AMPECT
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.
Overview
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.
- 39 out of 100 people had their tumour shrink with Nab-sirolimus.
- There was no comparison group, so this number cannot tell you how much of the effect is due to the treatment.
- A response rate counts how many people had their tumours shrink by a set amount; shrinking is encouraging but does not by itself show that people live longer or feel better.
- These results apply to the people the trial enrolled: Advanced malignant PEComa: nab-sirolimus (albumin-bound sirolimus). People in a different situation may not see the same effect.
- Only 34 people took part, so the numbers are less certain than in a large trial.
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.
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