A study to determine if A4250 is safe and can be used to treat children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2.

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 60

Inclusion Criteria

1. A male or female patient, with clinical diagnosis of PFIC Type 1 or 2, between the ages of =6 months and =18 years at Visit 1 with a body weight above 5 kg
2. Patient must have clinic genetic confirmation of PFIC-1 or PFIC-2 through identification of biallelic pathogenic variants in either the ATP8B1 or ABCB11 genes
3. Patient must have elevated s-BA concentration, specifically measured to be =100 µmol/L, taken as the average of 2 samples at least 7 days apart (Visits 1 and 2) prior to randomization
4. Patient must have history of significant pruritus and a caregiver-reported observed scratching in the eDiary in the 2 weeks prior
to randomization
5. Patient and/or legal guardian must sign informed consent (and assent) as appropriate.
Patients who turn 18 years of age (or legal age per country) during the study will be required to re-consent in order to remain in the study
6. Patients will be expected to have a consistent caregiver for the duration of the study
7. Caregivers and age-appropriate patients (=8 years of age) must be willing and able to use an eDiary device as required by the study
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 2
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Patient with pathologic variations of the ABCB11 gene that predict complete absence of the BSEP protein
2. Patient with past medical history or ongoing presence of other types of liver disease including, but not limited to, the following:
a) Biliary atresia of any kind
b) Benign recurrent intrahepatic cholestasis
c) Suspected or proven liver cancer or metastasis to the liver on imaging studies
d) Histopathology on liver biopsy is suggestive of alternate non-PFIC related etiology of cholestasis
3. Patient with past medical history of ongoing chronic diarrhea
4. Any patient with suspected or confirmed cancers except for basal cell carcinoma
5. 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
6. Patient with surgical history of disruption of the enterohepatic circulation excluding those who have undergone a successful reversal procedure that has permanently restored flow of bile acids from the liver to the duodenum
7. Patient has had a liver transplant or a liver transplant is planned within 6 months of randomization
8. Decompensated liver disease, coagulopathy, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy
9. Patient suffers from uncontrolled, recalcitrant pruritic condition other than PFIC.
10. Patient who has been previously treated with an IBAT inhibitor whose pruritus has not responded to treatment

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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