Obeticholic Acid for Biliary Atresia
What You Need to Know Before You Apply
What is the purpose of this trial?
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.
Will I have to stop taking my current medications?
The trial information does not specify if you need to stop taking your current medications. However, if you are on anticoagulation therapy, you cannot participate in the trial.
Is obeticholic acid safe for humans?
How is the drug Obeticholic Acid unique for treating biliary atresia?
What data supports the effectiveness of the drug Obeticholic Acid for treating biliary atresia?
Obeticholic Acid has been shown to be effective in treating primary biliary cholangitis, a liver disease, especially in patients who do not respond well to the standard treatment, ursodeoxycholic acid. This suggests it may have potential benefits for other liver-related conditions, although specific data for biliary atresia is not provided.12579
Who Is on the Research Team?
Lynda Szczech, MD
Principal Investigator
Intercept Pharmaceuticals
Are You a Good Fit for This Trial?
This trial is for children under 18 with non-syndromic biliary atresia who've had a successful hepatoportoenterostomy, indicated by bilirubin levels below 2 mg/dL. Children younger than 2 may join later, pending safety reviews.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Dose Titration
Participants receive OCA or placebo with dose titration every 2 weeks for the first 6 weeks
Age Expansion Treatment
Participants continue at the tolerated dose for approximately 24 months
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Obeticholic Acid
Obeticholic Acid is already approved in United States, European Union for the following indications:
Find a Clinic Near You
Who Is Running the Clinical Trial?
Intercept Pharmaceuticals
Lead Sponsor