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Major study on rare childhood liver diseases halted

NCT ID NCT00571272

Knowledge-focused Sponsor: Arbor Research Collaborative for Health 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 This study
Paused for now. It may or may not start again.
Ongoing
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study followed children and young adults with genetic liver diseases that cause bile buildup. The goal was to track how these diseases progress over time, including the need for liver transplants or other complications. No treatments were tested; the aim was simply to learn more about these rare conditions.

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 1,675 people

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

Started

Nov 2007

Expected to finish

May 2029

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

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

Who is studied

The study population will consist of 400 participants with Alagille syndrome, 400 with alpha-1 trypsin deficieny (of which up to 25 may be siblings of participants), 300 with PFIC (or BRIC), and 50 with bile acid synthesis defects.

Ages

Up to 25 years

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. Children and young adults diagnosed with one of the four cholestatic diseases from birth through 25 years old. 2. Siblings of participants with alpha-1-antitrypsin deficiency, who are affected with alpha-1-antitrypsin deficiency, but have no evidence of liver disease. 3. Both sexes, all races and ethnic groups. 4. Participant meets the enrollment criteria for one of the four cholestatic liver diseases. 5. Patient and/or parent/legal guardian have the ability to provide written informed consent for enrollment. Exclusion Criteria: 1\. Inability to comply with the longitudinal follow-up described in the protocol.

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Conditions

The condition(s) this trial relates to.

Alagille syndrome alpha 1-antitrypsin deficiency benign recurrent intrahepatic cholestasis cholestasis Cholestasis, progressive familial intrahepatic 1 hereditary disease inborn disorder of bile acid synthesis liver disorder progressive familial intrahepatic cholestasis

As listed by the trial registrant

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

Alagille syndrome Alpha 1 antitrypsin deficiency Liver diseases

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    17 sites in 2 countries. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • Baylor School of Medicine

    Houston, Texas, 77030, United States

  • Children's Healthcare of Atlanta - Emory University

    Atlanta, Georgia, 30322, United States

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital of Los Angeles

    Los Angeles, California, 90027, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Cincinnati's Children's Memorial Hospital

    Cincinnati, Ohio, 60190, United States

  • Johns Hopkins University Hospital

    Baltimore, Maryland, 21287, United States

  • Mount Sinai School of Medicine

    New York, New York, 10029, United States

  • Riley Hospital for Children

    Indianapolis, Indiana, 46202, United States

  • Seattle Children's Hospital

    Seattle, Washington, 98105, United States

  • St. Louis University - Cardinal Glennon Children's Medical Center

    St Louis, Missouri, 63104, United States

  • The Hospital for Sick Children

    Toronto, Ontario, M5G 1X8, Canada

  • UPMC Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15224, United States

  • University of California at San Francisco (UCSF)

    San Francisco, California, 94143, United States

  • University of Utah

    Salt Lake City, Utah, 84113, United States

  • Washington University School of Medicine/St. Louis Children's Hospital

    St Louis, Missouri, 63110, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.