In short
Clinical trials are investigating AMMONIUM (15N) CHLORIDE in people with urea cycle disorders and in healthy volunteers. The study is looking at safety, tolerability, and other clinical measures in a Phase 1 setting. It also includes participants with an abnormal heterozygous ornithine transcarbamylase (OTC) genotype.
Key points
- AMMONIUM (15N) CHLORIDE is being studied in a Phase 1 clinical trial that focuses on urea cycle disorders. The trial includes healthy volunteers and people with an abnormal heterozygous OTC genotype. Researchers are mainly looking at safety and tolerability. They are also measuring adverse events, vital signs, ECG results, laboratory tests, and physical exams. The study is authorised and plans to enroll 144 participants.
Trial overview
The available trial for AMMONIUM (15N) CHLORIDE is NCT06247670, a Phase 1 study that is authorised and planned to enroll 144 people. It is described as a double-blind, placebo-controlled, single and multiple ascending dose study of CMP-CPS-001 in healthy volunteers and participants with abnormal heterozygous ornithine transcarbamylase (OTC) genotype.
The trial is linked to urea cycle disorders (UCD), which are the conditions being studied in this research. The brief summary says the main purpose is to evaluate the safety and tolerability of CMP-CPS-001 when given subcutaneously, while AMMONIUM (15N) CHLORIDE is one of the listed interventions in the study.
Who can participate
This study includes healthy volunteers and people with an abnormal heterozygous OTC genotype. In simple terms, that means the researchers are studying both people without the disease and people with a specific genetic change related to OTC.
The trial does not list detailed public eligibility rules in the provided data, so the main known target groups are the two groups named in the title.
What the study measures
The primary outcome measures are incidence of adverse events, vital signs, ECG, clinical laboratory assessment, and physical examination. These are standard ways to check whether participants stay safe during a trial and whether any changes happen in the body.
Adverse events are any unwanted medical problems that happen during the study, whether or not they are caused by the treatment. Vital signs, ECG, lab tests, and physical exams help the research team watch for changes in heart function, body systems, and overall health.
Study design and phase
The study is interventional, which means participants receive study treatments as part of the research plan. It is also double-blind and placebo-controlled, meaning neither the participants nor the study team may know who gets the active study treatment or placebo during the trial.
The title also says the study uses single and multiple ascending dose parts. This means the researchers are testing different dose levels over time to learn more about safety in a careful step-by-step way.
AMMONIUM (15N) CHLORIDE is listed among the interventions as an oral drug, while the study also includes sodium chloride for injection, [13C]sodium acetate given orally, and CMP-CPS-001 given subcutaneously.
What this means for patients
For patients and families, this trial is mainly about early research rather than proving that a treatment works in everyday care. The focus is on whether the study approach is safe and tolerable in people with urea cycle disorders and in healthy volunteers.
Because this is a Phase 1 study, the results are usually used to guide later research, not to make final treatment decisions. The data provided do not include final results, so the main information available is the study purpose, the groups being studied, and the safety measures being tracked.