New shot targets rare liver disorder in early human tests

NCT ID NCT06247670

First seen Jun 27, 2026 ยท Last updated Jun 27, 2026

Summary

This early-stage study tests a new drug called CMP-CPS-001 in healthy adults and women who carry a gene for OTC deficiency, a rare liver condition that can cause dangerous ammonia buildup. The main goal is to check the drug's safety and how the body processes it. Participants receive either the drug or a placebo as a shot under the skin.

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Conditions

The condition(s) this trial relates to.

ornithine carbamoyltransferase deficiency urea cycle disorder

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Erasmus MC

    Rotterdam, Netherlands

  • Nucleus Network Brisbane (also known as Q-Pharm Pty Ltd)

    Herston, Queensland, Australia