Biliary Atresia, Post-hepatoportoenterostomy
Conditions
Brief summary
Time to the first occurrence of any of the following clinical events., Time to first occurrence of: Death, Liver transplant, PELD score ≥17/MELD ≥15, Hospitalization (as defined by a stay of 24 hours or greater) for new onset or recurrence of: Variceal bleed; Hepatic encephalopathy (as defined by a West Haven score of ≥2); Spontaneous bacterial peritonitis (confirmed by diagnostic paracentesis), Clinically evident ascites related to poral hypertension (diuretic-resistant ascites requiring therapeutic paracentesis at a frequency of at least twice in a month)
Detailed description
PK exposure of OCA: Plasma unconjugated OCA (parent), glyco-OCA, tauro-OCA, and total OCA, Biomarkers of hepatobiliary function: GGT, total and direct (conjugated) bilirubin, Biomarkers of FXR activation (PD): Plasma FGF-19, C4, endogenous bile acids, Noninvasive assessment of liver stiffness (if available at site): Transient elastography, Disease progression: Plasma levels of fat-soluble vitamins (D and K), Safety and tolerability of OCA: Treatment-emergent adverse events (TEAEs) including serious adverse events (SAEs), electrocardiogram (ECG), physical exam, clinical laboratory results, and vital signs
Interventions
Sponsors
Intercept Pharmaceuticals Inc.
Eligibility
Sex/Gender
All
Age
0 Years to 64 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Time to the first occurrence of any of the following clinical events., Time to first occurrence of: Death, Liver transplant, PELD score ≥17/MELD ≥15, Hospitalization (as defined by a stay of 24 hours or greater) for new onset or recurrence of: Variceal bleed; Hepatic encephalopathy (as defined by a West Haven score of ≥2); Spontaneous bacterial peritonitis (confirmed by diagnostic paracentesis), Clinically evident ascites related to poral hypertension (diuretic-resistant ascites requiring therapeutic paracentesis at a frequency of at least twice in a month) | — |
Secondary
| Measure | Time frame |
|---|---|
| PK exposure of OCA: Plasma unconjugated OCA (parent), glyco-OCA, tauro-OCA, and total OCA, Biomarkers of hepatobiliary function: GGT, total and direct (conjugated) bilirubin, Biomarkers of FXR activation (PD): Plasma FGF-19, C4, endogenous bile acids, Noninvasive assessment of liver stiffness (if available at site): Transient elastography, Disease progression: Plasma levels of fat-soluble vitamins (D and K), Safety and tolerability of OCA: Treatment-emergent adverse events (TEAEs) including serious adverse events (SAEs), electrocardiogram (ECG), physical exam, clinical laboratory results, and vital signs | — |
Countries
Belgium, France, Germany, Italy, Netherlands, Poland, Spain
Outcome results
None listed