Summary

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

Description

Summary

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)

Details

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

Keywords

PFIC1 PFIC2 Pediatric Cholestasis A4250 (odevixibat)

Eligibility

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

Locations

  • 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

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Albireo
ID
NCT03566238
Phase
Phase 3
Study Type
Interventional
Last Updated