Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Cholic Acid · 2 trials · 2 indications
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)
The level of atypical urinary bile acid secretion was scored using a scale of: 0, normal; 1, slight; 2, significant; or 3, marked. A Cochran-Mantel-Haenszel (CMH) test with modified ridit scoring was used to compare the difference between the score at baseline and the worst post-baseline score during treatment with cholic acid in this single-arm trial.
| Arm | Type | Description |
|---|---|---|
| Cholic Acid Capsule | EXPERIMENTAL | Manufactured cholic acid capsules |
| Cholic Acid | OTHER | Active drug |
| 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...
Cholic Acid is used for inborn errors of bile acid synthesis and bile acid synthesis defect, which are rare diseases affecting bile acid production. It is a small molecule therapy being developed by Mirum Pharmaceuticals, Inc. (MIRM) and is currently in Phase 3 clinical development.
Cholic Acid is a bile acid replacement therapy. It works by providing exogenous cholic acid to compensate for the deficient bile acid synthesis in patients with inborn errors of bile acid synthesis or bile acid synthesis defect, thereby restoring normal bile acid function.
Cholic Acid is being developed by Mirum Pharmaceuticals, Inc., a biopharmaceutical company. The company's stock ticker is MIRM. Mirum is conducting clinical trials to evaluate the efficacy and safety of Cholic Acid in patients with rare bile acid synthesis disorders.
Cholic Acid is in Phase 3 clinical development. It is an investigational drug and has not been approved by regulatory authorities. The Phase 3 trials have been completed, and the drug is being evaluated for the treatment of inborn errors of bile acid synthesis and bile acid synthesis defect.
Cholic Acid has been studied in two completed Phase 3 clinical trials. The first, NCT01115582, enrolled 16 participants with inborn errors of bile acid synthesis. The second, NCT01438411, was an open-label continuation study enrolling 53 participants with bile acid synthesis defect. Both trials were conducted in the United States.
Cholic Acid is a specific bile acid used for replacement therapy in patients with bile acid synthesis defects. It is distinct from other bile acid products, such as chenodeoxycholic acid or ursodeoxycholic acid, which have different chemical structures and clinical uses. Cholic Acid is being developed specifically for rare inborn errors of bile acid synthesis.