A review of study designs and outcomes of phase I clinical studies of nanoparticle agents compared with small-molecule anticancer agents

Clinical Cancer Research

Volumn 19, Issue 12, 2013, Pages 3309-3315

  Caron, Whitney P ^a   Morgan, Katherine P ^a   Zamboni, Beth A ^e   Zamboni, William C ^a,b,c,d  

^a University of North Carolina at Chapel Hill   (United States)

^b UNIVERSITY OF NORTH CAROLINA   (United States)

^c Carolina Center of Cancer Nanotechnology Excellence   (United States)

^d North Carolina Biomedical Innovation Network   (United States)

^e CARLOW UNIVERSITY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANTINEOPLASTIC AGENT; BELOTECAN; ETIRINOTECAN PEGOL; IRINOTECAN; LIPOSOMAL VINCRISTINE; LIPOSOMAL VINORELBINE; LURTOTECAN; MBP 426; NANOPARTICLE; NAVELBINE; NK 105; OXALIPLATIN; PACLITAXEL; PEGYLATED LIPOSOMAL BELOTECAN; PEGYLATED LIPOSOMAL IRINOTECAN; PEGYLATED LIPOSOMAL OXALIPLATIN; PEGYLATED PACLITAXEL; UNCLASSIFIED DRUG; VINCRISTINE;

AREA UNDER THE CURVE; ARTICLE; DISEASE MODEL; DOG; DRUG COST; DRUG DISPOSITION; DRUG DOSE ESCALATION; HUMAN; MAXIMUM TOLERATED DOSE; NONHUMAN; OUTCOME ASSESSMENT; PHASE 1 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; SOLID TUMOR;

ANIMALS; ANTINEOPLASTIC AGENTS; CLINICAL TRIALS, PHASE I AS TOPIC; DISEASE MODELS, ANIMAL; DOGS; DOSE-RESPONSE RELATIONSHIP, DRUG; DRUG DELIVERY SYSTEMS; HUMANS; MAXIMUM TOLERATED DOSE; NANOPARTICLES; NEOPLASMS; NEOPLASMS, EXPERIMENTAL; RATS;

EID: 84879495288     PISSN: 10780432     EISSN: 15573265     Source Type: Journal    
DOI: 10.1158/1078-0432.CCR-12-3649     Document Type: Article

References (46)

