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RecruitingNCT04073290PEARLUpdated Jan 28, 2025

Prevention of Post-TIPS Hepatic Encephalopathy by Administration of Rifaximin and Lactulose

A Phase 4 interventional study of Rifaximin 550 milligram Oral Tablet [XIFAXAN] and Placebo oral tablet in Hepatic Encephalopathy, Cirrhosis, Liver and Portal Hypertension, sponsored by Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA). Recruiting at 6 sites in 2 countries. Open to participants aged 18 Years to 80 Years. Per ClinicalTrials.gov, last updated 2025-01-28.

Sponsored by Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) · Phase 4, Interventional, and Prevention

Phase
Phase 4
Study type
Interventional
Enrollment
238
Allocation
Randomized
Ages
18 Years to 80 Years
Sex
All
01

Study summary

Rationale: Hepatic encephalopathy (HE) is a major and common complication in patients with liver cirrhosis. HE can be classified in the extensive range of neurocognitive deterioration as minimal HE (MHE), covert HE (grade I), or overt HE (OHE, grade II-IV). Liver cirrhosis is the most common cause of portal hypertension (PH). Patients who develop complications of PH, like variceal bleeding or refractory ascites, can benefit from a Transjugular Intrahepatic Portosystemic Shunt (TIPS) placement. Unfortunately, post-TIPS HE is a common and often severe complication. Incidence of new onset or worsening of HE after TIPS is approximately 20-45%. Currently there is no strategy to prevent post-TIPS HE.

Read the detailed description

Objective: To assess the incidence of post-TIPS OHE within the first three months after prophylactic administration of lactulose and rifaximin versus placebo in patients who undergo Transjugular Intrahepatic Portosystemic Shunt (TIPS) placement.

Study design: A multicentre, randomized, placebo-controlled, double blind study.

Study population: Adult consecutive patients undergoing elective TIPS placement (for refractory ascites or secondary prophylaxis in variceal bleeding) in all Dutch academic centres where TIPS procedures are performed: Amsterdam UMC, location Academic Medical Centre (AMC), Erasmus MC, Leiden University Medical Centre (LUMC), Maastricht University Medical Centre+ (MUMC+), Radboud University Medical Centre (Radboudumc), University Medical Centre Groningen (UMCG), and University Hospitals Leuven (UZ Leuven) in Belgium.

Intervention: Rifaximin 550 milligram (mg) b.i.d. will be prescribed, in combination with a starting dose of 25 milliliter (mL) lactulose b.i.d. and further dependent on the amount of daily bowel movements, with the objective not to exceed more than two soft stools per day. Intervention will start 72 hours before TIPS placement, and will last till three months after TIPS placement. The control group will receive placebo in combination with lactulose (as described above).

Main study parameters/endpoints: Primary endpoint is the development of OHE within three months after TIPS placement determined by the West Haven criteria. Secondary endpoints are 90 day mortality; development of a second episode of OHE within the first three months; development of OHE in the period between three and twelve months after TIPS placement; development of MHE between TIPS placement and twelve months after placement; the increase of the psychometric hepatic encephalopathy score (PHES) and simplified one minute animal naming test (S-ANT1) compared to baseline. Differences in molecular composition of peripheral / portal blood samples at TIPS placement. Furthermore, quality of life will be assessed.

02

Conditions studied

  • Hepatic Encephalopathy
  • Cirrhosis, Liver
  • Portal Hypertension
  • Liver Diseases
  • Pathological Processes

Keywords

  • Rifaximin
  • Lactulose
  • post-TIPS HE
  • Prevention
03

Who can participate

Ages eligible
18 Years to 80 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  1. Elective TIPS placement for refractory ascites or recurrent variceal bleeding:

    Recurrent tense ascites and one or more of the following criteria:

    i. Not responding to the maximal dose of diuretics (400 milligram spironolactone and 160 milligram furosemide).

    ii. Kidney insufficiency (Creatinine > 135 umol/L) induced by diuretics. iii. Electrolyte disturbances (Sodium \< 125 mmol/L, Potassium > 5.5 mmol/L) induced by diuretics.

    iv. Not tolerating higher dose of diuretics (e.g. because of subjective side effects like muscle cramps).

    Recurrent variceal bleeding, not responsive to treatment with endoscopic band ligation and beta-blockers, with a high risk of failure of endoscopic treatment:

    i. Patients with a variceal bleeding and Child-Pugh C (10-13 points) cirrhosis or ii. Patients with a variceal bleeding, Child-Pugh B and an active bleeding during endoscopy

  2. Age ≥18 years
  3. Confirmed liver cirrhosis as documented by liver biopsy, elastography (e.g. Fibroscan) or combination of usual radiological and biochemical criteria.
  4. Signed informed consent

Exclusion criteria

Exclusion Criteria:

  1. Any absolute contraindications for TIPS placement
  2. Use of ciclosporin
  3. Life-threatening variceal bleeding with emergency TIPS placement which can not be delayed 72 hours
  4. Age > 80 years
  5. Non-cirrhotic portal hypertension
  6. Portal vein thrombosis (main trunk)
  7. HIV
  8. Current or recent (\<3 months) use of rifaximin
  9. Overt neurologic diseases such as Alzheimer's disease, Parkinson's disease
  10. Pregnant or breastfeeding women
  11. Patients refusing or unable to sign informed consent
04

Study design

Phase
Phase 4
Primary purpose
Prevention
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Triple (Participant, Care provider, Investigator)
Enrollment
238 participants (estimated)

