Progressive Familial Intrahepatic CholestasisNCT04071197
Gastrostomy-Biliary Diversion: Innovative Management for Bile Canalicular Transport Disorders
A study testing Gastostomy-biliary tube and External biliary diversion, internal biliary diversion and nasobiliary tube in people with Progressive Familial Intrahepatic Cholestasis, run by National Liver Institute, Egypt at 1 site in Egypt.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Progressive Familial Intrahepatic Cholestasis
- What is being tested?
- Gastostomy-biliary tube and External biliary diversion, internal biliary diversion and nasobiliary tube
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteEgypt
- Study dates
- 10 Mar 2020to 1 Apr 2021
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Who cannot join
- Severe portal hypertensive gastropathy
- Decompensated cirrhosis
Trial locations and contacts
1 site in 1 country.
Egypt1 site, 1 recruiting
- Recruiting
Pediatric Hepatology, Gastroenterology and Nutrition Department, National Liver Institute, Menoufia University
Shibīn al Kawm, Menofiya, 32511
Ahmad M Sira, M.D., +2-048-222-2740, asira@liver.menofia.edu.eg; ahmadsira@hotmail.com
Hanaa A El-Araby, MD
About this study
Official title: 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.
Read the 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.
- Being tested
- Gastostomy-biliary tube, External biliary diversion, internal biliary diversion and nasobiliary tube
- Keywords
- bile canalicular transport disorders
- Medical subject headings
- Cholestasis, progressive familial intrahepatic 1
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Gastostomy-biliary tubeDevice
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
External biliary diversion, internal biliary diversion and nasobiliary tubeDevice
All biliary diversion modalities other than gastrobiliary tube
| Study group | Role | What they receive |
|---|---|---|
| Gastrostomy-biliary tube | Experimental | 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 Receives: Device: Gastostomy-biliary tube |
| Other biliary diversion modalities | Experimental | The control group will include those cases with other modalities of therapy as external biliary diversion, internal biliary diversion, and nasobiliary tube Receives: Device: External biliary diversion, internal biliary diversion and nasobiliary tube |
What is being measured
The primary outcome is the main question the study is designed to answer.
Occurrence of bleeding
number of patients with bleeding
Measured: 6 months
Occurrence of infection
number of patients with infection
Measured: 6 months
Secondary outcomes (2)
Serum bilirubin level
number of patients with normal bilirubin
Measured: 12 months
Improvement of pruritus
number of patients with no pruritus
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Aug 2019
- Study start10 Mar 2020
- Primary completion1 Oct 2020 (estimated)
- Study completion1 Apr 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Progressive Familial Intrahepatic CholestasisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- 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
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- National Liver Institute, Egypt
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Ahmad M Sira, M.D., Principal investigator, Pediatric Hepatology Dep; National Liver Institute, Menoufia University, Egypt
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04071197
- Sponsor's protocol ID
- GBD-PED-BCTD
- Status last verified
- Aug 2020
- First submitted
- 25 Aug 2019
- Last updated
- 24 Aug 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
van der Woerd WL, van Mil SW, Stapelbroek JM, Klomp LW, van de Graaf SF, Houwen RH. Familial cholestasis: progressive familial intrahepatic cholestasis, benign recurrent intrahepatic cholestasis and intrahepatic cholestasis of pregnancy. Best Pract Res Clin Gastroenterol. 2010 Oct;24(5):541-53. doi: 10.1016/j.bpg.2010.07.010.
PubMed 20955958Kaur S, Sharma D, Wadhwa N, Gupta S, Chowdhary SK, Sibal A. Therapeutic interventions in progressive familial intrahepatic cholestasis: experience from a tertiary care centre in north India. Indian J Pediatr. 2012 Feb;79(2):270-3. doi: 10.1007/s12098-011-0516-8. Epub 2011 Jul 19.
PubMed 21769524Mochizuki K, Obatake M, Takatsuki M, Nakatomi A, Hayashi T, Okudaira S, Eguchi S. Partial internal biliary diversion for patients with progressive familial intrahepatic cholestasis type 1. Pediatr Surg Int. 2012 Jan;28(1):51-4. doi: 10.1007/s00383-011-3018-x.
PubMed 22033772Stapelbroek JM, van Erpecum KJ, Klomp LW, Venneman NG, Schwartz TP, van Berge Henegouwen GP, Devlin J, van Nieuwkerk CM, Knisely AS, Houwen RH. Nasobiliary drainage induces long-lasting remission in benign recurrent intrahepatic cholestasis. Hepatology. 2006 Jan;43(1):51-3. doi: 10.1002/hep.20998.
