Phase II Trial of nab-Sirolimus in Patients With Advanced Malignant Perivascular Epithelioid Cell Tumors (AMPECT): Long-Term Efficacy and Safety Update
Clinical trials frequently measure different variables that are collected at different times. These results are often published as updates which allow the investigators to provide all the results from a clinical trial in a timely manner. In the publication, “Phase II Trial of nab-Sirolimus in Patients With Advanced Malignant Perivascular Epithelioid Cell Tumors (AMPECT): Long-Term Efficacy and Safety Update,” the investigators provide an update from the phase II Advanced Malignant Perivascular Epithelioid Cell Tumors (AMPECT) trial (ClinicalTrials.gov identifier: NCT02494570) with nab-Sirolimus, which is an albumin-bound nanoparticle that inhibits a protein called mTOR and is intravenously administered. Nab-sirolimus is approved in the United States for the treatment of patients with locally advanced, unresectable, or metastatic malignant perivascular epithelioid cell tumor (PEComa).
In this publication, the investigators report the final efficacy and safety data 3 years after the primary analysis was completed. They confirm that the overall response rate was 38.7% with the median progression-free survival at 10.6 months. The median duration of response was 39.7 months, and the median overall survival at completion was 53.1 months. The most common treatment-related adverse events were stomatitis (82.4%) and fatigue and rash (each 61.8%). These results demonstrate the clinical benefit of nab-sirolimus in patients with advanced malignant PEComa.