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

Gilead Sciences

Status: Completed

Conditions: Primary Sclerosing Cholangitis

Study type: Observational

Funder types: Industry

Identifiers

NCT02247934

A-13601-000

Details and patient eligibility

About

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.

Full 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.

Enrollment: 26 patients

Sex: All

Ages: 18 to 70 years old

Volunteers: No Healthy Volunteers

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)

Trial design

26 participants in 2 patient groups

Concept elicitation interviews (Step I)

Description:

Approximately 20 participants will be included.

Cognitive interviews (Step II)

Description:

Approximately 30 participants will be included.