An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency

This study is specifically looking for baby boys who are between 24 hours and 7 months old and weigh between 3.5 kg and 13.5 kg. To join, a baby must have a confirmed diagnosis of severe OTC deficiency, meaning they had a very high ammonia level and symptoms shortly after birth. They should also be receiving the usual treatments for OTC deficiency, like a special diet and medications.

There are also some reasons why a baby might not be able to join. For example, babies who have serious birth injuries affecting their brain, or those needing a liver transplant soon, would not be eligible. If a baby has other serious health problems, such as major organ damage, or has already had gene therapy, they also wouldn't be able to take part. The study also needs parents or guardians who can understand and agree to the study requirements.

We don’t have all the details about what participating involves, but generally, studies like this include regular hospital visits for check-ups, blood tests, and other assessments to monitor your baby's health and how the treatment is working. The study drug, ECUR-506, is given once as an infusion into a vein. There will be long-term follow-up to see how your baby is doing after the treatment. The total duration of participation isn't specified here, but gene therapy studies often involve monitoring for several years.