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A basket trial tests one drug in patients whose cancers share a mutation or marker, whatever organ the cancer started in; each organ type is a basket, and the trial asks whether the drug works across them.
The basket design turns the usual logic of a cancer trial inside out. Instead of one cancer and one drug, it takes one molecular target and enrols any cancer that carries it, sorting patients into baskets by tumour type. Each basket is usually a small single-arm cohort with response rate as its endpoint, and the trial can close baskets that show no responses while expanding the ones that do. Baskets are the natural design for rare alterations that appear in a few percent of many different cancers, where no single-cancer trial could ever recruit enough patients.
The worked example is NAVIGATE, the larotrectinib trial that enrolled adults and children with TRK fusion-positive solid tumours of any type. Pooled with the adult phase 1 and the paediatric SCOUT study, 75 percent of the first 55 patients responded across 17 tumour types, and the result supported the 2018 US accelerated approval of larotrectinib for a genomic marker regardless of tumour site, the first of its kind. ROAR was a basket trial of dabrafenib and trametinib in BRAF V600E-mutant cancers whose anaplastic thyroid cancer cohort of 36 patients showed a 56 percent response rate and produced the first targeted therapy for that disease. NCI-COG Pediatric MATCH was a screening protocol with a dozen single-agent arms: in the first 1,000 children sequenced, 31.5 percent had an actionable alteration and 13.1 percent enrolled on a treatment arm, proving that the plumbing works even though most single drugs did little.
The design has known weaknesses. Small baskets give wide confidence intervals, so a handful of responses can look like a breakthrough. A marker can be a true driver in one organ and a passenger in another, which is why BRAF inhibitors work in melanoma and hairy cell leukaemia but not in BRAF-mutant colorectal cancer without a partner drug. And a basket trial cannot show that patients live longer, because there is no control arm; the response rate and its durability are what regulators weigh, usually with a confirmatory commitment attached.
Showing the molecule this term concerns: Larotrectinib.
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