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Can a decade of real-world data refine treatment for rare bile acid disorders?

NCT ID NCT03115086

Knowledge-focused Sponsor: Mirum Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

Study facts

What this study's own registry entry says, in plain language.

Participants

About 55 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jul 2017

Expected to finish

Jul 2039

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

All patients with a diagnosis for which Cholbam is indicated are eligible for inclusion in the Registry.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Male and female patients, of any age. 2. 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. 3. The patient has a diagnosis for which Cholbam is indicated. 4. The patient is or will be treated with Cholbam at the time of signing the informed consent form (ICF) (enrollment). Exclusion Criteria: 1\. Patients who, by judgement of the Investigator, will not be able to comply with the requirements of the protocol will be excluded

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Conditions

The condition(s) this trial relates to.

inborn disorder of bile acid synthesis Peroxisomal Disorders Zellweger spectrum disorders

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Bile acid synthesis disorders

Contacts and locations

Locations

  • Children's Center For Digestive Health

    Atlanta, Georgia, 30342, United States

  • Children's Healthcare of Atlanta

    Atlanta, Georgia, 30329, United States

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Golisano Children's Hospital, URMC

    Rochester, New York, 14642, United States

  • Icahn School Of Medicine at Mount Sinai

    New York, New York, 10029, United States

  • Kidz Pediatric Multispecialty Group

    Hollywood, Florida, 33021, United States

  • Mayo Clinic

    Rochester, Minnesota, 55902, United States

  • Monroe-Carell Jr. Children's Hospital at Vanderbilt

    Nashville, Tennessee, 37232, United States

  • Montefiore Medical Center

    New York, New York, 10032, United States

  • NYU Langone - NYU Grossman School of Medicine

    New York, New York, 10016, United States

  • Nemours duPont Pediatrics Specialty Clinic

    Jacksonville, Florida, 32207, United States

  • Nicklaus Children's Hospital

    Miami, Florida, 33155, United States

  • Ochsner Hospital for Children

    New Orleans, Louisiana, 70121, United States

  • Office of Dr. Esperanza Font-Montgomery MD

    Columbia, Missouri, 65201, United States

  • Primary Children's Hospital

    Salt Lake City, Utah, 84113, United States

  • Stanford School of Medicine

    Palo Alto, California, 94305, United States

  • The Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • The Community Health Clinic

    Topeka, Indiana, 46571, United States

  • UC San Francisco

    San Francisco, California, 94158, United States

  • UNC Chapel Hill

    Chapel Hill, North Carolina, 27599, United States

  • University of Alabama Birmingham

    Birmingham, Alabama, 35233, United States

  • University of Minnesota

    Minneapolis, Minnesota, 55454, United States

  • University of Oklahoma

    Oklahoma City, Oklahoma, 73104, United States

  • Washington University at St Louis

    St Louis, Missouri, 63110, United States