An intra-patient placebo-controlled phase I trial to evaluate the safety and tolerability of intradermal IMM-101 in melanoma.

BACKGROUND: IMM-101 is a heat-killed innate and adaptive immune-activating mycobacterial product; a phase I study aimed to determine its safety and tolerability in individuals with melanoma. PATIENTS AND METHODS: An intra-patient placebo-controlled study evaluated the safety and tolerability of three doses, namely, 0.1 (1 mg/ml), 0.5 (5 mg/ml) and 1.0 mg (10 mg/ml) of IMM-101 in stage III or IV melanoma. Each dose was administered in ascending order to one of the three cohorts. RESULTS: Based on observations from patients administered the 0.1-mg dose, it was considered appropriate to proceed with dosing the patients in the 0.5-mg dose cohort and then the 1.0-mg cohort (n = 6 per cohort). Treatment-emergent adverse events that would be considered typical of a post-vaccination state (including joint pains/aches, headaches and influenza-like symptoms) occurred at all dose levels, along with injection site reactions. These were mainly mild in intensity, resolved in a matter of days and responded well to supportive care. During post-study follow-up, two clinical responses (15%) were observed in patients with stage IV disease. CONCLUSION: IMM-101 is safe and well tolerated and there is a rationale for studying IMM-101 at a nominal 1.0-mg dose to complement conventional cytotoxic therapy for patients with advanced cancer.