Berger M, Grignani G, Ferrari S, Biasin E, Brach del Prever A, Aliberti S, Saglio F, Aglietta M, Fagioli F Phase 2 trial of two courses of cyclophosphamide and etoposide for relapsed high-risk osteosarcoma patients. Cancer. 2009 Jul 1;115(13):2980-7. doi: 10.1002/cncr.24368. Erratum in: Cancer. 2009 Nov 1;115(21):5126. Massimo, Berger [corrected to Berger, M]; Giovanni, Grignani [corrected to Grignani, G]; Stefano, Ferrari [corrected to Ferrari, S]; Eleonora, Biasin [corrected to Biasin, E]; Adalberto, Brach Del Prever [corrected to Brach del Prever, A]; Sandra, Aliberti [corrected to Aliberti, S]; Francesco, Saglio [corrected to Saglio, F]; Masimo, Aglietta [corrected to Aglietta, M]; Franca, Fagioli [corrected to Fagioli, F].
Bernthal NM, Federman N, Eilber FR, Nelson SD, Eckardt JJ, Eilber FC, Tap WD Long-term results (>25 years) of a randomized, prospective clinical trial evaluating chemotherapy in patients with high-grade, operable osteosarcoma. Cancer. 2012 Dec 1;118(23):5888-93. doi: 10.1002/cncr.27651. Epub 2012 May 30.
Linch M, Miah AB, Thway K, Judson IR, Benson C Systemic treatment of soft-tissue sarcoma-gold standard and novel therapies. Nat Rev Clin Oncol. 2014 Apr;11(4):187-202. doi: 10.1038/nrclinonc.2014.26. Epub 2014 Mar 18. Review.
McNall-Knapp RY, Williams CN, Reeves EN, Heideman RL, Meyer WH Extended phase I evaluation of vincristine, irinotecan, temozolomide, and antibiotic in children with refractory solid tumors. Pediatr Blood Cancer. 2010 Jul 1;54(7):909-15. doi: 10.1002/pbc.22460.
Schöffski P, Cornillie J, Wozniak A, Li H, Hompes D Soft tissue sarcoma: an update on systemic treatment options for patients with advanced disease. Oncol Res Treat. 2014;37(6):355-62. doi: 10.1159/000362631. Epub 2014 Apr 17. Review.
Semiglazov V RECIST for Response (Clinical and Imaging) in Neoadjuvant Clinical Trials in Operable Breast Cancer. J Natl Cancer Inst Monogr. 2015 May;2015(51):21-3. doi: 10.1093/jncimonographs/lgv021.
Yoo C, Lee J, Rha SY, Park KH, Kim TM, Kim YJ, Lee HJ, Lee KH, Ahn JH Multicenter phase II study of everolimus in patients with metastatic or recurrent bone and soft-tissue sarcomas after failure of anthracycline and ifosfamide. Invest New Drugs. 2013 Dec;31(6):1602-8. doi: 10.1007/s10637-013-0028-7. Epub 2013 Sep 14.
Interventional studies are often prospective and are specifically tailored to evaluate direct impacts of treatment or preventive measures on disease.
Observational studies are often retrospective and are used to assess potential causation in exposure-outcome relationships and therefore influence preventive methods.
Expanded access is a means by which manufacturers make investigational new drugs available, under certain circumstances, to treat a patient(s) with a serious disease or condition who cannot participate in a controlled clinical trial.
Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.
Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.