Multicenter, single-arm, open-label, phase 2 registrational study evaluating FYARRO in
34 patients (safety population)1,2
Primary analysis† at 6 months after the last patient initiated therapy1
Second analysis performed 2 years after last patient initiated therapy (1.5 years following primary analysis)1
Final analysis performed at 2.5 years after the last patient initiated therapy2
*A maximum of 2 dose reductions by 25% each (to 75 and then 56 mg/m2) were permitted for toxicity.1
†The primary analysis was preplanned to occur when the last enrolled patient had been treated for 6 months.1
AMPECT=Advanced Malignant PEComa Trial; PEComa=perivascular epithelioid cell tumor; RECIST=Response Evaluation Criteria in Solid Tumors; SD=stable disease.
|Selected baseline characteristics||All patients (N=34)|
|Age, median (range)||60 years (27 to 78)|
|Pacific Islander or Hawaiian||3%|
|Locally advanced (inoperable)||15%|
|Number of metastatic sites|
|Prior systemic treatment‡||12%|
Included docetaxel, doxorubicin, gemcitabine, ifosfamide, and olaratumab.
AMPECT=Advanced Malignant PEComa Trial; ECOG=Eastern Cooperative Oncology Group; mTOR=mechanistic target of rapamycin; nab=nanoparticle albumin-bound; PEComa=perivascular epithelioid cell tumor.
References: 1. Wagner AJ, Ravi V, Riedel RF, et al. nab-Sirolimus for patients with malignant perivascular epithelioid cell tumors. J Clin Oncol. 2021;39(33):3660-3670. doi:10.1200/JCO.21.01728 2. Wagner AJ, Ravi V, Riedel RF, et al. Final analysis from AMPECT, an open-label phase 2 registration trial of nab-SIROLIMUS for patients with advanced malignant perivascular epithelioid cell tumors (PEComa). Abstract presented at: CTOS Virtual Annual Meeting; November 12, 2021. 3. Sanfilippo R, Jones RL, Blay JY, et al. Role of chemotherapy, VEGFR inhibitors, and mTOR inhibitors in advanced perivascular epithelioid cell tumors (PEComas). Clin Cancer Res. 2019;25(17):5295-5300. doi:10.1158/1078-0432.CCR-19-0288