CI

At a glance

ClinicalIndex Comparison Record
Phase 3Completed· 85 enrolled
Drug / intervention
Cholic Acidsdrug
Likely dose
Cholic acid 10-15 mg/kg body weight/day orallyAI-extracted
Key inclusion· 5
  • ✓Clinical or biochemical evidence of liver disease, unexplained fat-soluble vitamin malabsorption, or peroxisomal dysfunction that compromises bile acid biosynthesis
  • ✓Infants younger than 3 months of age
  • ✓Children with cholestasis defined as conjugated bilirubin >2 mg/dL or increased serum bile acids
  • ✓Older subjects of any age with cholestatic liver disease if urine screens suggest inborn errors of bile acid metabolism
Key exclusion· 0

None specified.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT00007020
NCT00007020Phase 3Completed
CompareCT.gov

Investigation in the Pathogenesis of Liver Disease in Patients With Inborn Errors of Bile Acid Metabolism

Mirum Pharmaceuticals, Inc.·interventional·Posted Dec 7, 2000·Updated Oct 3, 2023

In Brief

A Phase 3 clinical trial evaluating Cholic Acids for Infantile Refsum's Disease and 4 related conditions. Completed, enrolled 85 participants across 1 site.

Detailed 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

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsInfantile Refsum's Disease, Zellweger Syndrome, Adrenoleukodystrophy, Peroxisomal Disorders, Cholestasis
CountriesUnited States
CollaboratorsChildren's Hospital Medical Center, Cincinnati

Timeline

Phase 3CompletedFinished
199219931994199519961997199819992000200120022003200420052006200720082009201020112012201320142015201620172018201920202021202220232024202520262027
First PostedDec 7, 2000
Enrollment StartJan 1, 1992
Primary CompletionDec 1, 2009
TodayJul 2, 2026
Enrollment to primary: 17.9 yearsPosted 25.6 years ago

Interventions

Cholic Acidsdrug

10-15 mg/kg body weight/day taken orally.