Status and phase
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About
This study will evaluate the efficacy, safety and tolerability, as well as PK/PD of OCA in eligible pediatric participants with biliary atresia with successful hepatoportoenterostomy (HPE, also known as a Kasai portoenterostomy). The double-blind period comprises of 2 phases: dose titration phase and age expansion treatment phase.
Enrollment
Sex
Ages
Volunteers
Inclusion criteria
Exclusion criteria
Prior liver transplant or active status on transplant list.
Participants diagnosed with biliary atresia splenic malformation (BASM).
Conjugated (direct) bilirubin ≥ upper limit of normal (ULN) of site-specific reference range. If conjugated bilirubin is not available: total bilirubin ≥2 mg/dL (34.2 mol/L).
Platelets <120,000/μL
International normalized ratio (INR) ≥1.5.
Current or history of complications of decompensated chronic liver disease including:
Height and weight Z-score <-2 per site-specific reference ranges.
Acholic (pale) stools.
Aspartate aminotransferase (AST) >4x ULN.
Alanine aminotransferase >4x ULN
GGT >500 Units per Liter (U/L)
On anticoagulation therapy
Albumin <3.5 grams per deciliter (g/dL).
Inability to swallow tablets (i.e., tablet or mini-tablet formulations).
Primary purpose
Allocation
Interventional model
Masking
144 participants in 2 patient groups, including a placebo group
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Central trial contact
Scott Birnbaum
Data sourced from clinicaltrials.gov
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