Phase 3
N=16
Efficacy of To Be Marketed (TBM) Cholic Acid Capsules Used to Treat Children With Inborn Errors of Bile Acid Synthesis
Inborn Errors of Bile Acid Synthesis
Bottom Line
View on ClinicalTrials.gov: NCT01115582 ↗Enrolled (actual)
16
Serious AEs
6.3%
Results posted
Dec 2016
Primary outcome: Primary: Serum Transaminases — 31.4; 30.9; 62.7; 65.0 U/L
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
- Aug 2010
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Serum Transaminases |
31.4; 30.9; 62.7; 65.0 | — |
| PRIMARY Serum and Urine Bile Acids |
12.37; 2.762; 14.11; 2.011; 159.85; 19.958 | — |
| SECONDARY Adverse Events |
9 | — |
| SECONDARY Blood Pressure |
106.9; 109.6; 63.9; 65.4 | — |
| SECONDARY Physical Examination |
0; 0 | — |
| SECONDARY Total Bilirubin |
0.35; 0.32 | — |
Summary
This is a study in a small population of children who have inborn errors of bile acid synthesis who are currently taking established doses of the currently used cholic acid capsules prepared at the Cincinnati Children's Hospital Pharmacy. The study is designed to compare the efficacy of these currently used capsules with the efficacy of the same treatment provided in a cholic acid capsule that is made by a company that will be marketed after FDA approval.
At baseline, patients receive established doses of cholic acid capsules prepared at the Cincinnati Children's Hospital Medical Center Pharmacy. During the study, patients receive the same treatment provided in the to-be-marketed (TBM) cholic acid capsule. Hence, patients serve as their own controls, with baseline values presenting the reference value (CCHMC cholic acid capsule) and values after 30 days treatment presenting the value for the investigational treatment (TBM cholic acid capsule).
Eligibility Criteria
Inclusion Criteria
- must have stable transaminase levels within 2 times the upper limits of the normal range.
- must have a diagnosis of an inborn error of bile acid synthesis.
- must have signed the written informed consent/assent document before study start.
- must be currently receiving currently used cholic acid therapy under IND 45,470.
- must be willing and able to comply with all study assessments and procedures.
- must be able to make two visits (Visit 1 and Visit 2) to the study site.
Exclusion Criteria
- is not currently receiving cholic acid therapy for inborn errors of bile acid synthesis under IND 45,470.
- is unable or unwilling to comply with study requirements.
Data sourced from ClinicalTrials.gov (NCT01115582). 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.