Phase 3
N=85
Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid
Infantile Refsum's Disease · Zellweger Syndrome · Adrenoleukodystrophy · Peroxisomal Disorders · Cholestasis
Bottom Line
View on ClinicalTrials.gov: NCT00007020 ↗Enrolled (actual)
85
Serious AEs
25.3%
Results posted
Jul 2020
Primary outcome: Primary: Number of Participants With Excretion of Atypical Bile Acids in Urine by Category — 10; 51; 11; 9 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Cholic Acids (Drug)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- Mirum Pharmaceuticals, Inc.
- Primary completion
- Dec 2009
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Excretion of Atypical Bile Acids in Urine by Category |
10; 51; 11; 9; 16; 4 | — |
| SECONDARY Change in Liver Function Tests (LFTs) Measured in Serum |
14; 49; 16; 13; 8; 2 | — |
| SECONDARY Liver Histology |
19; 16; 5; 4; 3; 0 | — |
| SECONDARY Height and Weight |
30.1; 44.0; 21.3; 42.3 | — |
| SECONDARY Adverse Events |
79; 38 | — |
Summary
OBJECTIVES:
I. To Evaluate the therapeutic efficacy of cholic acid during provision of compassionate treatment to patients with identified inborn errors of bile acid synthesis and metabolism
II. To assess the safety and tolerability of cholic acid
Eligibility Criteria
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
Clinical or biochemical evidence of liver disease, unexplained fat-soluble vitamin malabsorption, or peroxisomal dysfunction that compromises bile acid biosynthesis
Inclusion criteria for enrollment were:
- Infants 2mg/dl or increased serum bile acids
- Older subjects of any age with cholestatic liver disease if urine screens suggested that they had inborn errors of bile acid metabolism
- Confirmation of a diagnosis of an inborn error of bile acid synthesis based upon urine analysis by FAB-MS to determine whether specific abnormalities in bile acid synthesis are indicated
- The patient and/or parent/legal guardian must have signed the written informed consent document before study start.
- The patient must be willing and able to comply with all study assessments and procedures.
Data sourced from ClinicalTrials.gov (NCT00007020). 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.