An Open-Label, Phase 3 Study to Evaluate the Efficacy and Safety of TAK-625 in the Treatment of Subjects With Progressive Familial Intrahepatic Cholestasis
The main aim of the study is to check if TAK-625 improves symptoms of Progressive Familial Intrahepatic Cholestasis (PFIC), side effect from the study treatment or TAK-625, and how much TAK-625 stays in their blood over time. This will help the study sponsor (Takeda) to work out the best dose to give people in the future. The participants will be treated with TAK-625 for up to the end of study (about 34 months). Participants will visit their study clinic 15 times from the start of study. After 15 times visits, participants will visit their study clinic every 12 weeks up to the end of study.
• The participant is Japanese male or female with a body weight \>=3.0 kg and who is \>=1 month of age at the time of informed consent.
• The participant has a cholestasis as manifested by total serum bile acid (sBA) \>=3\^ upper limit of the normal range (ULN) (applies to the primary cohort only).
• The participant has an average morning ItchRO (Obs) score \>=1.5 during 4 consecutive weeks of the screening period, leading to the baseline visit (Week 0/Visit 2). Since it is difficult to evaluate pruritus in infants, participants \<12 months of age at screening whose pruritus is unavoidably difficult to be evaluated are not necessarily required to meet the above score.
• The caregiver has completed at least 21 valid\* morning ItchRO (Obs) entries during 4 consecutive weeks of the screening period, leading to the baseline visit (Week 0/Visit 2) (\*valid=completed and not answered as I don't know; the maximum allowed invalidreports=7, no more than 2 invalid reports during the last 7 days before the baseline visit \[Week 0/Visit 2\]).
• The participant has a diagnosis of progressive familial intrahepatic cholestasis (PFIC) based on:
• Chronic cholestasis as manifested by persistent (\>6 months\*) pruritus in addition to biochemical abnormalities and/or pathological evidence of progressive liver disease. (\* =\<6 months is acceptable for participants \<12 months of age).
• AND
• For Primary cohort:
• a) The participant has a genetic testing result consistent with disease-causing variation in ABCB11 (PFIC2), based on a genotyping.
• For Supplemental cohort:
⁃ The participant has a genetic testing results consistent with disease causing variation in ATP8B1 (PFIC1), ABCB4 (PFIC3), or tight junction protein 2 gene (TJP2) (PFIC4), based on a genotyping.
⁃ The participant has a PFIC phenotype without a known mutation or with another known mutation not described above.
⁃ The PFIC participant has internal or external biliary diversion surgery history, and the internal or external biliary diversion surgery was reversed.
• The participant (whenever possible) and caregiver are able to be contacted by phone for scheduled remote visits (participant contacts \[phone calls\]).
• Both a caregiver and participant above the age of assent are capable of reading and understanding the questionnaires.
• The same caregiver should be contacted during this study. The ItchRO (Obs) should be completed by the same caregiver for consistency during this study, even if the participant is an adult (over 18 years old).