Clinical trial NCT02488993: Real World Outcomes Study of Hepatic Encephalopathy Patients' Experience on Rifaximin (PROSPER)
Official title: PROSPER: Prospective Real World Outcomes Study of Hepatic Encephalopathy Patients' Experience on Rifaximin-α (TARGAXAN®/XIFAXAN®) 550 mg
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hepatic Encephalopathy
Lead sponsor: Norgine
Sponsor class: INDUSTRY
First posted: 2015-07-02
Enrollment: 389
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with cirrhosis aged 18 years or over who are enrolled within 12 weeks of resolution of an episode of overt HE associated with a hospital visit, who are eligible for treatment with rifaximin-α 550 mg.

Brief summary: This study evaluates hepatic encephalopathy (HE) and liver-related hospitalization rates and duration of stay in patients with HE treated with rifaximin-α 550mg compared to patients receiving other therapies.

This registry study aims to comprehensively and rigorously characterize the impact of rifaximin-α 550 mg on hospitalization, clinical safety and effectiveness outcomes, and quality of life in patients with HE in Europe and Australasia.

Detailed description: Whilst clinical trial data and a few small observational studies have demonstrated the potential of rifaximin-α 550 mg to reduce overt HE episodes and hospitalizations, there is a need by physicians, commissioners and other healthcare professionals caring for patients with HE to understand the impact of management with rifaximin-α 550 mg on healthcare resource use in real world clinical practice. Currently available data are from evaluations undertaken in single UK centres. In addition, the overall burden of HE has not been well characterized, including the impact on patient quality of life, or impaired productivity incurred by patients' caregivers. This multinational, multicentre disease registry study aims to comprehensively and rigorously characterize the impact of rifaximin-α 550 mg on hospitalization, clinical safety and effectiveness outcomes, and quality of life in patients with HE in Europe and Australasia. The study will expand upon previous work in the following ways:

* Provide characterization of the burden of HE and the impact of rifaximin-α 550 mg in a real world setting, to complement RCT evidence;
* Allow for more rigorous characterization of the impact of rifaximin-α 550 mg on patient outcomes for individuals with HE over time. This will be achieved by prospectively enrolling patients not taking rifaximin-α 550 mg to characterize outcomes over time in the absence of treatment, and by assessing the quality of life and workplace productivity implications of HE.

There will be no change to the management of patients for the purposes of this study beyond normal clinical practice.

Eligibility criteria: Inclusion Criteria:

* Diagnosis of cirrhosis;
* Patient is ≥18 years of age;
* Patient is enrolled within 12 weeks of resolution of an episode of overt HE associated with a hospital visit;
* Patient is able to provide informed consent to participate in the study (individually or via caregiver);
* Patient meets clinical eligibility to receive rifaximin-α 550 mg in the opinion of the participating physician, regardless of HE treatment actually received.

Exclusion Criteria:

* West Haven Conn score of ≥2 at time of study entry (i.e. at provision of informed consent);
* Mental health disorder such as dementia or psychosis which makes diagnoses of HE questionable;
* Prior treatment with rifaximin in the 12 months before qualifying episode of overt HE associated with a hospital visit;
* Contraindications to the use of rifaximin-α as per local summary of product characteristics

Primary outcomes:
measure: HE and liver related hospitalization rate in patients receiving rifaximin- α 550mg compared to other therapies
timeFrame: 12 months
Secondary outcomes:
measure: All-cause hospitalization rate in patients with HE receiving rifaximin-α 550 mg compared to those receiving other therapies
timeFrame: 12 months
measure: Global Evaluation of Treatment Effectiveness of rifaximin-α 550 mg compared to other therapies
timeFrame: 12 months
measure: Chronic Liver Disease Questionnaire (CLDQ) to evaluate effectiveness of rifaximin-α 550 mg compared to other therapies
timeFrame: 12 months
measure: Number of sublects with adverse events after rifaximin-α 550 mg compared with other therapies
timeFrame: 12 months
measure: Work productivity and Activity Impairment Questionnaire to evaluate quality of life in patients with HE
timeFrame: 12 months
Other outcomes:
Locations:
city: Birmingham
country: United Kingdom
facility: Queen Elizabeth Hospital
Study officials:
affiliation: Norgine
name: Richard Ng
role: STUDY_DIRECTOR
References:
citation: Krag A, Schuchmann M, Sodatonou H, Pilot J, Whitehouse J, Strasser SI, Hudson M. Design of the Prospective Real-world Outcomes Study of hepatic encephalopathy Patients' Experience on Rifaximin-alpha (PROSPER): an observational study among 550 patients. Hepatol Med Policy. 2018 Jan 8;3:4. doi: 10.1186/s41124-017-0029-9. eCollection 2018.