Albireo Pharma has commenced patient enrolment in Phase III PEDFIC-1 clinical trial of A4250 for the treatment of patients with progressive familial intrahepatic cholestasis (PFIC).

The single, randomised, placebo-controlled trial aims to enrol around 60 PFIC patients, aged six months to 18 years, who have elevated serum bile acid (sBA) levels and pruritus.

The open-label extension trial will also evaluate long-term safety and durability of response of A4250.

During the trial, patients will receive a 40μg/kg or 120μg/kg oral dose of A4250 or placebo once daily for 24 weeks.

“The results of the Phase II trial gives us optimism and confidence that A4250 could be an excellent medical treatment option.”

The trial’s primary endpoint for the US Food and Drug Administration (FDA) will be an evaluation of change in pruritus, while the primary endpoint for the European Medicines Agency (EMA) will be sBA responder rate.

It will enrol patients in the US, Canada, Europe, the Middle East and Australia.

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PEDFIC-1 trial principal investigator Richard Thompson said: “The results of the Phase II trial, where A4250 reduced serum bile acids and decreased pruritus in most patients, while showing a favourable overall tolerability profile, gives us optimism and confidence that A4250 could be an excellent medical treatment option.”

A4250 is an investigation product that seeks to treat rare paediatric cholestatic liver diseases.

It is an ileal bile acid transporter (IBAT) inhibitor currently being studied to prevent PFIC, which causes progressive, life-threatening liver disease, and in many cases it leads to cirrhosis and liver failure within the first ten years of life.

There is currently no approved pharmacological treatment available for PFIC.