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Also known as Cholic, Cholic Acid, Cholic Acid Capsules
Cholic Acids · 3 trials · 7 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.
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 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. |
| Cholic Acids | DRUG | 10-15 mg/kg body weight/day taken orally. |
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 Acids is an investigational small molecule being developed for Infantile Refsum's Disease, a rare inherited disorder. It is also being studied in related conditions including Zellweger Syndrome, Adrenoleukodystrophy, Peroxisomal Disorders, and Cholestasis. The drug is currently in Phase 3 clinical development.
Cholic Acids is being developed by Mirum Pharmaceuticals, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol MIRM. The company is focused on rare disease therapies and is advancing Cholic Acids through clinical trials for Infantile Refsum's Disease.
Cholic Acids is in Phase 3 clinical development. It is an investigational drug and has not been approved by the FDA. The drug is being studied for Infantile Refsum's Disease and related peroxisomal disorders, with one completed Phase 3 trial supporting its development.
Cholic Acids has one completed clinical trial, NCT00007020, titled "Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid." This Phase 3 study enrolled 85 participants with conditions including Infantile Refsum's Disease, Zellweger Syndrome, Adrenoleukodystrophy, Peroxisomal Disorders, and Cholestasis.
Cholic Acids is an investigational drug and is not FDA approved. It is currently in Phase 3 clinical development for Infantile Refsum's Disease. The drug has completed one clinical trial, but approval status has not been established.