Clinical trial NCT02247934: Development of a Patient-Reported Outcome Measure to Assess Symptoms in Patients With Primary Sclerosing Cholangitis (PSC)
Official title: Development of a Patient-Reported Outcome Measure to Assess Symptoms in Patients With Primary Sclerosing Cholangitis (PSC)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Primary Sclerosing Cholangitis
Keywords: Primary sclerosing cholangitis
PSC
Lead sponsor: Gilead Sciences
Sponsor class: INDUSTRY
First posted: 2014-09-25
Enrollment: 26
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Time perspective: CROSS_SECTIONAL
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants will be recruited from approximately 5 clinical sites based in the United States.

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.

Eligibility criteria: 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)

Primary outcomes:
measure: List Primary Sclerosing Cholangitis (PSC) Symptoms for inclusion in patient questionnaire
timeFrame: Up to 12 months
Secondary outcomes:
Other outcomes:
Locations:
city: Aurora
country: United States
facility: University of Colorado Hospital, Denver Health Medical Center
state: Colorado
city: Miami
country: United States
facility: University of Miami Miller School of Medicine - Schiff Center for Liver Diseases
state: Florida
city: Bethesda
country: United States
facility: Evidera
state: Maryland
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Seattle
country: United States
facility: Organ Care Research Swedish Medical Center
state: Washington
Study officials:
affiliation: Gilead Sciences
name: Rob Myers, MD
role: STUDY_DIRECTOR
References: