Not yet recruitingRare disease

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

Verified siteInvestigational

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What is this trial about?

A plain-language summary of the goals, design and what participants do

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.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 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
Step 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
Step 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
Step 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
Step 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 trial?

5 criteria

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 trial?

13 criteria

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

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Verified sites

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Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
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Rotterdam, The Netherlands

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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.

What is already known about the treatment

CMP-CPS-001 - A novel investigational drug administered via subcutaneous (SC) injection, currently being studied in clinical trials for the treatment of Urea Cycle Disorders (UCD), particularly focusing on patients with Ornithine Transcarbamylase (OTC) deficiency. This experimental medication is being evaluated for its safety and tolerability in both healthy volunteers and individuals with abnormal OTC genotype. While the exact molecular mechanism is still under investigation through clinical trials, the drug is being developed to address metabolic disorders affecting the body's ability to process nitrogen-containing compounds, which is a key issue in patients with urea cycle disorders.

Investigated diseases

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.