This summarizes a clinical trial studying the effectiveness of a melanoma vaccine compared to standard of care for patients with unresectable melanoma. The randomized, double-blinded, placebo-controlled trial assigns patients to either receive the melanoma vaccine or a placebo by direct injection into a tumor site. The primary objective is to evaluate tumor response rates between the two groups. Secondary objectives include assessing safety, progression-free survival, and overall survival.

1. Scenario #5 Anne Marcy & Nancy Shum Introduction to Clinical Data Management

2. Melanoma : A form of cancer that begins in melanocytes (cells that make the pigment melanin). It may begin in a mole (skin melanoma), but can also begin in other pigmented tissues, such as in the eye or in the intestines. Melanoma can metastasize to virtually any organ site. Metastases most commonly occur in the skin or soft tissues, the lung, and the liver (visceral metastasis) Estimated new cases and deaths from melanoma in the United States in 2008: New Cases 62,480: Deaths: 8,420 (NCI)

4. American Joint Committee on Cancer Staging System for Cutaneous Melanoma. J Clin Oncol 2001; 19:3635-3548. 6% 9% 41% All other visceral mets with normal LDH or any distant mets with increased LDH (anyTanyNM1c) 3% 7% 57% Lung mets with normal LDH (anyTanyNM1b) 16% 19% 59% Distant skin, subcutaneous, or nodal mets with normal LDH (any TanyNM1a) IV 18% 27% 4 or more metastatic nodes, matted nodes/gross extracapsular extension, or in-transit met(s)/satellite(s) and metastatic nodes (anyTN3M0) 15% 24% 2-3 macroscopic regional nodes, ulcerated primary (T1-4bN2bM0) 24% 29% Single microscopic regional node, ulcerated primary (T1-4bN1bM0) IIIC 30-50% In-transit met(s)/satellite lesion(s) without metastatic lymph nodes (T1-4a/bN2cM0) 39% 46% 2-3 macroscopic regional nodes, nonulcerated primary (T1-4aN2bM0) 48% 59% Single regional nodal macrometastasis, nonulcerated primary (T1-4aN1bM0) 36% 50% 2-3 microscopic regional nodes, ulcerated primary (T1-4bN2aM0) 38% 53% Single regional nodal micrometastasis, ulcerated primary (T1-4bN1aM0) IIIB 57% 63% 2-3 microscopic regional nodes, nonulcerated primary (T1-4aN2aM0) 63% 69% Single regional nodal micrometastasis, nonulcerated primary (T1-4aN1aM0) IIIA 32% 45% > 4 mm with ulceration (T4bN0M0) IIC 54% 67% > 4 mm without ulceration (T4aN0M0) 51% 63% 2.01-4 mm with ulceration (T3bN0M0) IIB 64% 79% 2.01-4 mm without ulceration (T3aN0M0) 64% 77% 1.01-2 mm with ulceration (T2bN0M0) IIA 79% 89% 1.01-2 mm without ulceration (T2aN0M0) 83% 91% < 1 mm with ulceration or level IV/V (T1bN0M0) IB 88% 95% < 1 mm without ulceration and Clark Level II/III (T1aN0M0) IA 100% 100% Intraepithelial/in situ melanoma (TisN0M0) 0 10-year 5-year 1-year Overall Survival Histological Features/TNM Classification Stage

7. The Study Plan Develop research question Design study Implement study Analyze results Infer conclusions

8. Schematic Diagram of the Study Design Randomized Assessor Blinded Study Drug- Vaccine Placebo Controlled