Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC
Study Phase: Phase 3
Recruitment Status: Recruiting
Start Date: September 28, 2018
End Date: December 01, 2021
1. Completion of the 24-week Treatment Period of Study A4250-005 or withdrawn from Study A4250-005 due to patient/caregiver judgment of intolerable symptoms after completing at least 12 weeks of treatment
2. Signed informed consent and assent as appropriate
3. Patients expected to have a consistent caregiver for the duration of the study
4. Caregivers (and age appropriate patients) must be willing and able to use an eDiary device as required by the study Inclusion Criteria Cohort 2:
1. A male or female patient with a clinical diagnosis of PFIC and with a body weight ≥5 kg
2. Patient must have clinical genetic confirmation of PFIC
3. Patient must have elevated serum bile acid levels
4. Patient must have history of significant pruritus
5. Age appropriate patients are expected to have a consistent caregiver for the duration of the study
6. Caregivers and age-appropriate patients (≥8 years of age) must be willing and able to use an eDiary device as required by the study Exclusion Criteria Cohort 1:
1. Decompensated liver disease: coagulopathy, history, or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy
2. Sexually active males and females who are not using a reliable contraceptive method with ≤1% failure rate (such as hormonal contraception, intra-uterine device, or complete abstinence) throughout the duration of the study and 90 days thereafter
3. Patients not compliant with treatment in study A4250-005
4. Any other conditions or abnormalities which, in the opinion of the investigator or Medical Monitor, may compromise the safety of the patient, or interfere with the patient participating in or completing the study Exclusion Criteria Cohort 2:
1. Known pathologic variations of the ABCB11 gene that have been demonstrated to result in 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:
1. Biliary atresia of any kind
2. Benign recurrent intrahepatic cholestasis, indicated by any history of normal serum bile acids
3. Suspected or proven liver cancer or metastasis to the liver on imaging studies
4. Histopathology on liver biopsy is suggestive of alternate non-PFIC related etiology of cholestasis
3. Patient with past medical history or ongoing chronic (i.e., >3 months) diarrhea
4. Any patient with suspected or confirmed cancers except for basal cell carcinoma
5. Patient has had a liver transplant, or a liver transplant is planned within 6 months of the Screening/Inclusion Visit
6. Decompensated liver disease
7. Patient suffers from uncontrolled, recalcitrant pruritic condition other than PFIC
8. Patient previously treated with an IBAT inhibitor and whose pruritus did not respond to treatment
9. Sexually active males and females who are not using a reliable contraceptive method with ≤1% failure rate (such as hormonal contraception, intra-uterine device, or complete abstinence) throughout the duration of the study and 90 days thereafter
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Conditions:
- Cholestasis
- Cholestasis, Intrahepatic