{"entity":{"id":"ampect","kind":"trial","name":"AMPECT","aka":[],"tldr":"A single-arm trial of albumin-bound sirolimus in the ultra-rare tumour PEComa produced lasting responses in about four in ten patients and won the first approval for the disease.","summary":"AMPECT enrolled 34 patients with advanced malignant perivascular epithelioid cell tumour, 31 evaluable, treated with intravenous nab-sirolimus on days 1 and 8 of 21-day cycles. The independently assessed objective response rate was 39 percent, responses were durable and were seen mainly in tumours with TSC2 mutations, and toxicity was consistent with mTOR inhibition (mucositis, rash, hyperglycaemia). Published in the Journal of Clinical Oncology in 2021, it led to FDA approval of nab-sirolimus (Fyarro) in November 2021, the first drug approved for PEComa, and to the PRECISION 1 basket trial in TSC1/TSC2-altered solid tumours.","status":"positive","asOf":"2026-09-17","links":[{"label":"ClinicalTrials.gov NCT02494570","url":"https://clinicaltrials.gov/study/NCT02494570"},{"label":"Journal of Clinical Oncology 2021","url":"https://doi.org/10.1200/JCO.21.01728"}],"tags":["subtype-page"],"related":[],"cancers":["pecoma","sarcoma"],"sections":[],"technologies":[],"targets":["mtor"],"drugs":["sirolimus-albumin-bound"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"nct":"NCT02494570","phase":"2","setting":"Advanced malignant PEComa: nab-sirolimus (albumin-bound sirolimus)","sponsor":"Aadi Bioscience","result":"Objective response 39% (independent review), durable, enriched in TSC2-mutant tumours; FDA approval November 2021.","yearReported":2021,"enrolled":34,"outcomes":[{"endpoint":"Objective response rate (independent review)","primary":true,"unit":"percent","arms":[{"name":"Nab-sirolimus","n":31,"value":39}],"source":"https://doi.org/10.1200/JCO.21.01728"}]},"route":"/trials/ampect/","neighbours":{"cancer":[{"id":"pecoma","kind":"cancer","name":"Perivascular epithelioid cell tumour (PEComa)","route":"/cancers/pecoma/"},{"id":"sarcoma","kind":"cancer","name":"Sarcomas (soft tissue, bone, GIST)","route":"/cancers/sarcoma/"}],"target":[{"id":"mtor","kind":"target","name":"mTOR","route":"/targets/mtor/"}],"drug":[{"id":"sirolimus-albumin-bound","kind":"drug","name":"Sirolimus protein-bound particles","route":"/drugs/sirolimus-albumin-bound/"}]}}