15N ammonium chloride ureagenesis validation clinical trial
Research type
Research Study
Full title
An open-label, controlled, multi-site, Phase I clinical trial to assess the ureagenesis capacity in newborns and infants up to the age of 12 months with neonatal and infantile onset of urea cycle disorders (UCD) using a 15N ammonium chloride tracer compared to newborns and infants without UCD.
IRAS ID
300474
Contact name
Colin Buck
Contact email
Sponsor organisation
Unicyte AG
Eudract number
2021-000824-36
Duration of Study in the UK
2 years, 0 months, 29 days
Research summary
The urea cycle is the mechanism by which waste nitrogen, formed from the breakdown of proteins, is cleared from the body. In a healthy person, enzymes in the liver convert nitrogen into urea, which is then removed from the body in urine. People born with urea cycle disorders (UCDs) are missing an enzyme that enables this conversion, and as a result ammonia builds up in the bloodstream. High amounts of ammonia can cause coma and death. Children who survive often have brain damage and severe developmental delay as a result of the high ammonia levels.
UCDs are rare diseases, and there are limited treatment options available. Unicyte AG are developing a liver stem cell therapy to provide treatment options for the future.
The first step in the development of this therapy is to gain a better understanding of the way ureagenesis (the creation of urea) varies naturally in young children with UCD, as well as gaining a better understanding of the disease progression over time.
This study will use a non-radioactive, stable isotope of nitrogen (15N ammonium chloride tracer) to measure ureagenesis capacity in children with UCD over a period of nine months. The same tracer will be given to children without UCD over a period of three months to allow a comparison to be made.
To take part in this study, children must be 12 months or younger. Up to twenty children with UCD and 5-10 children without UCD are expected to take part. The children will receive physical and neurological examinations, and blood samples will be taken to analyse the ureagenesis.
The information from this study will be used in Unicyte’s next clinical study, to evaluate if their stem cell treatment is working. In the future, this study may contribute to establishing this tracer as an accepted way to quantify UCD disease severity. It may also help to determine the best treatment options for children with UCD.
REC name
London - London Bridge Research Ethics Committee
REC reference
21/LO/0585
Date of REC Opinion
27 Oct 2021
REC opinion
Further Information Favourable Opinion