1. Drummond DC, Meyer O, Hong K, Kirpotin DB, Papahadjopoulos D. Optimizing liposomes for delivery of chemotherapeutic agents to solid tumors. Pharmacol Rev 1999;51:691-743.
2. Papahadjopoulos D, Allen TM, Gabizon A, Mayhew E, Matthay K, Huang SK, et al. Sterically stabilized liposomes: Improvements in pharmacokinetics and antitumor therapeutic efficacy. Proc Natl Acad Sci U S A 1991;88:11460-4.
3. Zamboni WC. Liposomal, nanoparticle, and conjugated formulations of anticancer agents. Clin Cancer Res 2005;11:8230-4.
4. Zamboni WC. Concept and clinical evaluation of carrier-mediated anticancer agents. Oncologist 2008;13:248-60.
5. Park JW, Benz CC, Martin FJ. Future directions of liposome- and immunoliposome-based cancer therapeutics. Semin Oncol 2004;31(6 Suppl 13):196-205.
6. Zamboni WC, Tonda ME. New designs of clinical trials. Highlights in Oncol. Practice 2000;18:2-7.
7. Laverman P, Carstens MG, Boerman OC, Dams ET, Oyen WJ, van Rooijen N, et al. Factors affecting the accelerated blood clearance of polyethylene glycol-liposomes upon repeated injection. J Pharmacol Exp Ther 2001;298:607-12.
8. Woodle MC, Lasic DD. Sterically stabilized liposomes. Biochim Biophys Acta 1992;1113:171-99.
9. Zamboni WC, Maruca LJ, Strychor S, Zamboni BA, Ramalingam S, Edwards RP, et al. Bidirectional pharmacodynamic interaction between pegylated liposomal CKD-602 (S-CKD602) and monocytes in patients with refractory solid tumors. J Liposome Res 2011;21: 158-65.
10. Drug excretion: Pharmacokinetics in merck manual [homepage on the Internet]. 2012 [cited 2012 Nov 1]. Available from: http://www.merckmanuals.com/ professional/clinical-pharmacology/pharmacokinetics/drug-excretion.html.
11. Le Tourneau C, Lee JJ, Siu LL. Dose escalation methods in phase I cancer clinical trials. J Natl Cancer Inst 2009;101:708-20.
12. NCI Dictionary of Cancer Terms. National Cancer Institute at the National Institutes of Health. 2012 [cited 2012 Nov 1].
13. Zamboni WC, Yoshino K. Formulation and physiologic factors affecting the pharmacokinetics and pharmacodynamics of liposomal agents. Drug Delivery Systems 2010;25:58-70.
14. Sharifi S, Behzadi S, Laurent S, Forrest ML, Stroeve P, Mahmoudi M. Toxicity of nanomaterials. Chem Soc Rev 2012;41:2323-43.
15. Caron WP, Clewell H, Dedrick R, Ramanathan RK, Davis WL, Yu N, et al. Allometric scaling of pegylated liposomal anticancer drugs. J Pharmacokinet Pharmacodyn 2011;38:653-69.
16. Nyman DW, Campbell KJ, Hersh E, Long K, Richardson K, Trieu V, et al. Phase I and pharmacokinetics trial of ABI-007, a novel nanoparticle formulation of paclitaxel in patients with advanced nonhematologic malignancies. J Clin Oncol 2005;23:7785-93.
17. Ibrahim NK, Desai N, Legha S, Sonn-Shiong P, Theriault RL, Rivera E, et al. Phase I and pharmacokinetic study of ABI-007, a cremophor-free, protein stabilized, nanoparticle formulation of paclitaxel. Clin Cancer Res 2002;8:1038-44.
18. Imperiale SM, Tolcher AW, Sarantopoulos J, Batist G, Fontanilla-Gines J, Lu B. Phase 1 study of the sphingomyelin/cholesterol liposome formulation of vinorelbine in subjects with advanced solid tumors, hodgkin's disease, or non-hodgkin's lymphoma (NHL). European Society for Medical Oncology Conference; 2008 Jul 3-6; Lugano, Switzerland. Poster nr 56P.
19. Gelmon KA, Tolcher A, Diab AR, Bally MB, Embree L, Hudon N, et al. Phase I study of liposomal vincristine. J Clin Oncol 1999;17:697-705.
20. Borad MJ, Hamm JT, Rosen LS, Jameson GS, Utz J, Mulay M, et al. Phase I dose finding and pharmacokinetic study of NKTR-102 (PEGylated irinotecan):Early evidence of antitumor activity. J Clin Oncol 2008;26(May 20 suppl; abstr 13518).
21. Sankhala K, Mita A, Adinin R, Wood L, Beeram M, Bullock S, et al. A phase I, pharmacokinetic (PK) study of MBP-426, a novel liposome encapsulated oxliplatin. J Clin Oncol 2009;27(15S; abstr 2535).
22. Zamboni WC, Ramalingam S, Friedland DM, Edwards RP, Stoller RG, Strychor S, et al. Phase I and pharmacokinetic study of pegylated liposomal CKD-602 in patients with advanced malignancies. Clin Cancer Res 2009;15:1466-72.
23. Jones SF, Zamboni WC, Burris HA, Chan E, Infante JR, Keedy V, et al. Phase I and pharmacokinetic (PK) study of IHL-305 (pegylated liposomal irinotecan) in patients with advanced solid tumors. J Clin Oncol 2009;27(15s):suppl; abstr 2547.
24. Hamaguchi T, Kato K, Yasui H, Morizane C, Ikeda M, Ueno H, et al. A phase I and pharmacokinetic study of NK105, a paclitaxel-incorporating micellar nanoparticle formulation. Br J Cancer 2007;97: 170-6.
