Primary Biliary Cholangitis (PBC)
Conditions
Keywords
liver disease, pruritis, fatigue, poor sleep, brain fog, digital health technology, biometrics, Oura ring, primary biliary cholangitis, PBC
Brief summary
The purpose of this study is to explore the use of digital health technology for measuring symptoms and daily health in people with primary biliary cholangitis (PBC). Biometric data captured with a wearable device will be compared to questionnaire responses (patient-reported outcomes, or PROs) and laboratory test results to test the hypothesis that changes in biometrics can be correlated to changes in disease activity.
Detailed description
Patients enrolled are intended to start a second-line therapy (2L) for control of their PBC - the choice of therapy will be at the discretion of the treating hepatologist. Once 2L therapy is initiated, the study will assess for correlations between changes in biochemistries (alkaline phosphatase, or ALP) with biometrics and PRO results.
Interventions
None listed
Sponsors
Stanford University
Gilead Sciences
Study design
Observational model
CASE_ONLY
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Inclusion Criteria: 1. PBC diagnosis based on meeting 2 of 3 criteria: 1. Cholestatic liver biochemistry at disease outset (defined as elevation in the serum ALP level or GGT). 2. Associated autoantibody (AMA or PBC-associated antinuclear antibody by immune fluorescence or anti-PDC, anti-Gp210, or anti-Sp100) at a titer of greater than or equal to 1:40. 3. Diagnostic or compatible liver biopsy. 2. Ability to provide informed consent 3. Ability to complete digital questionnaires 4. Elevated alkaline phosphatase * Abbreviations: alkaline phosphatase \[ALP\] gamma-glutamyl-transferase \[GGT\] antimitochondrial antibody \[AMA\] Primary Biliary Cholangitis \[PBC\] Plasmacytoid dendritic cells \[PDC\] glycoprotein 210 \[Gp210\] Scleroderma 100 \[Sp100\]
Exclusion criteria
1. Uncontrolled chronic inflammatory condition that is likely to contribute to symptoms of poor fatigue and sleep and confound results. 2. Active malignancy requiring treatment
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Sleep scores to assess sleep quality will be measured in PBC patients using a wearable device | 7 months (1 month of screening and 6 months wearing device) | Sleep Score 0 - 100 where 0 is worst sleep experience and 100 is best sleep experience |
| SpO2 (saturation of peripheral oxygen) levels to assess sleep quality | 7 months (1 month of screening and 6 months wearing device) | — |
| Total time in bed, to assess sleep quality | 7 months (1 month of screening and 6 months wearing device) | Number of hours per day spent in bed |
| Total sleep, to assess sleep quality | 7 months (1 month of screening and 6 months wearing device) | Number of hours per day spent asleep |
| Sleep stage time series (REM), to assess sleep quality | 7 months (1 month of screening and 6 months wearing device) | Number of hours per day spent in REM (Rapid Eye Movement) sleep |
| Sleep stage time series (deep), to assess sleep quality | 7 months (1 month of screening and 6 months wearing device) | Number of hours per day spent in deep sleep |
| Sleep stage time series (light), to assess sleep quality | 7 months (1 month of screening and 6 months wearing device) | Number of hours per day spent in light sleep |
| Number of "get ups" during sleep, to assess sleep quality | 7 months (1 month of screening and 6 months wearing device) | Number of times "getting up" during the night while trying to sleep |
| Temperature deviation from baseline, to assess sleep quality | 7 months (1 month of screening and 6 months wearing device) | change in temperature over time during sleep cycle |
| Heart rate | 7 months (1 month of screening and 6 months wearing device) | — |
| Respiratory rate | 7 months (1 month of screening and 6 months wearing device) | — |
| Calories burned during exercise to assess daily activity | 7 months (1 month of screening and 6 months wearing device) | Number of calories burned during each exercise session |
| Calories burned throughout the day to assess daily activity | 7 months (1 month of screening and 6 months wearing device) | total number of calories burned each day |
| Distance traveled during exercise to assess daily activity | 7 months (1 month of screening and 6 months wearing device) | Number of miles traveled during each exercise session |
| Number of steps to assess daily activity | 7 months (1 month of screening and 6 months wearing device) | total number of steps taken each day |
| Change in PBC-40 fatigue domain score in patients with PBC | 7 months (1 month screening and 6 months wearing the device) | The PBC-40 fatigue domain allows a potential value range of 11 to 55 with higher values denoting worse fatigue. A score of \>33 identifies patients with moderate-severe fatigue and a mean change in this score by 5 points after 3 months has been used in other studies to denote clinically meaningful significance. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in alkaline phosphatase (ALP) levels | Baseline and following 2L therapy (at doctor's discretion, within 7 month timeline) | ALP levels (where lower levels reflect better control of PBC disease) will be measured in patients both before and after a second-line therapy (2L) is started. |
| Changes in Daily Fatigue Impact Scale. | 7 months (1 month screening and 6 months wearing the device) | The Daily Fatigue Impact Scale measures how fatigue interferes with daily functioning. Scores range from 0 to 32, with higher numbers indicating a greater severity of fatigue. |
| Change in Epworth Sleepiness Scale (ESS) score | 7 months (1 month screening and 6 months wearing the device) | The ESS score (the sum of 8 item scores, 0-3) can range from 0 to 24. A result from 11 to 24 indicates excessive (abnormal) daytime sleepiness. |
Countries
United States
Contacts
PRINCIPAL_INVESTIGATORAparna Goel, MD
Stanford University
Outcome results
None listed