Cholic Acid
- Generic Name
- Cholic Acid
- Brand Names
- Cholbam, Orphacol
- Drug Type
- Small Molecule
- Chemical Formula
- C24H40O5
- CAS Number
- 81-25-4
- Unique Ingredient Identifier
- G1JO7801AE
- Background
A major primary bile acid produced in the liver and usually conjugated with glycine or taurine. It facilitates fat absorption and cholesterol excretion. [PubChem]
Cholic acid, formulated as Cholbam capsules, is approved by the United States Food and Drug Administration as a treatment for children and adults with bile acid synthesis disorders due to single enzyme defects, and for peroxisomal disorders (such as Zellweger syndrome).
- Indication
Oral cholic acid is indicated for: treatment of bile acid synthesis disorders due to single enzyme defects; and as adjunctive treatment of peroxisomal disorders including Zellweger spectrum disorders in patients who exhibit manifestations of liver disease, steatorrhea or complications from decreased fat soluble vitamin absorption.
- Associated Conditions
- Inborn Errors of Bile Acid Synthesis, Zellweger Spectrum Disorder, Peroxisomal disorders (PDs)
- Associated Therapies
- -
Smith-Lemli-Opitz Syndrome and Cholic Acid
- First Posted Date
- 2018-10-26
- Last Posted Date
- 2024-04-30
- Lead Sponsor
- University of Nebraska
- Target Recruit Count
- 12
- Registration Number
- NCT03720990
- Locations
- 🇺🇸
Colorado Children's Hospital, Aurora, Colorado, United States
🇺🇸University of Nebraska Medical Center, Omaha, Nebraska, United States
The REPLACE Registry for Cholbam® (Cholic Acid)
- First Posted Date
- 2017-04-14
- Last Posted Date
- 2023-10-02
- Lead Sponsor
- Mirum Pharmaceuticals, Inc.
- Target Recruit Count
- 55
- Registration Number
- NCT03115086
- Locations
- 🇺🇸
The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
🇺🇸Kidz Pediatric Multispecialty Group, Hollywood, Florida, United States
🇺🇸Nemours duPont Pediatrics Specialty Clinic, Jacksonville, Florida, United States
Open Label, Continuation Study of Cholic Acid in Subjects With Inborn Errors of Bile Acid Synthesis
- First Posted Date
- 2011-09-22
- Last Posted Date
- 2023-10-03
- Lead Sponsor
- Mirum Pharmaceuticals, Inc.
- Target Recruit Count
- 53
- Registration Number
- NCT01438411
- Locations
- 🇺🇸
Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, United States
Efficacy of To Be Marketed (TBM) Cholic Acid Capsules Used to Treat Children With Inborn Errors of Bile Acid Synthesis
- First Posted Date
- 2010-05-04
- Last Posted Date
- 2023-10-03
- Lead Sponsor
- Mirum Pharmaceuticals, Inc.
- Target Recruit Count
- 16
- Registration Number
- NCT01115582
- Locations
- 🇺🇸
Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, United States
Cholic Acid for Hepatic Steatosis in Lipodystrophy
- First Posted Date
- 2007-04-06
- Last Posted Date
- 2019-01-17
- Lead Sponsor
- University of Texas Southwestern Medical Center
- Target Recruit Count
- 18
- Registration Number
- NCT00457639
- Locations
- 🇺🇸
University of Texas Southwestern Medical Center, Dallas, Texas, United States
Study of Bile Acids in Patients With Peroxisomal Disorders
- Conditions
- First Posted Date
- 1999-10-19
- Last Posted Date
- 2015-03-25
- Lead Sponsor
- University of Cincinnati
- Target Recruit Count
- 25
- Registration Number
- NCT00004442
Drug Development Updates
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