New shot targets rare liver disorder in early human tests

NCT ID NCT06247670

First seen Jun 27, 2026 · 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.

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

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Other studies related to the condition(s) this trial covers.