Status:
COMPLETED
Open Label, Continuation Study of Cholic Acid in Subjects With Inborn Errors of Bile Acid Synthesis
Lead Sponsor:
Mirum Pharmaceuticals, Inc.
Conditions:
Bile Acid Synthesis Defect
Eligibility:
All Genders
Phase:
PHASE3
Brief 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.
Detailed Description
This is a Phase 3, open-label, single center, nonrandomized study. This continuation protocol will consist of eligible subjects who have previously received cholic acid through the Cincinnati Children...
Eligibility Criteria
Inclusion
- 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.
- The subject and/or parent/legal guardian must have provided informed consent prior to study start.
- The subject must have a diagnosis of an inborn error of bile acid synthesis.
- The subject must be willing and able to comply with all study assessments and procedures.
- Subjects with other organ dysfunction will not be excluded
Exclusion
Key Trial Info
Start Date :
January 1 2010
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 1 2016
Estimated Enrollment :
53 Patients enrolled
Trial Details
Trial ID
NCT01438411
Start Date
January 1 2010
End Date
July 1 2016
Last Update
October 3 2023
Active Locations (1)
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1
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229