The REPLACE Registry for Cholbam® (Cholic Acid)

Running, not enrolling

Conditions studied: Bile Acid Synthesis Disorders

In brief

This is a prospective, observational, non-interventional patient registry study designed to document product safety and effectiveness outcomes for 10 years in patients treated with Cholbam, including those who have been using Cholbam for at least 30 days (existing users) and those who are first-time initiators of Cholbam.

Key facts

Study ID

NCT03115086

Run by

Mirum Pharmaceuticals, Inc.

People needed

55

Starts

2017-07-10

Expected to finish

2039-07-01

Last updated by the study team

2026-07-31

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Male and female patients, of any age.
• The patient and/or the patient's parent/legal guardian is willing and able to provide signed informed consent, and the patient, if less than 18 years of age, is willing to provide assent as appropriate and in accordance with local regulatory, IRB, and EC requirements.
• The patient has a diagnosis for which Cholbam is indicated.
• The patient is or will be treated with Cholbam at the time of signing the informed consent form (ICF) (enrollment).

You may not qualify if…

• Patients who, by judgement of the Investigator, will not be able to comply with the requirements of the protocol will be excluded

Where it is running

• University of Alabama Birmingham — Birmingham, Alabama, United States
• Stanford School of Medicine — Palo Alto, California, United States
• UC San Francisco — San Francisco, California, United States
• Kidz Pediatric Multispecialty Group — Hollywood, Florida, United States
• Nemours duPont Pediatrics Specialty Clinic — Jacksonville, Florida, United States
• Nicklaus Children's Hospital — Miami, Florida, United States
• Children's Healthcare of Atlanta — Atlanta, Georgia, United States
• Children's Center For Digestive Health — Atlanta, Georgia, United States
• The Community Health Clinic — Topeka, Indiana, United States
• Ochsner Hospital for Children — New Orleans, Louisiana, United States
• University of Minnesota — Minneapolis, Minnesota, United States
• Mayo Clinic — Rochester, Minnesota, United States
• Office of Dr. Esperanza Font-Montgomery MD — Columbia, Missouri, United States
• Washington University at St Louis — St Louis, Missouri, United States
• NYU Langone - NYU Grossman School of Medicine — New York, New York, United States
• Icahn School Of Medicine at Mount Sinai — New York, New York, United States
• Montefiore Medical Center — New York, New York, United States
• Golisano Children's Hospital, URMC — Rochester, New York, United States
• UNC Chapel Hill — Chapel Hill, North Carolina, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• University of Oklahoma — Oklahoma City, Oklahoma, United States
• The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Monroe-Carell Jr. Children's Hospital at Vanderbilt — Nashville, Tennessee, United States
• Primary Children's Hospital — Salt Lake City, Utah, United States

Full record on ClinicalTrials.gov

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