Asset profile
Pegargiminase
Small molecule
Evidence outlook · lead readout
NCT01910012 · Acute Myeloid Leukemia
The reported results come from study designs that frequently fail to replicate under controlled conditions — see the rationale below.
NCT01910012: This was a single-arm, unblinded study with no control group and only 43 patients, so there was never a head-to-head comparison to demonstrate the drug's effect versus placebo or another treatment. No results have been posted, but even once posted, the lack of randomization and control means this design cannot rise above weak evidence.
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