25. Gerrits CJH, Creemers GJ, Schellens JHM, Wissel P, Th Planting AS, Kunka R, et al. Phase I and pharmacological study of the new topoisomerase I inhibitor GI147211, using a daily X 5 intravenous administration. Br J Cancer 1996;73:744-50.
26. Pitot HC, Goldberg RM, Reid JM, Sloan JA, Skaff PA, Erlichman C, et al. Phase I dose-finding and pharmacokinetic trial of irinotecan hydrochloride (CPT-11) using a once every three week dosing schedule for patients with advanced solid tumor malignancy. Clin Cancer Res 2000;6:2236-44.
27. Jackson DV, Sethi VS, Spurr CL, Willard V, White DR, Richards F, et al. Intravenous vincrisine infusion: phase I trial. Cancer 1981;48:2559-64.
28. Extra JM, Espie M, Calvo F, Ferme C, Mignot L, Marty M. Phase I study of oxaliplatin in patients with advanced cancer. Cancer Chemother Pharmacol 1990;25:299-303.
29. Eisenhauer EA, O'Dwyer PJ, Christian M, Humphrey JS. Phase I clinical trial design in cancer drug development. J Clin Oncol 2000; 18:684-92.
30. Goldman DP, Berry SH, McCabe MS, Kilgore ML, Potosky AL, Schoenbaum ML, et al. Incremental treatment costs in national cancer institute-sponsored clinical trials. JAMA 2003;289:2970-7.
31. Schell R, Caron WP, Walsh MD, Zamboni WC. Relationship between pharmacokinetic (PK) sampling schema and reported PK variability of liposomes in patients. In: Proceedings of the 102nd Annual Meeting of the American Association for Cancer Research; 2011 Apr 2-6; Orlando, FL. Philadelphia (PA): AACR; 2011. Poster nr 1295.
32. Caron WP, Song G, Kumar P, Rawal S, Zamboni WC. Interpatient pharmacokinetic and pharmacodynamic variability of carrier-mediated anticancer agents. Clin Pharmacol Ther 2012;91:802-12.
33. Song G, Wu H, Yoshino K, Zamboni WC. Factors affecting the pharmacokinetics and pharmacodynamics of liposomal drugs. J Liposome Res 2012;22:177-92.
34. Giovinazzo H, Kumar P, Sheikh A, Ivanovic M, Walsh MD, Caron WP, et al. Technetium-99m sulfur colloid (TSC) as a phenotypic probe for predicting pharmacokinetics (PK) and palmar-plantar erythrodysethesia (PPE) toxicity of PEGylated liposomal doxorubicin (PLD) in patients (pts) with recurrent epiethelial ovarian caner (EOC). J Clin Oncol (suppl; abstr 5050) 2012;2591.
35. Mahmood I. Interspecies scaling of maximum tolerated dose of anticancer drugs: Relevance to starting dose for phase I clinical trials. Am J Ther 2001;8:109-16.
36. Allen TM, Martin FJ. Advantages of liposomal delivery systems for anthracyclines. Semin Oncol 2004;31(6 Suppl 13):5-15.
37. Vail DM, Amantea MA, Colbern GT, Martin FJ, Hilger RA, Working PK. Pegylated liposomal doxorubicin: Proof of principle using preclinical animal models and pharmacokinetic studies. Semin Oncol 2004;31(6 Suppl 13):16-35.
38. Gabizon AA, Tzemach D, Horowitz AT, Shmeeda H, Yeh J, Zalipsky S. Reduced toxicity and superior therapeutic activity of a mitomycin C lipid-based prodrug incorporated in pegylated liposomes. Clin Cancer Res 2006;12:1913-20.
39. Gabizon AA. Liposomal drug carrier systems in cancer chemotherapy: Current status and future prospects. J Drug Target 2002;10:535-8.
40. Zhigaltsev IV, Maurer N, Akhong QF, Leone R, Leng E, Wang J, et al. Liposome-encapsulated vincristine, vinblastine and vinorelbine: A comparative study of drug loading and retention. J Control Release 2005;104:103-11.
41. Zamboni WC, Strychor S, Joseph E, Walsh DR, Zamboni BA, Parise RA, et al. Plasma, tumor, and tissue disposition of STEALTH liposomal CKD-602 (S-CKD602) and nonliposomal CKD-602 in mice bearing A375 human melanoma xenografts. Clin Cancer Res 2007;13:7217-23.
42. Sapra P, Moase EH, Ma J, Allen TM. Improved therapeutic responses in a xenograft model of human B lymphoma (namalwa) for liposomal vincristine versus liposomal doxorubicin targeted via anti-CD19 IgG2a or fab' fragments. Clin Cancer Res 2004;10:1100-11.
43. Haaz MC, Rivory LP, Riche C, Robert J. The transformation of irinotecan (CPT-11) to its active metabolite SN-38 by human liver microsomes. differential hydrolysis for the lactone and carboxylate forms. Naunyn Schmiedebergs Arch Pharmacol 1997;356:257-62.
44. Mathijssen RH, van Alphen RJ, Verweij J, Loos WJ, Nooter K, Stoter G, et al. Clinical pharmacokinetics and metabolism of irinotecan (CPT-11). Clin Cancer Res 2001;7:2182-94.
45. Iyer L, King CD, Whitington PF, Green MD, Roy SK, Tephly TR, et al. Genetic predisposition to the metabolism of irinotecan (CPT-11). role of uridine diphosphate glucuronosyltransferase isoform 1A1 in the glucuronidation of its active metabolite (SN-38) in human liver microsomes. J Clin Invest 1998;101:847-54.
46. European Medicines Agency Evaluation of Medicines for Human Use (EMEA) Assessment Report for Abraxane. Procedure No. EMEA/H/C/778. 2007. Null.