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FIRSTMAPPP was the first randomised trial ever completed in metastatic phaeochromocytoma and paraganglioma, rare hormone-producing tumours; the kinase inhibitor sunitinib kept 36 percent of patients free of progression at a year against 19 percent on placebo, giving the disease its first drug with randomised evidence.
FIRSTMAPPP was an academic, double-blind, placebo-controlled phase 2 trial of the ENDOCAN-COMETE and ENSAT networks at 14 centres in four European countries, randomising 78 adults with progressive metastatic phaeochromocytoma or paraganglioma (a third with germline SDHx variants, two thirds previously treated) to sunitinib or placebo. The primary endpoint was progression-free survival at 12 months.
The primary endpoint was met: 14 of 39 patients on sunitinib (36 percent) were progression-free at 12 months against 7 of 39 on placebo (19 percent). Asthenia and hypertension were the common grade 3 or 4 adverse events; one drug-related death from rectal bleeding occurred. The corpus's metastatic phaeochromocytoma and paraganglioma page cites FIRSTMAPPP for sunitinib alongside belzutifan and cabozantinib.
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.
78 enrolled.
14 of 39; 90% CI 23 to 50 · 7 of 39; 90% CI 11 to 31
SourceShares Small-molecule kinase inhibitors and the tag soc-trials.
Shares Small-molecule kinase inhibitors and the tag soc-trials.
Shares Small-molecule kinase inhibitors and the tag soc-trials.
Shares Small-molecule kinase inhibitors and the tag soc-trials.
Shares Small-molecule kinase inhibitors and the tag soc-trials.
Shares Small-molecule kinase inhibitors and the tag soc-trials.
Shares Small-molecule kinase inhibitors and the tag soc-trials.
Shares Small-molecule kinase inhibitors and the tag soc-trials.