Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT00007020 | Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid | PHASE3 | COMPLETED | 85 | — | — | Jan 1, 1992 | Dec 1, 2009 | Oct 3, 2023 | 1 | United States |
Patients with excretion of atypical bile acids in urine by category, from worst status before treatment (baseline, BL) to best status on treatment (OT)
| Arm | Type | Description |
|---|---|---|
| Cholic Acid | OTHER | - |
| Name | Type | Description |
|---|---|---|
| Cholic Acids | DRUG | 10-15 mg/kg body weight/day taken orally. |
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 \< age 3 months * Children p...