Unicyte AG, the sponsor of this trial, will use this information in other clinical trials to help them develop stem cell treatment. Information from the reLiver-1 trial will help determine if the stem cell therapy is working or not. This trial may help develop an accepted way to define UCD disease severity. It may also help to decide the best treatment options for babies with UCD. About Urea Cycle
Disorders:
Urea Cycle Disorders (UCDs) are very rare, inherited diseases that affect approximately 140 newborns in Europe each year. Current therapies have improved the care of Urea Cycle Disorders, but it remains a very severe and serious disease. The Urea Cycle plays an important role in how humans process protein and remove ammonia from the blood stream. When protein is eaten, the body breaks it down into amino acids, which are used to help the body grow and repair tissue. Any parts of the amino acids not used are turned into a waste product called ammonia. Ammonia is removed in the Urea Cycle, where enzymes in the liver turn the ammonia into urea, which then leaves the body in the urine. In Urea Cycle Disorders one of these six enzymes is not working correctly. If this happens, the ammonia cannot leave the body, which causes high ammonia levels in the blood. Babies and older patients with UCD are either missing, or do not make enough of, one of these six enzymes. This means they cannot process waste from protein correctly, and ammonia builds up in the blood stream. Ammonia is highly toxic and can cause very serious problems, such as coma or even death. There are therapies for patients with a Urea Cycle Disorder, but there is no cure for Urea Cycle Disorders. These treatments reduce high ammonium concentrations in the blood in combination with a low protein diet, but they do not always work and babies can still suffer from high ammonia levels in the blood. Liver transplantation can also be used as a treatment but cannot be performed in newborns.
