Active clinical trials for "Perivascular Epithelioid Cell Neoplasms"

This is a phase Ⅰb/Ⅱ multi-center study of safety and efficacy of Sirolimus for Injection (Albumin-bound) in patients with advanced malignant perivascular epithelioid cell tumor (PEComa).

This trial is a translational, open-label, multi-sites, prospective and retrospective cohort study of 500 patients aimed at clinical and biological characterization of sarcoma of rare subtype. 400 patients will be included in this prospective cohort study; they will be identified in the investigating centers in the context of either routine care or a clinical study protocol. Retrospective cases of patients (100 cases in total) will be identified in all centers through the GSF/GETO clinical databases already setted up (including the clinical base Conticabase).

Perivascular epithelioid cell tumors (PEComas) are rare and are characterized by the expression of myomelanocytic markers. They are a complex family that includes angiomyolipomas, lymphangioleiomyomatoses and other soft tissue and visceral tumors. Due to the low prevalence of these tumors, the natural history is unclear; furthermore, a molecular classification integrating clinical, pathological and molecular parameters has not been described to date.

A phase 2 multi-center investigation of efficacy of nab-sirolimus (formerly known as ABI-009 or nab-rapamycin) in patients with advanced malignant perivascular epithelioid cell tumor (PEComa).

Study objectives: The primary objective is to determine the efficacy of BEZ235 on Objective Response Rate (best response on study) according to RECIST 1.1 criteria The secondary objectives are: To determine the progression free survival rate at 32 weeks in the included population To assess the duration of response among responders To evaluate time to response To evaluate the time to progression To assess the overall survival To evaluate safety and tolerability of BEZ235 The exploratory objectives are: To identify molecular and genomic profiles of PEComas and their potential relationship to clinical outcome by analyzing PIK3CA, Ras, Raf, TSC, AKT and PTEN alteration in tumor samples (archival or fresh pre-treatment tumor biopsy) and PIK3CA in circulating DNA. To determine biomarkers relevant to BEZ235 activity by analyzing the expression of phosphoproteins p-AKT, p-S6, p-4EBP1 at screening and during treatment as well as biomarkers for the proliferation (Ki-67) and apoptosis (PARP) (only if fresh tissue (optional) is available). Study population: The patient population consists of patients 18 years old or older with progressive unresectable/advanced or metastatic malignant PEComas previously treated for unresectable/advanced/metastatic disease with 1 to 2 prior lines of chemotherapy. Patients must have adequate hematologic, renal, cardiac and hepatic functions and not be previously treated with a mTOR inhibitor. Number of patients: 16 to 33 patients Overview of study design: This is a prospective, multicenter, open-label, single arm, two-stage phase II study to investigate the efficacy and tolerability of BEZ235 in patients with progressive metastatic or unresectable/advanced malignant PEComas. The patient should have received 1 or 2 prior lines of chemotherapy. BEZ235 will be administered until disease progression. Sixteen patients will be enrolled into Stage 1 and observed for at least 32 weeks at which time an interim analysis will be performed (plus eventually 4-5 weeks for confirmation of responses occurring on or closely before this cut-off date). If the number of patients with a response (CR or PR) is 2 or less, the trial will be stopped for futility. If 3 or more patients experience a response enrollment will continue up to 33 patients (Stage 2). An Independent Data Monitoring Committee (IDMC) will be constituted for reviewing the interim analysis.

PEComa is rare tumor affecting particularly patients with Tuberous Sclerosis. Biological similarities were seen between PEComa and infantile hemangioma. Propranolol is highly efficient to treat infantile hemangioma and we believe that this drug can also be useful for the treatment of PEComa. Purpose : to understand the mechanism of action of propranolol in PEComas and related pediatric vascular lesions and to select possible novel targets of betablockers in vascular tumors related to PEComas by using YAP oncogene activation as a molecular marker

Expanded Access for an Intermediate-size Population for ABI-009 (Sirolimus Albumin-bound Nanoparticles for Injectable Suspension) in Patients with Advanced Perivascular Epithelioid Cell Tumors (PEComa) and Patients with a Malignancy with Relevant Genetic Mutations or mTOR Pathway Activation

Perivascular epithelioid cell tumors are rare and characterized by the expression of myomelanocytic markers. They belong to a complex tumor family that includes angiomyolipomas, lymphangioleiomyomatosis and other soft-tissue tumors. Given their rarity, the natural history of pecomas is not yet understood, and a comprehensive classification that integrates clinical, pathological and molecular features has not been achieved as of today. This study is a national multicenter retrospective study to better understand the natural history of PEComas, excluding lymphangioleiomyomatosis and classic triphasic angiomyolipomas. The primary purpose is the identification of prognostic markers impacting the relapse. Secondaries purposes are to identify prognostic markers impacting the overall survival and to have a better understanding of natural history