Dose-levels and first signs of efficacy in contemporary oncology phase 1 clinical trials

PLoS ONE

Volumn 6, Issue 3, 2011, Pages

  Ferte, Charles ^a,b   Soria, Jean Charles ^b   Penel, Nicolas ^a,c  

^a CENTRE OSCAR LAMBRET   (France)

^b INSTITUT GUSTAVE ROUSSY   (France)

^c UNIV LILLE   (France)

Author keywords

[No Author keywords available]

Indexed keywords

CYTOTOXIC AGENT;

CANCER COMBINATION CHEMOTHERAPY; CANCER THERAPY; DOSE RESPONSE; DRUG ACTIVITY; DRUG DOSE REGIMEN; DRUG EFFICACY; MAXIMUM TOLERATED DOSE; MOLECULARLY TARGETED THERAPY; MONOTHERAPY; PHASE 1 CLINICAL TRIAL (TOPIC); REVIEW; ARTICLE; HUMAN; META ANALYSIS; TREATMENT OUTCOME;

CLINICAL TRIALS, PHASE I AS TOPIC; DOSE-RESPONSE RELATIONSHIP, DRUG; HUMANS; MAXIMUM TOLERATED DOSE; TREATMENT OUTCOME;

EID: 79952328883     PISSN: None     EISSN: 19326203     Source Type: Journal    
DOI: 10.1371/journal.pone.0016633     Document Type: Review

References (29)

