Cystic Fibrosis Liver DiseaseNCT05037643
Pre-emptive Transjugular Intrahepatic Portosystemic Shunt (TIPS) in Cystic Fibrosis Related Liver Disease
A study testing TIPS in people with Cystic Fibrosis Liver Disease and Portal Hypertension Due to Cystic Fibrosis (Disorder), run by University Hospital, Ghent.
Trial summary
Portal hypertension (PHT) and its sequelae are the most clinically important manifestation in cystic fibrosis related liver disease (CFLD), with end-stage liver failure as a late and rare manifestation. Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Cystic Fibrosis Liver Disease, Portal Hypertension Due to Cystic Fibrosis (Disorder), Portal Hypertension, Noncirrhotic
- What is being tested?
- TIPS
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- No sites listed yet
- Study dates
- Jun 2007to Dec 2020
- 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 Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Cystic fibrosis
- Liver disease detected by an abnormal physical examination (hepatomegaly or splenomegaly, confirmed on US), abnormalities of liver function tests (increase of AST, ALT, GGT levels above the upper normal limits) or ultrasonographic evidence of liver involvement (US liver score ≥ 5).
- Indirect signs of portal hypertension on Doppler US
- Progressive portal hypertension / liver disease on bi-monthly follow-up, evaluated by physical examination, blood analysis and US
Who cannot join
- Cirrhosis on biopsy
- Symptomatic portal hypertension (Portosytemic pressure gradient > 10 mmHg)
About this study
Official title: Pre-emptive Transjugular Intrahepatic Portosystemic Shunt (TIPS) in Pediatric Patients With Cystic Fibrosis Related Liver Disease and Non-cirrhotic Portal Hypertension
Portal hypertension (PHT) and its sequelae are the most clinically important manifestation in cystic fibrosis related liver disease (CFLD), with end-stage liver failure as a late and rare manifestation. The aim is to evaluate the safety and efficacy of a pre-emptive Transjugular Intrahepatic Portosystemic Shunt (TIPS) for the prophylaxis of variceal bleeding in pediatric CFLD patients with subclinical non-cirrhotic portal hypertension (NCPH)
Read the detailed description
Cystic fibrosis (CF) is the most frequent autosomal recessive disorder in Caucasians caused by a mutation in the cystic fibrosis transmembrane conductance regulator gene. Cystic Fibrosis associated Liver Disease (CFLD) is a well-known complication and includes a wide range of hepatobiliary diseases. The clinical outcome in CFLD is largely determined by PHT and its sequelae. Variceal bleeding is the most feared complication. Currently, there is no medical therapy to delay or reverse clinically established CFLD.Treatment focuses on screening for and managing the complications of portal hypertension and optimizing nutritional status. Eventually, liver transplantation (LT) is an effective therapeutic option for CF patients with end-stage liver failure, treatment resistant, and complicated portal hypertension.
TIPS placement is a well-established procedure for portal vein decompression in adults. In retrospective case studies of severe CFLD cirrhosis in pediatric populations, TIPS has been proven a feasible option in acute or refractory variceal bleeding and as a bridge to LT. In CFLD patients, little data are available on the long-term outcome of treatments to mitigate PHT. In this trial, the primary aim was to evaluate the safety and efficacy of a pre-emptive TIPS for the prophylaxis of variceal bleeding in non-cirrhotic CFLD with early PHT. The secondary aim was to investigate the long-term clinical outcome of a pre-emptive TIPS and in more particular, whether TIPS might effectively postpone LT.
