Open Label, Continuation Study of Cholic Acid in Subjects With Inborn Errors of Bile Acid Synthesis

• Status: Completed
• Trial end date: 2016-07-01
• Target enrollment: 0
• Participant gender: All

Summary

The primary purpose of the study is to evaluate the therapeutic efficacy and safety of cholic acid in subjects with identified inborn errors of bile acid synthesis.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Retrophin, Inc.; Travere Therapeutics, Inc.
• Treatments: Bile Acids and Salts; Cholic Acids

Criteria

Inclusion Criteria:

Subjects who received cholic acid through CCHMC protocols 91-10-10 or CAC-002-01 and meet the following criteria are eligible for study participation.

1. The subject and/or parent/legal guardian must have provided informed consent prior to study start.

2. The subject must have a diagnosis of an inborn error of bile acid synthesis.

3. The subject must be willing and able to comply with all study assessments and procedures.

4. Subjects with other organ dysfunction will not be excluded