Phase 3
N=53
Open Label, Continuation Study of Cholic Acid in Subjects With Inborn Errors of Bile Acid Synthesis
Bile Acid Synthesis Defect
Bottom Line
View on ClinicalTrials.gov: NCT01438411 ↗Enrolled (actual)
53
Serious AEs
35.9%
Results posted
Aug 2020
Primary outcome: Primary: Change in Atypical Urinary Bile Acid Excretion by FAB-MS (Fast-Atom-Bombardment Ionization-Mass Spectrometry) — 25; 6; 10; 10 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Cholic Acid (Drug)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- Mirum Pharmaceuticals, Inc.
- Primary completion
- Jul 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in Atypical Urinary Bile Acid Excretion by FAB-MS (Fast-Atom-Bombardment Ionization-Mass Spectrometry) |
25; 6; 10; 10; 25; 11 | — |
| SECONDARY Evaluation of Serum Transaminases: ALT |
27; 9; 7; 5; 20; 15 | — |
| SECONDARY Evaluation of Serum Transaminases: AST |
17; 17; 5; 8; 15; 17 | — |
| SECONDARY Clinical Laboratory Results: Bilirubin |
3.678; 3.785 | — |
| SECONDARY Clinical Laboratory Results: Gamma Glutamyl Transferase (GGT) |
38.8; 16.4 | — |
| SECONDARY Clinical Laboratory Results: Alkaline Phosphatase |
293.7; 39.6 | — |
| SECONDARY Clinical Laboratory Results: Prothrombin Time |
17.977; 1.045 | — |
| SECONDARY Physical Examinations: Height |
26.767; 25.540 | — |
| SECONDARY Physical Examinations: Body Weight |
38.030; 33.227 | — |
| SECONDARY Incidence of Adverse Events |
44 | — |
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.
Eligibility 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.
- 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
Data sourced from ClinicalTrials.gov (NCT01438411). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.