- Conditions
- Cystic Fibrosis Liver Disease, Portal Hypertension Due to Cystic Fibrosis (Disorder), Portal Hypertension, Noncirrhotic
- Being tested
- TIPS
- Keywords
- Transjugular Intrahepatic Portosystemic Shunt, Gastrointestinal bleeding, Hypersplenism
- Medical subject headings
- Hypertension, Portal, Gastrointestinal Hemorrhage, Hypersplenism
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TIPSProcedure or surgery
Transjugular Intrahepatic Portosystemic Shunt is performed to prevent / treat symptomatic portal hypertension
| Study group | Role | What they receive |
|---|---|---|
| Pre-emptive TIPS in Cystic Fibrosis Related Liver Disease and non-cirrhotic portal hypertension | Experimental | Patients with CFLD without cirrhosis were eligible for a pre-emptive TIPS, when early (asymptomatic) signs of portal hypertension. All procedures were performed under general anaesthesia by an experienced interventional radiologist. Depending on the patient's age and physiognomy, TIPS was created following a conventional transjugular technique as for adults or by a dedicated combined percutaneous transhepatic-transjugular (PIPS) approach for small children. Routinely, an expanded polytetrafluoroethylene-covered endoprosthesis was used for shunt creation. If the sheath could not be negotiated into the main portal vein, a self-expandable, non-covered stent was placed. We did not pursue a minimum gradient reduction.
Percutaneous liver biopsy was performed during TIPS procedure to confirm the diagnosis of fibrosis or cirrhosis. Receives: Procedure: TIPS |
What is being measured
The primary outcome is the main question the study is designed to answer.
Variceal bleeding
Variceal bleeding, diagnosed on endoscopy, is regarded as the main complication of portal hypertension from cystic fibrosis liver disease
Measured: Through study completion, an average of 10 year
Secondary outcomes (1)
Hypersplenism
Thrombocytopenia causes an increased bleeding tendency. Increasing splenomegaly causes earlier concerns with abdominal distension and decreased appetite by gastric compression. In symptomatic hypersplenism, a surgical splenorenal shunt or splenectomy might be indicated.
Measured: Through study completion, an average of 10 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Sep 2021
- Study startJun 2007
- Primary completionDec 2020
- Study completionDec 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic Fibrosis Liver DiseaseStudies of the same condition, open and under way.
- More trials run by University Hospital, GhentThe sponsor's other registered studies.
- 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
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Prospective single-arm study
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Ghent
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Luc Defreyne, M.D., Ph.D, Principal investigator, University Ghent Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05037643
- Sponsor's protocol ID
- B67020071504
- Status last verified
- Aug 2021
- First submitted
- 16 Aug 2021
- Last updated
- 8 Sep 2021
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Sep 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pre-emptive Transjugular Intrahepatic Portosystemic Shunt (TIPS) in Pediatric Patients With Cystic Fibrosis Related Liver Disease and Non-cirrhotic Portal Hypertension
- ClinicalTrials.gov ID
- NCT05037643
- Acronym / sponsor ID
- B67020071504
- Conditions
- Cystic Fibrosis Liver Disease, Portal Hypertension Due to Cystic Fibrosis (Disorder), Portal Hypertension, Noncirrhotic
- Keywords
- Transjugular Intrahepatic Portosystemic Shunt, Gastrointestinal bleeding, Hypersplenism
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- University Hospital, GhentUniversity, hospital or other
- Study start
- Jun 2007 (actual)
- Primary completion
- Dec 2020 (actual)
- Study completion
- Dec 2020 (actual)
- First posted
- 8 Sep 2021 (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.
Portal hypertension (PHT) and its sequelae are the most clinically important manifestation in cystic fibrosis related liver disease (CFLD), with end-stage liver failure as a late and rare manifestation. The aim is to evaluate the safety and efficacy of a pre-emptive Transjugular Intrahepatic Portosystemic Shunt (TIPS) for the prophylaxis of variceal bleeding in pediatric CFLD patients with subclinical non-cirrhotic portal hypertension (NCPH)
Detailed description
Cystic fibrosis (CF) is the most frequent autosomal recessive disorder in Caucasians caused by a mutation in the cystic fibrosis transmembrane conductance regulator gene. Cystic Fibrosis associated Liver Disease (CFLD) is a well-known complication and includes a wide range of hepatobiliary diseases. The clinical outcome in CFLD is largely determined by PHT and its sequelae. Variceal bleeding is the most feared complication. Currently, there is no medical therapy to delay or reverse clinically established CFLD.Treatment focuses on screening for and managing the complications of portal hypertension and optimizing nutritional status. Eventually, liver transplantation (LT) is an effective therapeutic option for CF patients with end-stage liver failure, treatment resistant, and complicated portal hypertension.
