We are studying a new gene therapy to see if it is safe and effective for patients with severe Crigler-Najjar syndrome who need phototherapy. This trial will help us understand how it may improve their health and quality of life.
Health conditions and diseases that the clinical trial is designed to study and treat.
Crigler-Najjar syndrome is a rare genetic disorder that impairs the liver's ability to process bilirubin, leading to dangerously high levels in the blood. Patients often require treatments like phototherapy to reduce bilirubin levels and prevent complications.
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.
