Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Albireo
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
Age
0–18
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
A male or female participant with a clinical diagnosis of PFIC Type 1 or 2 and with a body weight above 5 kg
Participant must have clinical genetic confirmation of PFIC-1 or PFIC-2
Participant must have elevated serum bile acid (s-BA) concentration
Participant must have history of significant pruritus and a caregiver reported observed scratching in the eDiary
Participant and/or legal guardian must sign informed consent (and assent) as appropriate.
Participants will be expected to have a consistent caregiver(s) for the duration of the study
Caregivers and age-appropriate participants (≥8 years of age) must be willing and able to use an eDiary device as required by the study
You may not be if
Participant with pathologic variations of the ABCB11 gene that predict complete absence of the bile salt export pump (BSEP) protein
Participant with past medical history or ongoing presence of other types of liver disease including, but not limited to, the following:
1. Biliary atresia of any kind
2. Benign recurrent intrahepatic cholestasis, indicated by any history of normal s BAs
3. Suspected or proven liver cancer or metastasis to the liver on imaging studies
4. Histopathology on liver biopsy that is suggestive of alternate non-PFIC related etiology of cholestasis
Participant with past medical history or ongoing chronic diarrhea
Any participant with suspected or confirmed cancers except for basal cell carcinoma
Participant with a past medical history of chronic kidney disease with an impaired renal function and a glomerular filtration rate \<70 mL/min/1.73 m\^2
Participant with surgical history of disruption of the enterohepatic circulation (biliary diversion surgery) within 6 months prior to start of Screening Period
Participant has had a liver transplant or a liver transplant is planned within 6 months of randomization
Decompensated liver disease
Participant suffers from uncontrolled, recalcitrant pruritic condition other than PFIC
Participant who has been previously treated with an IBAT inhibitor whose pruritus has not responded to treatment
Clareo Health | This Study Will Investigate the Efficacy and Safety of A4250 in Children With PFIC Types 1 or 2