PubMed 16374853
Study at a glance
From the registry record, last updated by the sponsor on 24 Aug 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Gastrostomy-Biliary Diversion: Innovative Management for Children With Bile Canalicular Transport Disorders
- ClinicalTrials.gov ID
- NCT04071197
- Acronym / sponsor ID
- GBD-PED-BCTD
- Keywords
- bile canalicular transport disorders
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- National Liver Institute, EgyptUniversity, hospital or other
- Study start
- 10 Mar 2020 (actual)
- Primary completion
- 1 Oct 2020 (estimated)
- Study completion
- 1 Apr 2021 (estimated)
- First posted
- 28 Aug 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- 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
| Arm | Type | Receives |
|---|---|---|
| Gastrostomy-biliary tube | Experimental | Device: Gastostomy-biliary tubeThe 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 |
| Other biliary diversion modalities | Experimental | Device: External biliary diversion, internal biliary diversion and nasobiliary tubeThe control group will include those cases with other modalities of therapy as external biliary diversion, internal biliary diversion, and nasobiliary tube |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (2 listed)
- Severe portal hypertensive gastropathy
- Decompensated cirrhosis
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Gastostomy-biliary tubegastrostomy 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 | Gastrostomy-biliary tube |
| External biliary diversion, internal biliary diversion and nasobiliary tubeAll biliary diversion modalities other than gastrobiliary tube | Device | Other biliary diversion modalities |
Primary outcome
Occurrence of bleeding
Time frame: 6 months
number of patients with bleeding
Occurrence of infection
Time frame: 6 months
number of patients with infection
2 secondary outcomes
Serum bilirubin level
Time frame: 12 months
Improvement of pruritus
Time frame: 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Aug 2020.
- Background: PubMed 20955958van der Woerd WL, van Mil SW, Stapelbroek JM, Klomp LW, van de Graaf SF, Houwen RH. Familial cholestasis: progressive familial intrahepatic cholestasis, benign recurrent intrahepatic cholestasis and intrahepatic cholestasis of pregnancy. Best Pract Res Clin Gastroenterol. 2010 Oct;24(5):541-53. doi: 10.1016/j.bpg.2010.07.010.
- Background: PubMed 21769524Kaur S, Sharma D, Wadhwa N, Gupta S, Chowdhary SK, Sibal A. Therapeutic interventions in progressive familial intrahepatic cholestasis: experience from a tertiary care centre in north India. Indian J Pediatr. 2012 Feb;79(2):270-3. doi: 10.1007/s12098-011-0516-8. Epub 2011 Jul 19.
- Background: PubMed 22033772Mochizuki K, Obatake M, Takatsuki M, Nakatomi A, Hayashi T, Okudaira S, Eguchi S. Partial internal biliary diversion for patients with progressive familial intrahepatic cholestasis type 1. Pediatr Surg Int. 2012 Jan;28(1):51-4. doi: 10.1007/s00383-011-3018-x.
- Background: PubMed 16374853Stapelbroek JM, van Erpecum KJ, Klomp LW, Venneman NG, Schwartz TP, van Berge Henegouwen GP, Devlin J, van Nieuwkerk CM, Knisely AS, Houwen RH. Nasobiliary drainage induces long-lasting remission in benign recurrent intrahepatic cholestasis. Hepatology. 2006 Jan;43(1):51-3. doi: 10.1002/hep.20998.
Related trials for Progressive Familial Intrahepatic Cholestasis
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
RecruitingObservational study
A Study of the Effectiveness, Safety and the Long-term Outcomes of Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay) in China
Progressive Familial Intrahepatic Cholestasis
Active, not recruitingObservational study
Odevixibat Outcomes in Patients With PFIC Versus an External Control Cohort (OvEC-PFIC)
PFIC - Progressive Familial Intrahepatic Cholestasis
RecruitingObservational study
A Study of the Effectiveness, Safety and the Long-term Outcomes of Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay) in South Korea
Progressive Familial Intrahepatic Cholestasis
RecruitingObservational study
Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)
Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ahmad M Sira, M.D. principal investigator
Pediatric Hepatology Dep; National Liver Institute, Menoufia University, Egypt
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Gastostomy-biliary tube and External biliary diversion, internal biliary diversion and nasobiliary tube in people with Progressive Familial Intrahepatic Cholestasis, run by National Liver Institute, Egypt at 1 site in Egypt.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 24 Aug 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Egypt. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Ahmad M Sira, M.D. (+2-048-222-2740, asira@liver.menofia.edu.eg; ahmadsira@hotmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Occurrence of bleeding (time frame: 6 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04071197, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Aug 2020.