Progressive familial intrahepatic cholestasis trial

Clinical trial for children with progressive familial intrahepatic cholestasis delivers encouraging results

Hands holding sample bottle and syringe in lab

A multinational clinical trial, led by King’s College Hospital honorary consultant in paediatric hepatology, Prof Richard Thompson, and sponsored by Albireo Pharma, has delivered extremely encouraging results for children living with progressive familial intrahepatic cholestasis (PFIC).

PFIC is a genetic condition which is caused by mutations in genes - first identified by Prof Thompson -that produce key proteins involved in the secretion of bile from the liver. Instead of being secreted, the bile acids build up in liver cells and blood of those affected, resulting in severe liver disease. A key symptom of the condition is severe itching, with many children with PFIC going on to require a liver transplant due to chronic liver disease, cirrhosis and liver cancer.

In this world-first randomised placebo-controlled Phase III trial in PFIC, named PEDFIC1, investigators tested the effects of the experimental drug Odevixibat in children with PFIC. Firstly, the drug was noted to be well tolerated with minimal side effects and few children experienced diarrhoea, an anticipated complication of other PFIC treatments.

Compared to those receiving a placebo, children with PFIC that were given Odevixibat had significantly reduced itching as well as a marked reduction in bile acid in the blood. By tackling the elevated bile acids which are at the heart of PFIC, investigators are also hopeful that this drug could go some way to reduce the number of children needing liver transplants. Following these exciting results, Albireo Pharma is now looking to submit the drug for approval by regulatory agencies in both the US and Europe.

Study lead Professor Richard Thompson said, “I am really pleased that the study has produced the results that we hoped for. The drug looks like it is capable of changing the natural history of the disease, not just improving the symptoms. Both are really important to the children and their families.”

Congratulations on all those involved in the trial, in particular Chief Investigator Prof Richard Thompson, King’s Primary Investigator, Dr Tassoss Grammatikopoulos and King’s Paediatric Research Nurse, Katie Tupper.