Development of a Patient-Reported Outcome Measure to Assess Symptoms in Patients With Primary Sclerosing Cholangitis (PSC)

Completed

• Conditions: Primary Sclerosing Cholangitis

• Registration Number: NCT02247934
• Lead Sponsor: Gilead Sciences

Brief Summary

The objectives of this qualitative study are to elicit concepts about symptoms that are important to patients with primary sclerosing cholangitis (PSC), as well as the key impacts of symptoms on patients' day-to-day functioning.

Detailed Description

In Step I, concept elicitation one-on-one interviews (via telephone or in person) will be conducted. The aims of the concept elicitation interviews are to:

1. Identify the most common symptoms, as well as the most important symptoms, for patients with PSC, and

2. Assess whether these symptoms impact day-to-day functioning

An instrument will be developed based on the symptoms reported and in Step II of the study, the performance of the instrument will be assessed with further interviews.

Study Timeline

• Study First Submitted: 2014-09-05
• Study First Submitted QC: 2014-09-23
• Study First Posted: 2014-09-25
• Study Start: 2014-10
• Primary Completion: 2015-10
• Study Completion: 2015-10
• Status Verified: 2016-02
• Last Update Submitted: 2016-02-08
• Last Update Posted: 2016-02-09

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 26

Inclusion Criteria

• Confirmed diagnosis of PSC based on liver biopsy or diagnostic imaging, with diagnosis made at least three months prior to screening
• Patient report of PSC symptoms associated with PSC diagnosis
• Able to speak, read, and understand English
• Willing and able to provide written informed consent to participate in the research study

Exclusion Criteria

• Patients with other causes of liver or biliary disease, including viral hepatitis, alcoholic liver disease, primary biliary cirrhosis, and secondary sclerosing cholangitis
• Patient with history of liver cirrhosis
• Patients with history of cholangiocarcinoma
• Patients who have received a liver transplant
• Patients currently enrolled in PSC clinical trials; and
• STEP II ONLY: Enrolled in or participated in Step I of this study (concept elicitation)

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
List Primary Sclerosing Cholangitis (PSC) Symptoms for inclusion in patient questionnaire	Up to 12 months	Interview participants to generate a list of the most common symptoms, as well as the most important symptoms, experienced by patients with PSC, and whether each symptom impacts day-to-day functioning.

Trial Locations

Locations (5)

University of Colorado Hospital, Denver Health Medical Center; 🇺🇸 Aurora, Colorado, United States

University of Miami Miller School of Medicine - Schiff Center for Liver Diseases; 🇺🇸 Miami, Florida, United States

Duke University Medical Center; 🇺🇸 Durham, North Carolina, United States

Evidera; 🇺🇸 Bethesda, Maryland, United States

Organ Care Research Swedish Medical Center; 🇺🇸 Seattle, Washington, United States