1. Von Hoff DD, Turner J, (1991) Response rates, duration of response and dose response effects in phase I studies. Invest New Drugs 9: 115-21.
2. Itoh K, Sasaki Y, Miyata Y, Fujii H, Ohtsu T, et al. (1994) Therapeutic response and potential pitfalls in phase I clinical trials of anticancer agents conducted in Japan. Cancer Chemother Pharmacol 34 (6): 451-4.
3. Grunwald HW, (2007) Ethical and design issues of phase 1 clinical trials in cancer patients. Cancer Invest 25: 124-6.
4. Korn EL, Arbuck SG, Pluda JM, Simon R, Kaplan RS, et al. (2001) Clinical trial designs for cytostatic agents: are new approaches needed? J Clin Oncol 19 (1): 265-72.
5. Parulekar WR, Eisenhauer EA, (2004) Phase I trial design for solid tumor studies of targeted, non-cytotoxic agents: theory and practice. J Natl Cancer Inst 96 (13): 990-7.
6. Postel-Vinay S, Arkenau HT, Olmos D, et al. (2009) Clinical benefit in Phase-I trials of novel molecularly targeted agents: does dose matter? Br J Cancer 100 (9): 1373-8.
7. Jain RK, Lee JJ, Hong D, et al. (2010) Phase I oncology studies: evidence that in the era of targeted therapies patients on lower doses do not fare worse. Clin Cancer Res 16 (4): 1289-97.
8. Penta JS, Rozencweig M, Guarino AM, Muggia FM, (1979) Mouse and large-animal toxicology studies of twelve antitumor agents: relevance of starting dose for phase I clinical trials. Cancer Chemother Pharmacol 31: 247-50.
9. Penta JS, Rosner GL, Trump DL, (1992) Choice of the starting dose and escalation for phase I clinical studies of antitumour agents. Cancer Chemother Pharmacol 31: 247-50.
10. Kumar R, Knick VB, Rudolph SK, Johnson JH, Crosby RM, et al. (2007) Pharmacokinetic-pharmacodynamic correlation from mouse to human with pazopanib, a multikinase angiogenesis inhibitor with potent antitumor and antiangiogenic activity. Mol Cancer Ther 6: 2012-21.
11. Penel N, Leblond P, Lansiaux A, Clisant S, Dansin E, et al. (2010) Justification of the starting dose as the main determinant of accrual time in dose-seeking oncology phase 1 trials. Invest New Drugs 28 (6): 839-43.
12. Kerr DJ, (1994) Phase I clinical trials: adapting methodology to face new challenges. Ann Oncol 5 (Suppl 4): 67-70.
13. Dent SF, Eisenhauer EA, (1996) Phase I trial design: are new methodologies begin put into practice? Ann Oncol 7: 561-66.
14. Simon R, Friedlin B, Rubinstein L, Arbuck SG, Collins J, et al. (1997) Accelerated titration designs for phase 1 clinical trials in oncology. J Natl Cancer Inst 89: 1138-47.
15. Wadman M, (2006) London's disastrous drug trial has serious side effects for research. Nature 440 (7083): 388-9.
16. Stupp R, Bauer J, Pagani O, Gerard B, Cerny T, et al. (1998) Ventricular arrythmia and torsade de points: dose limiting toxicities of the DR-modulator S9788 in a phase 1 trial. Ann Oncol 9: 1233-42.
17. Ryan QC, Headlee D, Acharya M, Sparreboom A, Trepel JB, et al. (2005) Phase I and pharmacokinetic study of MS-275, a histone deacetylase inhibitor, in patients with advanced and refractory solid tumors or lymphoma. J Clin Oncol 23: 3912-22.
18. Azad NS, Posadas EM, Kwitkowski VE, Steinberg SM, Jain L, et al. (2008) Combination targeted therapy with sorafenib and bevacizumab results in enhanced toxicity and antitumor activity. J Clin Oncol 26: 3709-14.
19. George S, Blay JY, Casali PG, Le Cesne A, Stephenson P, et al. (2009) Clinical evaluation of continuously dosing of sunitinib malate in patients with advanced gastrointestinal stromal tumor after imatinib failure. Eur J Cancer 45: 1959-68.
20. Escudier B, Szczylik C, Hutson TE, Demkow T, Staehler M, et al. (2009) Randomized phase II trial of first-line treatment with sorafenib versus interferon alfa-2a in patients with metastatic renal cell carcinoma. J Clin Oncol 27: 1280-9.
21. Debiec-Rychter M, Sciot R, Le Cesne A, Schlemmer M, Hohenberger P, et al. (2006) EORTC Soft Tissue and Bone Sarcoma Group; Italian Sarcoma Group; Australasian GastroIntestinal Trials Group. KIT mutations and dose selection for imatinib in patients with advanced gastrointestinal stromal tumours. Eur J Cancer 42 (8): 1093-103.
22. Booth CM, Calvert AH, Giaccone G, Lobbezoo MW, Seymour LK, et al. (2008) Endpoints and other considerations in phase I studies of targeted anticancer therapy: recommendations from the task force on Methodology for the Development of Innovative Cancer Therapies (MDICT). Eur J Cancer 44 (1): 19-24.
23. Newsome BW, Ernstoff MS, (2008) The clinical pharmacology of therapeutic monoclonal antibodies in the treatment of malignancy; have the magic bullets arrived? Br J Clin Pharmacol 66 (1): 6-19.
24. Liu G, Dou S, Liang M, Chen X, Rusckowski M, et al. (2009) The ratio of maximum percent accumulation of the pretargeting agent and the radiolabelled effector is dependent of tumor size. Eur J Cancer 45: 3098-103.
25. Karaman MW, Herrgard S, Treiber DK, Gallant P, Atteridge CE, et al. (2008) A quantitative analysis of kinase inhibitor selectivity. Nat Biotechnol 26 (1): 127-32.
26. Gelmon KA, Eisenhauer EA, Harris AL, Ratain MJ, Workman P, (1999) Anticancer agents targeting signaling molecules and cancer cell environment: challenges for drug development? J Natl Cancer Inst 15: 1281-87.
27. Le Tourneau C, Lee JJ, Siu LL, (2009) Dose escalation methods in phase I cancer clinical trials. J Natl Cancer Inst 101 (10): 708-20.
28. Horstmann E, McCabe MS, Grochow L, Yamamoto S, Rubinstein L, et al. (2005) Risks and benefits of phase 1 oncology trials, 1991 through 2002. New Engl J Med 352: 895-904.
29. Gomez-Roca C, Koscielny S, Dromain C, Marzouk I, Bidault F, et al. (2009) Comparison between RECIST evaluations and variations in tumor growth dynamics in patients included in phase I trials. J Clin Oncol 27,15S (May 20 Supplement): 2515.