CO-5122

What is CO-5122?

CO-5122 is an investigational substance that is being studied for urea cycle disorders (UCD). The substance is also referred to by the development code CMP-CPS-001 in the trial registry record.^[1] Urea cycle disorders are rare genetic conditions in which the body cannot properly remove ammonia, a waste product of protein breakdown, from the blood. CO-5122 has no marketing authorisation in these records and remains an investigational medicine at this stage of development.

How does CO-5122 work?

The registry data do not describe the mechanism of action of CO-5122 in detail. The trial record indicates that CO-5122 is given by subcutaneous injection, which means it is injected under the skin.^[1] The study includes participants with an abnormal heterozygous ornithine transcarbamylase (OTC) genotype, which suggests the substance is being investigated in relation to the OTC enzyme, one of the enzymes involved in the urea cycle.

Which conditions is CO-5122 being studied for?

• Urea cycle disorders (UCD) — a group of rare inherited conditions that affect the body's ability to remove ammonia from the blood.^[1]

How is CO-5122 given?

In the authorised trial, CO-5122 is given by subcutaneous injection, which means it is injected under the skin.^[1] This is the only pharmaceutical form recorded for CO-5122 in the available data.

What do the clinical trials show?

One Phase 1 trial of CO-5122 is authorised, with a planned enrollment of 144 participants.^[1] The trial is described as a double-blind, placebo-controlled, single and multiple ascending dose study. This means that neither the participants nor the researchers know who is receiving CO-5122 and who is receiving a placebo, and that the dose is gradually increased in different groups of participants.

The participants include both healthy volunteers and people with an abnormal heterozygous ornithine transcarbamylase (OTC) genotype.^[1] The trial status is Authorised, which means it has been approved to begin but has not yet started recruiting participants.

What are the trials measuring?

The primary outcomes of the authorised trial focus on safety and tolerability. These include the incidence of adverse events, vital signs, electrocardiogram (ECG) measurements, clinical laboratory assessments, and physical examination.^[1] The brief summary states that the objective is to evaluate the safety and tolerability of CO-5122 when given subcutaneously to study participants.^[1]

Who can take part in these trials?

The authorised trial includes healthy volunteers and participants with an abnormal heterozygous ornithine transcarbamylase (OTC) genotype.^[1] The registry record does not provide further details on the specific inclusion and exclusion criteria for this study.