Continuation of cholic acid treatment for people with bile acid disorders who have previously taken part in clinical trials of cholic acid capsules.

• Conditions: Patients with inborn errors of bile acid synthesis and metabolism; MedDRA version: 14.0Level: PTClassification code 10070882Term: Inborn error in primary bile acid synthesisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2011-004528-36-Outside-EU/EEA
• Lead Sponsor: Asklepion Pharmaceuticals, LLC

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2011-10-06
• Last Update Posted: 19 March 2012

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

Patients completing protocol CAC-001-01 or transitioned from protocol 91-10-10 or children with newly diagnosed inborn errors of bile acid synthesis.
Any new, additional infants, children, and adolescents presenting for evaluation of cholestasis defined as a conjugated bilirubin > 2mg/dl or evaluation chronic liver disease, fat and fat soluble vitamin deficiency, growth failure or other signs or symptoms consistent with intestinal intraluminal bile acid deficiency. Patients will have undergone thorough evaluation to define the etiology of cholestasis using conventional screening studies (urine culture a-1-antitrypsin pheno¬type, endocrine studies, STORCH titers, thyroid function tests, liver ultrasound, liver function tests and percutaneous liver biopsy).
Older patients of any age with chronic liver disease if urine screens indicate that they have inborn errors of bile acid metabolism.

Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

None

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified