Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

New shot targets rare liver disorder in early human tests

NCT ID NCT06247670

Disease control Sponsor: CAMP4 Therapeutics Corporation Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

About 120 people

The number the study aims to enrol. It can still change while the study runs.

Started

Feb 2024

Expected to finish

Dec 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

16 to 65 years

Sex

Anyone

Healthy volunteers

Accepted

You do not need to have the condition being studied to take part.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Participants 18 (SAD, MAD, OTC in AUS) or 16 (OTC in EU) to 65 years inclusive at time of informed consent * BMI ≥18.0 and ≤32 kg/m2 at screening, and ≤110 kg * Willing and able to sign informed consent form * OTC cohorts: female and must have confirmed heterozygous OTC genotype Exclusion Criteria: * Any significant disease or disorder which, in the opinion of the Investigator, may either put the study participant at risk because of participation in the study, may influence the results of the study, or may affect the study participant's ability to participate in the study * Clinically relevant illness within 7 days before the first dose of study drug * History of intolerance to subcutaneous injection or relevant abdominal scarring * Laboratory results outside normal ranges at screening and judged as clinically relevant by the Investigator for liver function, kidney function, and platelets * Positive viral serology test results for human immunodeficiency virus type 1 or 2 antibodies, hepatitis B surface antigen or hepatitis C virus antibody * Any other safety laboratory result considered clinically significant and unacceptable by the Investigator

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Healthy volunteers are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

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.

Healthy volunteers Otc deficiency

Contacts and locations

Locations

  • Erasmus MC

    Rotterdam, Netherlands

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

    Herston, Queensland, Australia

More trials for these conditions

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