Long Term Extension Study of A4250 in Children with PFIC (PEDFIC 2)
Research type
Research Study
Full title
An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2)
IRAS ID
237500
Contact name
Tassos Grammatikopoulos
Contact email
Sponsor organisation
Ipsen Pharma
Eudract number
2017-002325-38
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 4 months, 1 days
Research summary
Progressive Familial Intrahepatic Cholestasis (PFIC) is a chronic liver disease with impaired bile flow and patients’ also experience a severe itch. PFIC exists worldwide and both girls and boys are equally affected.
Approximately half of PFIC patients undergo liver transplants. Many patients also undergo Partial External Biliary Diversion, which is a surgery to divert/drain bile away form the liver to try and improve persistent, treatment resistant
itching.
Currently, the treatment is palliative and there is no medical treatment approved for use in PFIC.
A4250 is a research medicine that is being developed to treat patients with PFIC. A4250 is believed to reduce the absorption of bile acids from the intestine and lower bile acid levels in the blood. A4250 has so far been tested on
healthy volunteers and in a study in children with any of several cholestatic (impaired bile flow) liver diseases, including some with PFIC.
This is a Phase 3, multi-centre, open-label extension study to investigate the long-term effectiveness and safety of a 120 microgramme daily dose of A4250 in children with PFIC Types 1 and 2 who have completed Study A4250-005: A Double-Blind, Randomized, Placebo-Controlled, Phase 3 Study to Demonstrate Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 1).
There are 2 stages to this study: Treatment Period and Follow Up. Approximately 60 participants will take part in the study at about 50 clinics in 15 countries.The study will last for approx 18 months.
Participants will need to have a number of different tests to determine if the study drug works and what side effects there might be, including: physical examinations, blood and urine tests, ultrasounds and vital sign checks.REC name
London - Dulwich Research Ethics Committee
REC reference
18/LO/1585
Date of REC Opinion
2 Nov 2018
REC opinion
Further Information Favourable Opinion