Biliary atresia
Conditions
Interventions
Single- Dose (SD) Phase: All participants will receive an OCA dose equivalent
to 1.5- mg dose for an adult on Day 1, and on the basis of blood test results
taken after that single OCA dose, the dose
Sponsors
Intercept Pharmaceuticals Inc.
Eligibility
Age
2 Years to 17 Years
Inclusion criteria
Inclusion criteria: 1. Male or female pediatric subjects >=2 to
Exclusion criteria
Exclusion criteria: 1. Prior liver transplant or active status on transplant list. 2. Conjugated (direct) bilirubin >=ULN of site specific reference range. 3. If conjugated bilirubin is not availble: total bilirubin >=2 mg/dL (34.2 µmol/ L). 4. Platelets =1.5. 6. Current or history of complications of decompensated chronic liver disease including: a. high-risk gastroesophageal varices and/or varcieal bleeding b. clinically evident ascites related to portal hypertension c. hepatic encephalopathy d. prior placement of portosystemic shunt e. hepatopulmonary syndrome or portopulmonary hypertension f. hepatorenal syndrome 7. Current intractable pruritus or requires systemic treatment for pruritus within 3 months of Screening (e.g., with bile acid sequestrants or rifampicin) 8. Height and weight Z-score 4x ULN 11. ALT >4x ULN 12. Gamma-glutamyl transferase (GGT) >500 U/L 13. Anticoagulation therapy 14. Albumin
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| - Safety and tolerability: Treatment-emergent AEs (TEAEs) including serious AEs (SAEs), electrocardiogram (ECG), physical exam, clinical laboratory results, vital signs. - PK: Plasma concentrations of unconjugated OCA, its conjugates (glycine conjugate of OCA [glyco-OCA] and taurine conjugate of OCA [tauro-OCA]), and total OCA in the SD and MD Phases | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary Endpoints -PD: Markers of FXR activation: Fibroblast growth factor-19 (FGF-19), 7-hydroxy-4-cholesten-3-one (C4), and endogenous bile acids. - PD: Biomarkers of hepatobiliary function: Alkaline phosphatase (ALP), AST, ALT, GGT, total and direct (conjugated) bilirubin, prothrombin time, INR, albumin, platelet count. Exploratory Endpoints -Acceptability and swallowability: Subject*s ability to swallow the dose regimen and overall acceptability of the dose regimen will be evaluated via a five-point criteria evaluation (refer to the protocol). - Palatability (Day 28): On the first day of the MD Phase, palatability of OCA will be assessed using a five-point facial hedonic scale with a correlated 100-point visual analog scale (FHS/VAS-5) in children >=4 years of age. Children | — |
Countries
Netherlands
Outcome results
None listed