A study to determine the long term effect and safety of A4250 in treatment of children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2.
- Conditions
- Progressive Familial Intrahepatic Cholestasis Types 1 and 2MedDRA version: 20.0 Level: PT Classification code 10076033 Term: Progressive familial intrahepatic cholestasis System Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2017-002325-38-FR
- Lead Sponsor
- Albireo AB
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- Not specified
- Target Recruitment
- 60
1. Completion of the 24-week Treatment Period of Study A4250-005 or withdrawn from Study A4250-005 due to patient/caregiver judgment of lack of improvement/intolerable symptoms after completing at least 12 weeks of treatment
2. Signed 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 to remain on the study
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
Are the trial subjects under 18? yes
Number of subjects for this age range: 60
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
Patients meeting any of the following criteria at Visit 1 will not be eligible for study participation:
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 barrier protection, hormonal contraception, intra-uterine device, or complete abstinence) throughout the duration of the study and 90 days thereafter (from signed informed consent through 90 days after last dose of study drug).
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
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: The primary objective of this study is to demonstrate a sustained effect of A4250 on serum bile acid (s-BAs) and pruritus in children with PFIC Types 1 and 2.;<br> Secondary Objective: • To evaluate the long-term safety and tolerability of repeated daily doses of A4250<br> • To evaluate the effect of A4250 on growth<br> • To evaluate the effect of A4250 on biliary diversion and/or listing for liver transplantation<br> • To evaluate the effect of A4250 on biochemical markers of cholestasis and liver disease<br> ;<br> Primary end point(s): The primary efficacy endpoint is:<br> Change from baseline in s-BA after 72 weeks of treatment.<br> ;Timepoint(s) of evaluation of this end point: Screening/ Inclusion Visit and week 72
- Secondary Outcome Measures
Name Time Method <br> Secondary end point(s): Secondary efficacy endpoints include:<br> • All-cause mortality, number of patients undergoing biliary diversion<br> surgery, or being listed for liver transplantation.<br> • Change in growth after initiation of A4250 treatment<br> • Change in AST to platelet ratio index (APRI) score and Fib-4 score<br> from baseline to Week 72<br> • Change in pediatric end-stage liver disease (PELD)/model for endstage<br> liver disease (MELD) score from baseline to Week 72<br> • Change in use of antipruritic medication<br> ;Timepoint(s) of evaluation of this end point: • Weeks 24, 48, and 72