Urea cycle disorders (UCD) research hub

A Double-blind, Placebo-controlled, Single and Multiple Ascending Dose Phase 1 Study of CMP-CPS-001 in Healthy Volunteers and Participants with Abnormal Heterozygous Ornithine Transcarbamylase (OTC) Genotype

This early-stage study is looking at a new medicine (CMP-CPS-001) for a rare condition called urea cycle disorder. Researchers want to see if it's safe and how it works in healthy people and those with inherited genetic changes related to the condition. It's the first time this medicine is being tested in humans.