Asset profile

Zamicastat

Small molecule

Phase 3DBHNo recent registered activity
Weaksignal

Evidence outlook · lead readout

NCT04316143 · Pulmonary Arterial Hypertension

The reported results come from study designs that frequently fail to replicate under controlled conditions — see the rationale below.

NCT04316143: The study was non-randomized, open-label, sequential, and had no placebo or active comparator; 33 patients were enrolled, with only 1, 4, and 17 patients contributing to key pharmacokinetic analyses at 50, 100, and 200 mg. Its primary endpoints were pharmacokinetic surrogate measures, and there was no formal comparative efficacy endpoint to meet or miss.

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