6 results
To assess the objective response rate (ORR), progression free survival (PFS), overall survival (OS), duration of response (DoR), toxicity, and quality of life (QoL) of patients with advanced SGC treated with cabozantinib in 3 cohorts: salivary duct…
The overall objective of this study is to evaluate the efficacy and safety of neoGAA in treatment-naïve patients with LOPD as compared to alglucosidase alfa, when this is administered biweekly for a period of 49 weeks. Also, there is an open-label…
Primary objectives: to evaluate the efficacy of three different single doses of PHA-022121 versus placebo in achieving angioedema symptom relief during acute attacks and the efficacy of prophylactic treatment with PHA-022121 versus placebo in…
To demonstrate that a dose reduction of 20-33% of CYP3A4 metabolized tyrosine kinase inhibitors in patients expressing the CYP3A4*22 gene (rs35599367 C>T in intron 6) does not result in a lower exposure (Ctrough) than the wildtype group with…
The purpose of this study is to find out how safe and effective the new drug PHA-022121 is for the treatment of Hereditary Angioedema. The main purpose of this research study is to test the effectiveness of 3 different doses of PHA-022121 in…
The primary objective of this study is to evaluate the efficacy of cabozantinib in combination with atezolizumab versus sorafenib in subjects with advanced HCC who have not received previous systemic anticancer therapy. An secondary objective is to…