for people ages 6 months to 18 years (full criteria)
at San Francisco, California and other locations
study started
estimated completion
Principal Investigator
Philip Rosenthal



Double blind, randomized, placebo controlled, Phase 3 study to investigate the efficacy and safety of low doses and high doses of A4250 compared to placebo in children with PFIC (deficiencies of familial intrahepatic cholestasis-1 or bile salt export pump).

Official Title

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)


Up to 50 sites in the following countries will take part in this study:

Australia, Belgium, Canada, France, Germany, Israel, Italy, Netherlands, Poland, Spain, Sweden, Turkey, United Kingdom, United States, and Saudi Arabia


PFIC1 PFIC2 Pediatric Cholestasis A4250 (odevixibat)


For people ages 6 months to 18 years

Key Inclusion Criteria:

  • A male or female patient with a clinical diagnosis of PFIC Type 1 or 2 and with a body weight above 5 kg
  • Patient must have clinical genetic confirmation of PFIC-1 or PFIC-2
  • Patient must have elevated s-BA concentration
  • Patient must have history of significant pruritus and a caregiver reported observed scratching in the eDiary
  • Patient and/or legal guardian must sign informed consent (and assent) as appropriate.
  • Patients will be expected to have a consistent caregiver(s) for the duration of the study
  • Caregivers and age-appropriate patients (≥8 years of age) must be willing and able to use an eDiary device as required by the study

Key Exclusion Criteria:

  • Patient with pathologic variations of the ABCB11 gene that predict complete absence of the BSEP protein
  • Patient with past medical history or ongoing presence of other types of liver disease including, but not limited to, the following:
  • Biliary atresia of any kind
  • Benign recurrent intrahepatic cholestasis, indicated by any history of normal s BAs
  • Suspected or proven liver cancer or metastasis to the liver on imaging studies
  • Histopathology on liver biopsy that is suggestive of alternate non-PFIC related etiology of cholestasis
  • Patient with past medical history or ongoing chronic diarrhea
  • Any patient with suspected or confirmed cancers except for basal cell carcinoma
  • Patient with a past medical history of chronic kidney disease with an impaired renal function and a glomerular filtration rate <70 mL/min/1.73 m2

  • Patient with surgical history of disruption of the enterohepatic circulation (biliary diversion surgery) within 6 months prior to start of Screening Period
  • Patient has had a liver transplant or a liver transplant is planned within 6 months of randomization
  • Decompensated liver disease
  • Patient suffers from uncontrolled, recalcitrant pruritic condition other than PFIC
  • Patient who has been previously treated with an IBAT inhibitor whose pruritus has not responded to treatment


  • University of California, San Francisco
    San Francisco California 94143 United States
  • Children's Hospital Los Angeles
    Los Angeles California 90027 United States

Lead Scientist at UCSF


in progress, not accepting new patients
Start Date
Completion Date
Phase 3
Study Type
Last Updated