. Storer BE. Design and analysis of phase I clinical trials. Biometrics 1989;45:925-937.
37. Thall PF, Estey EH. A Bayesian strategy for screening cancer treatments prior to phase II clinical evaluation. Stat Med 1993;12: 1197-1211.
38. Berry DA, Thor A, Cirrincione C et al. Scientific inference and predictions: multiplicities and convincing stories: a case study in breast cancer therapy. In: Bernardo JM, ed. Bayesian Statistics #5. New York: Clarendon, 1996.
39. Korn EL, Midthune D, Chen TT et al. A comparison of two phase I trial designs. Stat Med 1994;13:1799-1806.
40. Mick R, Retain M. Model guided determination of maximum tolerated dose in phase I clinical trials: evidence for increased precision. J Natl Cancer Inst 1993;85:217-223.
41. Newell DR. Pharmacologically based phase I trials in cancer chemotherapy. Hematol Oncol Clin North Am 1994;8:257-275.
42. Von Hoff DD, Kuhn J, Clark GM. Cancer clinical trials: methods and practice. In: Buyse S, Sylvester RJ, eds. Design and Conduct of Phase I Trials. New York: Oxford University Press, 1984: 210-220.
43. Freireich EJ, Gehan EA, Rall DP et al. Quantitative comparison of toxicity of anticancer agents in mouse, rat, hamster, dog, monkey, and man. Cancer Chemotherapy Report 1966;50:219-244.
44. Goldsmith MA, Slavik M, Carter SK. Quantitative prediction of toxicity in humans from toxicology in small and large animals. Cancer Res 1975;35:1354-1364.
45. Homan ER. Quantitative relationships between toxic doses of antitumor chemotherapeutic agents in animals and man. Cancer Chemotherapy Report 1972;3:13-19.
46. Pinkel D. The use of body surface area as a criteria of drug dosage in cancer chemotherapy. Cancer Res 1956;18:835-856.
47. Grieshaber CK, Marsoni S. Relation of preclinical toxicology to findings in early clinical trials. Cancer Treatment Reports 1986; 70:65-72.
48. Niedert JA. Dose intensive therapy without progenitor cell replacement. In: Armitage JO, Antman KH, eds. High Dose Cancer Therapy (2nd ed). Philadelphia: Williams & Wilkins, 1995: 226-242.
49. Collins JM, Zaharko DS, Dedrick RL et al. Potential roles for preclinical pharmacology in phase I clinical trials. Cancer Treatment Reports 1986;70:73-80.
50. O'Quigley J. Estimating the probability of toxicity at the recommended dose following a phase I clinical trial in cancer. Biometrics 1992;48:853-862.
51. Penta JS, Rozenscweig M, Guarino AM. Mouse and large-animal toxicology studies of twelve antitumor agents: relevance to starting dose for phase I clinical trials. Cancer Chemother Pharmacol 1979; 3:97-101.
52. Berdel WE, Knopf H, Fromm M et al. Influence of phase I early clinical trials on quality of life in cancer patients. Anticancer Res 1988;8:313-322.
53. Tomamichell M, Sessa C, Herzig S et al. Informed consent for phase I studies: evaluation of quantity and quality of information provided to patients. Ann Oncol 1995;6:363-369.
54. Simon R, Wittes R, Ellenberg SS. Randomized phase II clinical trials. Cancer Treatment Reports 1985;69:1375.
55. Wittes RE, Marsoni S, Simon R et al. The phase II trial. Cancer Treatment Reports 1985;69:1235.
56. Geller NL. Design of phase I and II clinical trials in cancer: a statistician's view. Cancer Invest 1992;2:483-491.
57. Khater C, Laub P, Gallo JM et al. Phase II clinical trials in oncology, in press.
58. Myers C. Anthracyclines and DNA intercalators. In: Holland JF, Frei E III, Bast RC Jr. et al, eds. Cancer Medicine (4th ed). Philadelphia: Williams & Wilkins, 1996.
59. Constanza ME, Korzan AH, Henderson IC et al. Amonofide, an active agent in metastatic breast cancer. Proceedings of ASCO 1990;9:91.
60. Dawson NA, Costanza ME, Korzun AH et al. Trimetrexate in untreated and previously treated patients with metastatic breast cancer: a Cancer and Leukemia Group B study. Med Pediatr Oncol 1991;19:283-288.
61. Frei E III. Curative cancer chemotherapy. Cancer Res 1985;45: 6523-7517.