Advanced Therapy Medicinal Products (ATMPs) comprising cell, gene, and tissue-engineered therapies have demonstrated enormous therapeutic benefits. However, their development is complex to be managed efficiently within currently existing regulatory frameworks. Legislation and regulation requirements for ATMPs must strike a balance between the patient safety while promoting innovations to optimize exploitation of these novel therapeutics. This paradox highlights the importance of on-going dynamic dialogue between all stakeholders and regulatory science to facilitate the development of pragmatic ATMP regulatory guidelines.
Background: Basal cell carcinoma (BCC) is the most commonly diagnosed skin cancer, with increasing incidence each year. Although the treatment of BCC is typically surgical, a non-surgical approach is highly desirable for patients with non-aggressive BCC recurrence, multiple tumors, poor general health, or with lesions in vulnerable areas. In the last few years, advanced cancer treatment options have targeted the Hedgehog pathway, known to regulate the proliferation of cancer stem cells and increase tumor invasiveness. Itraconazole, a triazole antifungal that inhibits ergosterol synthesis in fungi, have also proven a clinically significant tumor size reduction in BCC via inhibition of this pathway. However, serious adverse effects might preclude itraconazole use in broader population where multiple medications or comorbidities might be present. Preliminary studies demonstrate posaconazole, another member of the triazole family, may present a safer clinical alternative to the currently used pharmacological methods utilised in the management of BCC. Therefore, this phase II study aims to analyze the efficacy of posaconazole as a safe therapeutic option for nodular BCC lesions. Methods: This is a study protocol designed as a single center, randomized, double-blinded, placebo-controlled trial. Patients over 18 years of age, diagnosed with at least 2 nodular BCC lesions and that are awaiting to undergo surgery are eligible to participate in this phase II study. Before undergoing surgical excision, 170 patients (1:1 allocation) will be randomized to receive 800 mg daily of posaconazole or placebo for four weeks. Discussion: The main strengths of this study are the potential to increase general understanding of the therapeutic and metabolic impact of posaconazole. The results from this trial may also provide valuable insights to guide a larger clinical trial of posaconazole for inoperable BCC. The main limitations of the study consist of the limited intervention time frame of four weeks and the sample representativeness since it is a single-center study of patients with nodular BCC.
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