We are investigating a new treatment called ECUR-506 for boys under 9 months with neonatal onset OTC deficiency. The study will help us understand its safety and how well it works.
Health conditions and diseases that the clinical trial is designed to study and treat.
Ornithine Transcarbamylase Deficiency (OTC) is a rare genetic disorder that impairs the body's ability to remove ammonia from the bloodstream, leading to potentially serious health issues. Symptoms can include lethargy, vomiting, and confusion, especially after high-protein meals.
These questions help us understand your situation so we can let the trial team know whether you might be a potential match. This is not a medical evaluation and is not part of the official screening, the study doctor will make the final decision..
Experimental substances that are being investigated in this clinical trial. These are not yet approved for general use.
Don't worry if you don't know the medicines tested in the trial. There is a chance you have heard about the similar medicines. Check the list below to see if you are familiar with any of them.
Locations and medical facilities where this clinical trial is currently recruiting or conducting research. Select the locations where you would like to participate.
Disclaimer: Parts of this content have been automatically extracted from the EU Clinical Trials registry. While we strive for accuracy, please always contact the trial site or sponsor directly for correct and official information before making any decisions about participation. View on EU Clinical Trials.