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New national registry aims to unlock secrets of rare childhood liver diseases

NCT ID NCT07411716

Knowledge-focused Sponsor: Children's Hospital of Eastern Ontario Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 creates a Canadian registry for children under 18 with Alagille syndrome or genetic intrahepatic cholestasis, rare liver conditions that cause bile buildup. Researchers will collect health data from medical records over time to understand how these diseases progress and how current treatments work in real life. The goal is to improve care and outcomes for affected children across Canada.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

What this could lead to
If successful, this registry will provide crucial data on how these rare liver diseases progress over time, helping doctors improve treatment and care for affected children.
What could go wrong
This is an observational registry, not a treatment trial, so it won't directly test a new therapy. Results depend on consistent data collection and may take years to yield meaningful insights.

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 220 people

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

Started

Apr 2026

Expected to finish

Dec 2031

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

Pediatric participants (\<18 years old) with genetically confirmed or clinically diagnosed ALGS or any of the various subtypes of GIC.

Ages

Up to 18 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: * Pediatric participants (\<18 years old) with genetically confirmed or clinically diagnosed ALGS or any of the various subtypes of GIC, each associated with a distinct genetic mutation: A. PFIC Type 1 (FIC1 Deficiency) - Mutation in ATP8B1 gene. B. PFIC Type 2 (BSEP Deficiency) - Mutation in ABCB11 gene. C. PFIC Type 3 (MDR3 Deficiency) - Mutation in ABCB4 gene. D. PFIC Type 4 (TJP2 Deficiency) - Mutation in TJP2 gene. E. PFIC Type 5 (FXR Deficiency) - Mutation in NR1H4 gene. F. PFIC Type 6 (MYO5B-Associated) - Mutation in MYO5B gene. G. Progressive cholestasis of northwestern Quebec (PCNQ)-Mutation in UTP4 gene. Other novel PFIC-like conditions continue to be identified and may be included in the registry. If additional conditions are identified for inclusion in the registry, a protocol amendment will be submitted for REB approval. * Enrollment within Canadian pediatric liver centers participating in the registry. These include: Children's Hospital of Eastern Ontario (Ottawa, ON, Lead Site), CHU Sainte-Justine (Montreal, QC), McMaster Children's Hospital (Hamilton, ON), Montreal Children's Hospital (Montreal, QC), Alberta Children's Hospital (Calgary, AB), Stollery Children's Hospital (Edmonton, AB), Janeway Children's Health and Rehabilitation Centre (St. John's, NL), Jim Pattison Children's Hospital (Saskatoon, SK), Children's Hospital LHSC (London, ON), Children's Hospital IWK Health Centre (Halifax, NS), BC Children's Hospital (Vancouver, BC), HSC Winnipeg Children's Hospital (Winnipeg, MB), Hôpital de l'Enfant-Jésus (Quebec City, QC) * Written informed consent obtained from participant if they have the capacity, or parents/guardians, and assent from participants as appropriate. Exclusion Criteria: * Inability to comply with follow-up requirements (lost to follow-up)

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Conditions

The condition(s) this trial relates to.

Alagille syndrome cholestasis Cholestasis, progressive familial intrahepatic 1 cholestasis, progressive familial intrahepatic, 4 cholestasis, progressive familial intrahepatic, 5 cholestasis, progressive familial intrahepatic, 6 familial intrahepatic cholestasis hereditary North American Indian childhood cirrhosis intrahepatic cholestasis progressive familial intrahepatic cholestasis progressive familial intrahepatic cholestasis type 1 progressive familial intrahepatic cholestasis type 2 progressive familial intrahepatic cholestasis type 3

As listed by the trial registrant

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

Alagille syndrome (ALGS) Cholestasis, intrahepatic Pfic - progressive familial intrahepatic cholestasis

How to take part

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

  1. The places running it

    13 sites. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    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

  • Alberta Children's Hospital

    NOT_YET_RECRUITING

    Calgary, Alberta, Canada

  • BC Children's Hospital

    NOT_YET_RECRUITING

    Vancouver, British Columbia, Canada

  • CHU Sainte-Justine

    NOT_YET_RECRUITING

    Montreal, Quebec, Canada

  • CHU de Quebec - Universite Laval (Centre Mere-Enfant Soleil)

    NOT_YET_RECRUITING

    Qubec City, Quebec, Canada

  • Children's Hospital IWK Health Centre

    NOT_YET_RECRUITING

    Halifax, Nova Scotia, Canada

  • Children's Hospital LHSC

    NOT_YET_RECRUITING

    London, Ontario, Canada

  • Children's Hospital of Eastern Ontario

    RECRUITING

    Ottawa, Ontario, Canada

  • HSC Winnipeg Children's Hospital

    NOT_YET_RECRUITING

    Winnipeg, Manitoba, Canada

  • Janeway Children's Health and Rehabilitation Centre

    NOT_YET_RECRUITING

    St. John's, Newfoundland and Labrador, Canada

  • Jim Pattison Children's Hospital

    NOT_YET_RECRUITING

    Saskatoon, Saskatchewan, Canada

  • McMaster Children's Hospital

    NOT_YET_RECRUITING

    Hamilton, Ontario, Canada

  • Montreal Children's Hospital

    NOT_YET_RECRUITING

    Montreal, Quebec, Canada

  • Stollery Children's Hospital

    NOT_YET_RECRUITING

    Edmonton, Alberta, Canada

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