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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT05037643
Other study ID # B67020071504
Secondary ID
Status Completed
Phase N/A
First received
Last updated
Start date June 2007
Est. completion date December 2020

Study information

Verified date August 2021
Source University Hospital, Ghent
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical 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. 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)


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.


Recruitment information / eligibility

Status Completed
Enrollment 5
Est. completion date December 2020
Est. primary completion date December 2020
Accepts healthy volunteers No
Gender All
Age group N/A to 18 Years
Eligibility Inclusion Criteria: - 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 Exclusion Criteria: - Cirrhosis on biopsy - Symptomatic portal hypertension (Portosytemic pressure gradient > 10 mmHg)

Study Design


Intervention

Procedure:
TIPS
Transjugular Intrahepatic Portosystemic Shunt is performed to prevent / treat symptomatic portal hypertension

Locations

Country Name City State
n/a

Sponsors (1)

Lead Sponsor Collaborator
University Hospital, Ghent

Outcome

Type Measure Description Time frame Safety issue
Other Liver transplant/splenectomy-free survival Transplant/splenectomy-free survival time was calculated from date of TIPS to the following event: transplant, splenectomy and death from any cause. Through study completion, an average of 10 year
Other Model for end-stage liver disease (MELD) score The Model for End-Stage Liver Disease, or MELD, is a scoring system for assessing the severity of chronic liver disease. Through study completion, an average of 10 year
Other Body Mass Index (BMI) for age Z-score BMI-for age Z-scores are measures of relative weight adjusted for child age and sex defined by the Flemish growth charts Through study completion, an average of 10 year
Other Length for age Z-score Length-for age Z-scores are measures of relative length adjusted for child age and sex defined by the Flemish growth charts Through study completion, an average of 10 year
Primary Variceal bleeding Variceal bleeding, diagnosed on endoscopy, is regarded as the main complication of portal hypertension from cystic fibrosis liver disease Through study completion, an average of 10 year
Secondary 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. Through study completion, an average of 10 year
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