Gastrostomy-Biliary Diversion: Innovative Management for Bile Canalicular Transport Disorders

August 20, 2020 updated by: National Liver Institute, Egypt

Gastrostomy-Biliary Diversion: Innovative Management for Children With Bile Canalicular Transport Disorders

Progressive familial intrahepatic cholestasis (PFIC) is a group of disorders that can present early in life with cholestasis and intractable pruritus. Their treatment poses a great challenge, with medical treatment is not successful in many cases. Moreover, the available non-transplant surgeries carry many side effects and different degrees of efficacy. Partial external biliary diversion, internal biliary diversion, and ileal exclusion still lack widespread experience with many side effects. Nasobiliary stent placement has little tolerability, especially in younger age. Gastrobiliary tube is a novel modality for external biliary diversion in such patients.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Unknown

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

Progressive familial intrahepatic cholestasis (PFIC) is a group of disorders that can present early in life with cholestasis and intractable pruritus. They result primarily from defective transporters of different bile constituents on the canalicular membrane. The mildest form of these defective transporters can present by what is called benign recurrent intrahepatic cholestasis (BRIC).

More recently both disease categories that represent the severest (PFIC) and mildest (BRIC) forms of these transporters are better nomenclated as bile canalicular transport disorders, as some cases can start early as BRIC and later in life progress the PFIC phenotype. Moreover, a continuous spectrum of severity is present between what were previously known as PFIC and BRIC.

If the severest forms are not treated effectively they can have devastating outcomes reaching death. On the other hand, those known as BRIC can have severe attacks with unremitting pruritus that could be prolonged and sometimes persistent.

Their treatment poses a great challenge, with medical treatment is not successful in many cases. Moreover, the available non-transplant surgeries carry many side effects and different degrees of efficacy. Partial external biliary diversion is not always successful and has disfiguring effects and the stoma can have many side effects. Internal biliary diversion and ileal exclusion still lack widespread experience and encouraging results due to sometimes lack of efficacy and others due to side effects. Nasobiliary stent placement was tried in previous cases with a promising outcome, but with little tolerability, especially in younger age.

In spite liver transplant is the last resort for failing cases, the availability of successful non transplant therapy would be the preferable one if it is with adequate efficacy and little side effects. The innovative intervention proposed in this study is expected to have a good efficacy due to total biliary diversion rather than the partial ones obtained by other surgeries. Moreover, it avoids the disfiguring impact of the jeujenal stoma. More importantly, it can be removed at any time and be used at the times of severe flare of the disease.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Anticipated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
20

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Egypt
    • Menofiya
      • Shibīn Al Kawm, Menofiya, Egypt, 32511
        • Recruiting
        • Pediatric Hepatology, Gastroenterology and Nutrition Department, National Liver Institute, Menoufia University
        • Contact:
        • Sub-Investigator:
          • Hanaa A El-Araby, MD
        • Sub-Investigator:
          • Hosam-Eldin M Basiouny, MD
        • Sub-Investigator:
          • Hadeer S Allam, M.B.;B.Ch.
        • Sub-Investigator:
          • Esam A Elsheimy, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

1 year to 18 years (ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • PFIC cases that are previously responding to medical treatment with recent flare of the disease that doesn't respond to medical therapy
  • PFIC cases that are not responding to medical treatment and refusing non-transplant surgery and not indicated for liver transplant
  • BRIC cases with frequent attacks not responding to medical therapy and refusing or unable to tolerate nasobiliary stent

Exclusion Criteria:

  • Severe portal hypertensive gastropathy
  • Decompensated cirrhosis

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NON_RANDOMIZED
  • Interventional Model: PARALLEL
  • Masking: NONE

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
EXPERIMENTAL: Gastrostomy-biliary tube
The study group will be subjected to gastrostomy followed by ERCP with nasobiliary stent placement in the CBD with its distal end been exit from the previously performed gastrostomy instead of the nostril
Device: Gastostomy-biliary tube
gastrostomy followed by ERCP with nasobiliary stent placement in the CBD with its distal end been exit from the previously performed gastrostomy instead of the nostril
EXPERIMENTAL: Other biliary diversion modalities
The control group will include those cases with other modalities of therapy as external biliary diversion, internal biliary diversion, and nasobiliary tube
Device: External biliary diversion, internal biliary diversion and nasobiliary tube
All biliary diversion modalities other than gastrobiliary tube

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Occurrence of bleeding
Time Frame: 6 months
number of patients with bleeding
6 months
Occurrence of infection
Time Frame: 6 months
number of patients with infection
6 months

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Serum bilirubin level
Time Frame: 12 months
number of patients with normal bilirubin
12 months
Improvement of pruritus
Time Frame: 12 months
number of patients with no pruritus
12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
National Liver Institute, Egypt

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Ahmad M Sira, M.D., Pediatric Hepatology Dep; National Liver Institute, Menoufia University, Egypt

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
March 10, 2020

Primary Completion (ANTICIPATED)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
October 1, 2020

Study Completion (ANTICIPATED)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
April 1, 2021

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
August 25, 2019

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 25, 2019

First Posted (ACTUAL)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
August 28, 2019

Study Record Updates

Last Update Posted (ACTUAL)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
August 24, 2020

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 20, 2020

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • GBD-PED-BCTD

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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