TIPS placement is a well-established procedure for portal vein decompression in adults. In retrospective case studies of severe CFLD cirrhosis in pediatric populations, TIPS has been proven a feasible option in acute or refractory variceal bleeding and as a bridge to LT. In CFLD patients, little data are available on the long-term outcome of treatments to mitigate PHT. In this trial, the primary aim was to evaluate the safety and efficacy of a pre-emptive TIPS for the prophylaxis of variceal bleeding in non-cirrhotic CFLD with early PHT. The secondary aim was to investigate the long-term clinical outcome of a pre-emptive TIPS and in more particular, whether TIPS might effectively postpone LT.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Prospective single-arm study
| Arm | Type | Receives |
|---|---|---|
| Pre-emptive TIPS in Cystic Fibrosis Related Liver Disease and non-cirrhotic portal hypertension | Experimental | Procedure: TIPSPatients with CFLD without cirrhosis were eligible for a pre-emptive TIPS, when early (asymptomatic) signs of portal hypertension. All procedures were performed under general anaesthesia by an experienced interventional radiologist. Depending on the patient's age and physiognomy, TIPS was created following a conventional transjugular technique as for adults or by a dedicated combined percutaneous transhepatic-transjugular (PIPS) approach for small children. Routinely, an expanded polytetrafluoroethylene-covered endoprosthesis was used for shunt creation. If the sheath could not be negotiated into the main portal vein, a self-expandable, non-covered stent was placed. We did not pursue a minimum gradient reduction. Percutaneous liver biopsy was performed during TIPS procedure to confirm the diagnosis of fibrosis or cirrhosis. |
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Cystic fibrosis
- Liver disease detected by an abnormal physical examination (hepatomegaly or splenomegaly, confirmed on US), abnormalities of liver function tests (increase of AST, ALT, GGT levels above the upper normal limits) or ultrasonographic evidence of liver involvement (US liver score ≥ 5).
- Indirect signs of portal hypertension on Doppler US
- Progressive portal hypertension / liver disease on bi-monthly follow-up, evaluated by physical examination, blood analysis and US
Key exclusion criteria (2 listed)
- Cirrhosis on biopsy
- Symptomatic portal hypertension (Portosytemic pressure gradient > 10 mmHg)
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 |
|---|---|---|
| TIPSTransjugular Intrahepatic Portosystemic Shunt is performed to prevent / treat symptomatic portal hypertension | Procedure or surgery | Pre-emptive TIPS in Cystic Fibrosis Related Liver Disease and non-cirrhotic portal hypertension |
Primary outcome
Variceal bleeding
Time frame: Through study completion, an average of 10 year
Variceal bleeding, diagnosed on endoscopy, is regarded as the main complication of portal hypertension from cystic fibrosis liver disease
1 secondary outcome
Hypersplenism
Time frame: Through study completion, an average of 10 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Sep 2021.
- Individual participant dataSharing planned: No
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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.
Luc Defreyne, M.D., Ph.D principal investigator
University Ghent Hospital
Questions about this trial
What is this clinical trial studying?
A study testing TIPS in people with Cystic Fibrosis Liver Disease and Portal Hypertension Due to Cystic Fibrosis (Disorder), run by University Hospital, Ghent.
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 4 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: finished, as of the sponsor's last update on 8 Sep 2021. Status can change between updates, so confirm with the study contact before making plans.
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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Variceal bleeding (time frame: Through study completion, an average of 10 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 8 Sep 2021.