Supporting families of children with Urea Cycle Disorders

The reLiver-1 trial can help pave the way to new UCD therapies

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 reLiver-1 trial can help pave the way to new therapies for the treatment of UCD.

About the Trial

Study trial

The reLiver-1 study is a Phase I clinical trial that uses a non-toxic and non-radioactive tracer to measure ureagenesis capacity in babies up to 1 year of age with a Urea Cycle Disorder. Investigators in this study will use levels of ureagenesis measured in babies without the disorder (normal parameters of ureagenesis function) to compare the range of difference between UCD and non-UCD patients.

The reLiver-1 trial uses a new procedure to measure accurately how much urea your baby’s liver can produce. This procedure consists of giving your baby a diagnostic substance which is a powder (ammonium chloride) diluted in water – called “tracer”. This tracer is non-toxic and non-radioactive.

The use of the tracer in this trial is experimental, but it has been given to adults and children before and was found to be safe and well tolerated.

The information collected in the reLiver-1 trial will help us to better understand Urea Cycle Disorders and develop new therapies. In a subsequent study – reLiver-2, we will investigate the effects of human liver stem cells, administered to babies with a Urea Cycle Disorder, in reducing ammonia concentration and hyperammonaemia crisis and will be evaluated as a bridging treatment.

Study Approval

The reLiver-1 study is reviewed and approved by the Ethic Committees and Medical Authorities, according to the legal regulations.

What Do I Have to Do if My Baby Participates in the reLiver-1 Trial?

If you are interested in having your baby participate, you will be asked to visit the hospital so that the trial doctor can explain the trial details. You will also have the opportunity to ask the doctor any questions about the trial and about the tests and procedures your baby would undergo.

The trial doctor will need to examine your baby to see if this trial is a good fit for them. Before any tests or exams are done, you will need to sign a consent form. The tests and procedures to determine if your baby is eligible for the reLiver-1 trial may take place over several visits, depending on the health of your baby.

For additional information about clinical trials in general please see the FAQ page.

For Babies with a Urea Cycle Disorder

If the doctor decides the trial is right for your baby, you will need to bring them to the clinic for trial procedures during a day-time visit, 4 times with an interval of 12 weeks between each visit, over a 9-month period.

For Babies without a Urea Cycle Disorder

If the doctor decides the trial is right for your baby, you will need to bring them to the clinic for trial procedures during a day-time visit, 2 times with an interval of 12 weeks between each visit, over a 3-month period.

For All Babies

At the end of the trial, you will receive a follow up call – approximately 2 weeks after their final visit. The study doctor will check on how your baby is doing; your baby does not need to be present for this phone call.

It is completely voluntary to participate, and you can decide to withdraw your baby at any time. All trial-related procedures and visits are provided at no cost to you. There will be no financial benefit given to you or your baby for taking part in this trial. You will be reimbursed for costs associated with traveling to the trial visits if you choose to take part in the Travel Assistance Program.