Study of CMP-CPS-001 injection in healthy volunteers and patients with abnormal ornithine transcarbamylase (OTC) genotype for treating urea cycle disorders

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What is this study about?

This clinical trial studies the investigational drug CMP-CPS-001 in people with Urea Cycle Disorders (UCD), specifically those with an abnormal Ornithine Transcarbamylase (OTC) genetic variation. The study includes both healthy volunteers and individuals with this genetic condition. The medication is given as an injection under the skin (subcutaneous injection).

The study uses several compounds to help track how the body processes substances: [13C]Sodium Acetate and 15NH4Cl, which are taken by mouth as solutions. The purpose of this research is to evaluate if CMP-CPS-001 is safe and well-tolerated when given to study participants. Some participants will receive the study medication while others will receive placebo.

During the study, participants will receive either single doses or multiple doses of the medication. The researchers will monitor participants’ health through various medical tests and collect blood and urine samples to understand how the medication works in the body. The study medication will be tested at different dose levels to find the most appropriate dose.

1 Initial screening and evaluation

You will undergo initial health assessment to confirm eligibility for the study

Basic measurements will be taken including your body mass index (BMI) and weight

If you are female, genetic testing will confirm if you have the required heterozygous OTC genotype

2 Study medication administration

You will receive either CMP-CPS-001 or placebo through injection under the skin (subcutaneous injection)

The study follows ascending doses, meaning the dose amount may increase during the study

Some participants will receive single doses while others will receive multiple doses

3 Monitoring and tests

Regular health checks will include vital signs measurements and physical examinations

Blood samples will be taken to measure medication levels in your blood

Urine samples will be collected to measure medication levels

Heart activity will be monitored through ECG (heart rhythm recording)

You will undergo ureagenesis rate tests to measure how your body processes nitrogen

4 Additional study medications

You may receive 15NH4Cl (ammonium chloride) as a powder mixed with water to drink

You may receive [13C]Sodium Acetate as a powder mixed with water to drink

These medications help measure how your body processes nitrogen

5 Study duration

The study is expected to run from August 2025 to July 2026

Your individual participation duration will be determined by your assigned study group

Who Can Join the Study?

Patient must sign a written consent form to participate in the study
Patient must have a Body Mass Index (BMI) between 18.0 and 32.0, and weigh no more than 110 kilograms (BMI is a measure of body fat based on height and weight)
For specific study parts (Module 1 Part C and Module 2), only female patients with confirmed heterozygous OTC genotype can participate (this means having one copy of a specific genetic variation)
For Module 1: Patients must be between 18 and 65 years old when they give consent
For Module 2: Patients must be between 16 and 65 years old when they give consent

Who Cannot Join the Study?

History of liver transplantation or currently on liver transplant waiting list
Presence of active malignancy (cancer) or history of cancer within the past 5 years
Severe kidney problems (creatinine clearance less than 30 mL/min)
Current pregnancy or breastfeeding
Participation in another clinical trial within the past 30 days
Known allergic reactions to similar medications
Severe mental health conditions that could interfere with study compliance
Serious or unstable medical conditions that could affect study participation
Use of prohibited medications within 14 days before starting the study
Blood disorders that could affect safety during the study
History of drug or alcohol abuse within the past year
Unable to follow study procedures or attend scheduled visits
Any condition that study doctors consider unsafe for participation

Where you can join this trial?

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Verified Sites

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Other Sites

Site Name	City	Country	Status
Eonxuma Udsouiebhevk Mixvxqo Cfemghh Rnuezcuzz (sypwvrw Mgg	Rotterdam	The Netherlands

Trial status

Country	Status	Recruitment Start
The Netherlands	Not yet recruiting	15.08.2025

Trial locations

Investigated drugs:

CMP-CPS-001 is an investigational medication administered by subcutaneous injection (under the skin). It is being studied for people with a genetic condition affecting the ornithine transcarbamylase (OTC) enzyme. This medication is being tested to understand how safe it is and how well people tolerate it. The medication is still in early stages of research (Phase 1) and is being tested in both healthy volunteers and people who have abnormal OTC genes.

Investigated diseases:

Urea cycle disorder

Urea Cycle Disorders (UCD) – A group of rare genetic conditions where the body cannot properly process and remove nitrogen from the blood. In these disorders, enzymes needed to break down protein waste are missing or not working correctly. The condition leads to a buildup of ammonia in the blood, which can affect how the body functions. People with UCD may have difficulty processing protein from food. The disorder is present from birth, though symptoms can appear at different ages. The severity of symptoms can vary greatly between individuals.

Trial ID:

2025-520882-37-00

Protocol code:

CPS-101

NCT ID:

NCT06247670

Trial Phase:

Human Pharmacology (Phase I) – First administration to humans

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Study of CMP-CPS-001 injection in healthy volunteers and patients with abnormal ornithine transcarbamylase (OTC) genotype for treating urea cycle disorders

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?