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Completed

NCT Number: NCT05037643

Pre-emptive Transjugular Intrahepatic Portosystemic Shunt (TIPS) in Cystic Fibrosis Related Liver Disease

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)

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Key information

Conditions

Cystic Fibrosis Liver Disease Digestive System Diseases Gastrointestinal Diseases Gastrointestinal Hemorrhage Hemic and Lymphatic Diseases Hemorrhage Hypersplenism Hypertension, Portal Liver Diseases Lymphatic Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Portal Hypertension Due to Cystic Fibrosis (Disorder) Portal Hypertension, Noncirrhotic Splenic Diseases

Age range

Up to 18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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)

Treatment and study plan

TIPS

Procedure

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

Primary outcomes

  1. 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

Secondary outcomes

  1. Hypersplenism

    Time frame: Through study completion, an average of 10 year

    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.

Other outcomes

  1. Liver transplant/splenectomy-free survival

    Time frame: Through study completion, an average of 10 year

    Transplant/splenectomy-free survival time was calculated from date of TIPS to the following event: transplant, splenectomy and death from any cause.

  2. Model for end-stage liver disease (MELD) score

    Time frame: Through study completion, an average of 10 year

    The Model for End-Stage Liver Disease, or MELD, is a scoring system for assessing the severity of chronic liver disease.

  3. Body Mass Index (BMI) for age Z-score

    Time frame: Through study completion, an average of 10 year

    BMI-for age Z-scores are measures of relative weight adjusted for child age and sex defined by the Flemish growth charts

  4. Length for age Z-score

    Time frame: Through study completion, an average of 10 year

    Length-for age Z-scores are measures of relative length adjusted for child age and sex defined by the Flemish growth charts

Sponsors and collaborators

Lead sponsor

University Hospital, Ghent

Other

Registry information

Official study title

Pre-emptive Transjugular Intrahepatic Portosystemic Shunt (TIPS) in Pediatric Patients With Cystic Fibrosis Related Liver Disease and Non-cirrhotic Portal Hypertension

Important dates

Study start
2007
Primary completion
2020
Study completion
2020
First posted
Sep 8, 2021
Registry last updated
Sep 8, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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