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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT01115582 | Efficacy of To Be Marketed (TBM) Cholic Acid Capsules Used to Treat Children With Inborn Errors of Bile Acid Synthesis | PHASE3 | COMPLETED | 16 | — | — | Apr 1, 2010 | Aug 1, 2010 | Oct 3, 2023 | 1 | United States |
Concentration of serum alanine transaminase (ALT) and aspartate transaminase (AST)
Concentration of bile acids in serum (S) and urine (U). (abbreviations: chol.=cholenoic; monohydro=monohydroxy; dihydro=monohydro)
| Arm | Type | Description |
|---|---|---|
| Cholic Acid Capsule | EXPERIMENTAL | Manufactured cholic acid capsules |
| Name | Type | Description |
|---|---|---|
| Cholic acid | DRUG | The IUPAC name for cholic acid is 3 alpha,7alpha,12 alpha-trihydroxy-5 beta-cholanoic acid. The international nonproprietary name (INN) is cholic acid. Each patient will be given a box containing a 1 month supply of study drug. Each bottle will contain 90 capsules; each capsule will contain either 50 or 250 mg of manufactured cholic acid depending upon the child's weight. The study drug will be taken orally, in divided doses (as determined by the investigator), for a total daily dose of 10-15 mg/kg body weight. Parents of infants and young children who are unable to swallow the TBM cholic acid capsule will be instructed to sprinkle the contents of the capsule over 1-2 teaspoons of plain applesauce and feed it to the child. |
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 curren...