Phase II Trial of nab-Sirolimus in Patients With Advanced Malignant Perivascular Epithelioid Cell Tumors (AMPECT):...

Area of Science:

• Oncology
• Clinical Pharmacology
• Medical Imaging

Background:

• nab-sirolimus is approved for metastatic or locally advanced malignant PEComa.
• Primary analysis of the AMPECT trial reported initial efficacy but median duration of response (mDOR) was not reached.
• Clinical Trial Updates allow dissemination of mature data after primary endpoint reporting.

Purpose of the Study:

• To report final efficacy and safety data from the AMPECT trial for nab-sirolimus in advanced malignant PEComa.
• To assess long-term outcomes including median duration of response (mDOR) and overall survival (OS).
• To evaluate the safety profile of nab-sirolimus with extended follow-up.

Main Methods:

• Phase II clinical trial (AMPECT, NCT02494570) evaluating nab-sirolimus.
• Data cutoff: April 29, 2022, approximately 3 years after primary analysis.
• Efficacy assessed by independent radiologist review (RECIST v1.1); safety data collected throughout the study.

Main Results:

• Confirmed overall response rate (ORR) was 38.7% (95% CI, 21.8 to 57.8).
• Median duration of response (mDOR) reached 39.7 months (95% CI, 6.5 to NR).
• Median overall survival (OS) was 53.1 months (95% CI, 22.2 to NR).
• Most common treatment-related adverse events (TRAEs) included stomatitis (82.4%) and fatigue/rash (61.8%); no grade ≥4 TRAEs reported.

Conclusions:

• nab-sirolimus demonstrates durable clinical benefit in advanced malignant PEComa.
• Long-term follow-up confirms a substantial median duration of response exceeding 3 years.
• The safety profile remains consistent and manageable, supporting its role in treating this rare tumor type.