Study arms

  • Active comparator
    Rifaximin and lactulose

    Rifaximin 550 milligram b.i.d. combined with lactulose

    Drug: Rifaximin 550 milligram Oral Tablet [XIFAXAN] · Drug: Lactulose 667 milligram/milliliter Oral Solution

  • Placebo comparator
    Placebo and lactulose

    Placebo b.i.d. combined with lactulose

    Drug: Placebo oral tablet · Drug: Lactulose 667 milligram/milliliter Oral Solution

Interventions

  • DrugRifaximin 550 milligram Oral Tablet [XIFAXAN]

    Rifaximin 550 milligram b.i.d. 72 hours before TIPS placement till 3 months post-TIPS

    Also known as: TARGAXAN

  • DrugPlacebo oral tablet

    Placebo b.i.d. 72 hours before TIPS placement till 3 months post-TIPS

    Also known as: Placebo

  • DrugLactulose 667 milligram/milliliter Oral Solution

    Lactulose based on soft stool frequency, 72 hours before TIPS placement till 3 months post-TIPS

    Also known as: Lactulose syrup

05

What researchers measure

Primary outcomes

  1. post-TIPS Hepatic Encephalopathy

    post-TIPS Hepatic Encephalopathy

    Time frame: First 3 months after TIPS placement

Secondary outcomes

  1. Mortality

    Mortality

    Time frame: 90 days

  2. Transplant free survival

    Transplant free survival

    Time frame: One year

  3. time to development of post-TIPS HE episode(s)

    time to development of post-TIPS HE episode(s)

    Time frame: One year

  4. development of a second episode of post-TIPS HE

    development of a second episode of post-TIPS HE

    Time frame: 3 months

  5. development of post-TIPS HE between 3-12 months after TIPS placement

    development of post-TIPS HE between 3-12 months after TIPS placement

    Time frame: 3-12 months

  6. change in Psychometric Hepatic Encephalopathy Score (PHES) compared to baseline

    change in total PHES score compared to baseline (range -15 - +5) a lower score is a worse outcome

    Time frame: One year

  7. change in one-minute animal naming test compared to baseline

    change in one-minute animal naming test compared to baseline

    Time frame: One year

  8. differences in molecular composition of peripheral / portal blood samples

    differences in molecular composition of peripheral / portal blood samples at TIPS placement

    Time frame: One year

  9. differences in molecular composition of peripheral blood samples

    differences in molecular composition of peripheral blood samples at baseline, compared to day 10 post-TIPS, week 4, week 12, and week 52;

    Time frame: One year

Other outcomes

  1. Health related Quality of life

    Health related Quality of life, measured by EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) questionnaire

    Time frame: One year

  2. Disease rrelated Quality of life

    Health related Quality of life, Liver Disease Symptom Index (LDSI) 2.0 questionnaire.

    Time frame: One year

  3. Cost-effectiveness

    Cost-effectiveness, measured by a combined questionnaire, based on institute for Medical Technology Assessment (iMTA) Productivity Cost Questionnaire (iPCQ)/Medical Consumption Questionnaire (iMCQ)

    Time frame: One year

06

Study locations

6 of 6 sites recruiting
  • Universitaire Ziekenhuizen Leuven
    Leuven, Belgium
    • Frederik Nevens, prof. dr. · Contact
    Recruiting
  • Academic Medical Centre
    Amsterdam, Netherlands
    • Bart Takkenberg, dr. · Contact
    Recruiting
  • University Medical Center Groningen
    Groningen, Netherlands
    • F.J.C. Cuperus, Dr. · Contact
    Recruiting
  • Leiden University Medical Center
    Leiden, Netherlands
    • M. Coenraad, Dr. · Contact
    Recruiting
  • Radboud University
    Nijmegen, Netherlands
    • E.T.T.L. Tjwa, Dr. · Contact
    Recruiting
  • Erasmus Medical Center
    Rotterdam, Netherlands
    • S. Coenen, Drs. · Contact
    Recruiting
07

References and documents

Publications

  • de Wit K, Schaapman JJ, Nevens F, Verbeek J, Coenen S, Cuperus FJC, Kramer M, Tjwa ETTL, Mostafavi N, Dijkgraaf MGW, van Delden OM, Beuers UHW, Coenraad MJ, Takkenberg RB. Prevention of hepatic encephalopathy by administration of rifaximin and lactulose in patients with liver cirrhosis undergoing placement of a transjugular intrahepatic portosystemic shunt (TIPS): a multicentre randomised, double blind, placebo controlled trial (PEARL trial). BMJ Open Gastroenterol. 2020 Dec;7(1):e000531. doi: 10.1136/bmjgast-2020-000531. PubMed 33372103 ↗

Individual participant data

Plan to share: No

08

Registry details

Key details

Study ID
NCT04073290
Lead sponsor
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Collaborators
Erasmus Medical Center, Leiden University Medical Center, Maastricht University Medical Center, Radboud University Medical Center, University Medical Center Groningen, Universitaire Ziekenhuizen KU Leuven, Norgine
Responsible party
Dr. Bart Takkenberg (Study Chair, Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)) — Principal investigator
First posted
Aug 29, 2019
Start date
Jan 21, 2020
Primary completion
Sep 30, 2026 (estimated)
Completion
Dec 31, 2026 (estimated)
Last update
Jan 28, 2025

Study contacts

Koos de Wit, MD
Contact
leverresearch@amc.uva.nl
0031-20-5668468
Bart Takkenberg, MD, PhD
principal investigator · Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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