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Can a decade of real-world data refine treatment for rare bile acid disorders?
NCT ID NCT03115086
First seen Jul 31, 2026 · Last updated Jul 31, 2026
Summary
This study is a patient registry that will follow people of any age with bile acid synthesis disorders who are treated with Cholbam (cholic acid). The goal is to collect information over 10 years on how the drug performs in routine clinical care, including its safety, effectiveness, and impact on growth and vitamin levels. Participants include both those already taking Cholbam and those starting it for the first time. The registry will track outcomes like cholestasis, fat-soluble vitamin deficiencies, growth failure, and any side effects.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Cholbam (cholic acid)
- What this could lead to
- If successful, this registry could provide long-term safety and effectiveness data to better guide treatment for people with bile acid synthesis disorders.
- What could go wrong
- This is an observational registry, not a controlled trial, so it cannot prove cause and effect. Results depend on real-world data, which may vary and take years to accumulate.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Children's Center For Digestive Health
Atlanta, Georgia, 30342, United States
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Children's Healthcare of Atlanta
Atlanta, Georgia, 30329, United States
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
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Golisano Children's Hospital, URMC
Rochester, New York, 14642, United States
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Icahn School Of Medicine at Mount Sinai
New York, New York, 10029, United States
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Kidz Pediatric Multispecialty Group
Hollywood, Florida, 33021, United States
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Mayo Clinic
Rochester, Minnesota, 55902, United States
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Monroe-Carell Jr. Children's Hospital at Vanderbilt
Nashville, Tennessee, 37232, United States
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Montefiore Medical Center
New York, New York, 10032, United States
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NYU Langone - NYU Grossman School of Medicine
New York, New York, 10016, United States
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Nemours duPont Pediatrics Specialty Clinic
Jacksonville, Florida, 32207, United States
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Nicklaus Children's Hospital
Miami, Florida, 33155, United States
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Ochsner Hospital for Children
New Orleans, Louisiana, 70121, United States
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Office of Dr. Esperanza Font-Montgomery MD
Columbia, Missouri, 65201, United States
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Primary Children's Hospital
Salt Lake City, Utah, 84113, United States
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Stanford School of Medicine
Palo Alto, California, 94305, United States
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The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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The Community Health Clinic
Topeka, Indiana, 46571, United States
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UC San Francisco
San Francisco, California, 94158, United States
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UNC Chapel Hill
Chapel Hill, North Carolina, 27599, United States
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University of Alabama Birmingham
Birmingham, Alabama, 35233, United States
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University of Minnesota
Minneapolis, Minnesota, 55454, United States
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University of Oklahoma
Oklahoma City, Oklahoma, 73104, United States
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Washington University at St Louis
St Louis, Missouri, 63110, United States