Status:
TERMINATED
Obeticholic Acid in Pediatric Subjects With Biliary Atresia
Lead Sponsor:
Intercept Pharmaceuticals
Conditions:
Biliary Atresia
Eligibility:
All Genders
2-17 years
Phase:
PHASE2
Brief Summary
This is a Phase 2, multicenter, open-label, single dose and multi-dose, dose-finding study with an optional open-label extension (OLE) to assess the safety, tolerability, and pharmacokinetics of obeti...
Eligibility Criteria
Inclusion
- Key
- Male or female pediatric subjects ≥2 to \<18 years old
- Diagnosis of biliary atresia
- Demonstrated successful HPE (also known as Kasai portoenterostomy) as defined by total bilirubin \<2 mg/dL (34.2 μmol/L) at least 3 months post-HPE procedure.
- Able to swallow tablets (ie, tablet or mini-tablet formulation)
- Key
Exclusion
- Prior liver transplant or active status on transplant list
- Conjugated (direct) bilirubin ≥ULN of site specific reference range
- If conjugated bilirubin is not available: total bilirubin ≥2 mg/dL (34.2 μmol/L)
- Platelets \<150,000/μL
- INR ≥1.5
- Current or history of complications of decompensated chronic liver disease including:
- high-risk gastroesophageal varices and/or variceal bleeding
- clinically evident ascites related to portal hypertension
- hepatic encephalopathy
- prior placement of portosystemic shunt
- hepatopulmonary syndrome or portopulmonary hypertension
- hepatorenal syndrome
- Current intractable pruritus or requires systemic treatment for pruritus within 3 months of Screening (e.g., with bile acid sequestrants or rifampicin)
- Height and weight Z-score \<-2 per site specific ranges
- Acholic (pale) stools
- AST \>4x ULN
- ALT \>4x ULN
- GGT \>500 U/L
- Anticoagulation therapy
- Albumin \<3.5 g/dL
- Ongoing current cholangitis
- Choledochal cystic disease
- Renal disease defined as serum creatinine \>ULN for subject's age, prior to enrollment
Key Trial Info
Start Date :
July 1 2015
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 9 2023
Estimated Enrollment :
7 Patients enrolled
Trial Details
Trial ID
NCT05321524
Start Date
July 1 2015
End Date
March 9 2023
Last Update
March 28 2023
Active Locations (17)
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1
Clinques University Saint-Luc
Brussels, Belgium, 1200
2
CHU Lille
Lille, ME, France, 59037
3
Hopital de la Timone
Marseille, PACA, France, 13385
4
APHP- Hopital Necker Enfants Malades
Paris, France, 75015