Study to Assess Efficacy, Safety, Tolerability, Pharmacokinetics (PK), and Pharmacodynamics (PD) of Obeticholic Acid (OCA) Compared to Placebo in Pediatric Participants With Biliary Atresia, Post-hepatoportoenterostomy

Launched by INTERCEPT PHARMACEUTICALS · Nov 2, 2023

Keywords

ClinConnect Summary

This clinical trial is studying a medication called Obeticholic Acid (OCA) to see how well it works, how safe it is, and how the body processes it in children with a condition called biliary atresia. Biliary atresia is a liver disease that affects how bile flows from the liver to the intestines. This study is for children who have had a surgical procedure called hepatoportoenterostomy (HPE) to help with this condition. The trial will involve two main phases: first, adjusting the dose of OCA, and then expanding the treatment to different age groups.

To participate, children must be between birth and 17 years old, diagnosed with biliary atresia, and show that their HPE surgery was successful. However, children under 2 years old will only be enrolled after safety data has been reviewed. Certain health conditions may prevent participation, such as previous liver transplants or severe liver issues. Participants will take the medication and be monitored closely to ensure their safety and to evaluate how well the treatment is working. This trial is currently recruiting participants, and it's an important step in finding better treatments for children with biliary atresia.

Gender

ALL

Eligibility criteria

• Inclusion criteria:
• • Male or female pediatric participants from birth to \<18 years old. Note: Participants aged \<2 years old will not be enrolled until after review of safety data during the planned interim analysis and agreement from the Data Safety Monitoring Board (DSMB) that there is sufficient safety data to enroll this age group.
• • Diagnosis of non-syndromic biliary atresia.
• • Demonstrated successful HPE as defined by total bilirubin \<2 milligrams per deciliter (mg/dL) (34.2 micromoles per liter \[μmol/L\]) at least 3 months post-HPE procedure.
• 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:
• • 1. Gastroesophageal varices and/or variceal bleeding
• • 2. Clinically evident ascites related to portal hypertension
• • 3. Hepatic encephalopathy
• • 4. Prior placement of portosystemic shunt
• • 5. Hepatopulmonary syndrome or portopulmonary hypertension
• • 6. Hepatorenal syndrome
• • 7. Any evidence of portal hypertension based on imaging (e.g., cavernous transformation of portal vein, abdominal varices, etc.)
• • 8. Hepatocellular carcinoma
• • 9. Childs-Pugh B or C
• • 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).