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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.
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.
Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.
Shares Sirolimus protein-bound particles, mTOR and the tag subtype-page.
Shares Sarcomas (soft tissue, bone, GIST), Small-molecule kinase inhibitors and the tag subtype-page.
Shares Perivascular epithelioid cell tumour (PEComa), Sarcomas (soft tissue, bone, GIST) and the tag subtype-page.
Shares Perivascular epithelioid cell tumour (PEComa), Sarcomas (soft tissue, bone, GIST) and the tag subtype-page.
Shares Sarcomas (soft tissue, bone, GIST) and the tag subtype-page.
Shares Sarcomas (soft tissue, bone, GIST) and the tag subtype-page.
Shares Sarcomas (soft tissue, bone, GIST) and the tag subtype-page.
Shares Sarcomas (soft tissue, bone, GIST), Small-molecule kinase inhibitors and the tag subtype-page.