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This study will collect information from patients with Primary Biliary Cholangitis (PBC).
PBC is a progressive rare liver disease in which tubes in the liver called bile ducts are damaged. The liver damage in PBC may lead to scarring (cirrhosis). People living with PBC often face challenges like intense itching, trouble sleeping, and feeling constantly tired. These symptoms can make it hard for them to enjoy daily life and feel well overall.
While itching and sleep patterns can be difficult to measure accurately based on recall and may lack precision, the main aim of this study is to use a digital health tool to assess the severity of itchiness and sleep patterns in people with PBC. The tool will take the objective measurement for people so they do not have to recall their scratch and sleep patterns. The total study duration for each patient will be about 91 days.
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| Measure | Description | Time Frame |
|---|---|---|
| Number of Nocturnal Scratch Events | Defined as the total number of scratch events detected during sleep periods, monitored using the Nighttime sensor wear. This outcome will be assessed at the end of the wear period. | At weeks 0, 4, 8 and 12 |
| Frequency of Low- and High-Intensity Nocturnal Scratch Events | Defined as the frequency of scratch events categorized into low- and high-intensity levels during sleep periods, as detected by the Nighttime sensor wear. This outcome will be assessed at the end of the wear period. | At weeks 0, 4, 8 and 12 |
| Average Scratch Intensity Per Night | Defined as the average intensity of scratch events per night, calculated from data recorded by the Nighttime sensor wear. This outcome will be assessed at the end of the wear period. | At weeks 0, 4, 8 and 12 |
| Hourly Frequency of Nocturnal Scratch Events During Sleep | Defined as the frequency of nocturnal scratch events occurring per hour during sleep periods, recorded using the Nighttime sensor wear. This outcome will be assessed at the end of the wear period. | At weeks 0, 4, 8 and 12 |
| Average Duration of Scratch Events Per Night | Defined as the average duration of scratch events occurring during sleep periods, calculated using data collected via the Nighttime sensor wear. This outcome will be assessed at the end of the wear period. | At weeks 0, 4, 8 and 12 |
| Total Duration of Scratch Behavior Per Patient During Sleep Periods | Defined as the total duration of scratch events recorded for each patient during sleep periods using the Nighttime sensor wear. This outcome will be assessed at the end of the wear period. |
| Measure | Description | Time Frame |
|---|---|---|
| Demographic Data: Age at Enrollment | Defined as the age of patients recorded at the time of enrollment. | At enrollment visit (Week 0, Day 1) |
| Demographic Data: Sex at Enrollment | Defined as the sex of patients recorded at the time of enrollment. |
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Inclusion Criteria:
Exclusion Criteria:
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Adult patients diagnosed with PBC
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| Name | Affiliation | Role |
|---|---|---|
| Ipsen Medical Director | Ipsen | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Arizona Liver Center | Chandler | Arizona | 85224 | United States | ||
| University of Miami School of Medicine, Center for Liver Diseases |
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan, and dataset specifications.
Patient level data will be anonymized and study documents will be redacted to protect the privacy of study participants.
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Where applicable, data from eligible studies are available 6 months after the studied medicine and indication have been approved in the US and/or EU.
Further details on Ipsen's sharing criteria and process for sharing are available here (https://www.ipsen.com/science/clinical-trials/clinical-data-transparency/).
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| ID | Term |
|---|---|
| D008105 | Liver Cirrhosis, Biliary |
| ID | Term |
|---|---|
| D002780 | Cholestasis, Intrahepatic |
| D002779 | Cholestasis |
| D001649 | Bile Duct Diseases |
| D001660 | Biliary Tract Diseases |
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| At weeks 0, 4, 8 and 12 |
| Total Sleep Duration (Hours) | Defined as the total duration of sleep per patient, recorded by the Nighttime sensor wear. This outcome will be assessed at the end of the wear period. | At weeks 0, 4, 8 and 12 |
| Duration of Wakefulness After Sleep Onset (WASO, Minutes) | Defined as the total duration of wakefulness after the initial onset of sleep, measured using the Nighttime sensor wear. This outcome will be assessed at the end of the wear period. | At weeks 0, 4, 8 and 12 |
| Duration of Wakefulness During Scratch Periods | Defined as the total duration of wakefulness during scratch periods, monitored via the Nighttime sensor wear. This outcome will be assessed at the end of the wear period. | At weeks 0, 4, 8 and 12 |
| Average Sleep Duration Between Scratch Periods | Defined as the average duration of sleep occurring between scratch periods, as measured by the Nighttime sensor wear. This outcome will be assessed at the end of the wear period. | At weeks 0, 4, 8 and 12 |
| At enrollment visit (Week 0, Day 1) |
| Age at Diagnosis (As Available) | Defined as the patient's age at the time of diagnosis, as reported in their medical history. | At enrollment visit (Week 0, Day 1) |
| Age at First Symptoms (As Available) | Defined as the patient's age when first symptoms were reported, based on medical records or patient recollection. | At enrollment visit (Week 0, Day 1) |
| Time Duration Since PBC Diagnosis | Defined as the duration between the patient's initial diagnosis of PBC and the time of enrollment, as recorded in their medical history. | At enrollment visit (Week 0, Day 1) |
| Current PBC-Related Treatments | Defined as the treatments patients are currently receiving for PBC, including medication and other therapies, as reported at enrollment. | At enrollment visit (Week 0, Day 1) |
| Time Duration on Current PBC-Related Treatment | Defined as the duration for which patients have been receiving their current PBC-related treatment, as recorded during enrollment. | At enrollment visit (Week 0, Day 1) |
| Concomitant Medications for Itch/Scratch and/or Sleep Disturbances | Defined as the additional medications patients are currently using to manage itch/scratch and/or sleep disturbances, as reported at enrollment. | At enrollment visit (Week 0, Day 1) |
| Baseline Liver Function Tests: Alanine aminotransferase (ALT) Levels | At enrollment visit (Week 0, Day 1) |
| Baseline Liver Function Tests: Aspartate aminotransferase (AST) Levels | At enrollment visit (Week 0, Day 1) |
| Baseline Liver Function Tests: Alkaline phosphatase (ALP) Levels | At enrollment visit (Week 0, Day 1) |
| Baseline Liver Function Tests: Bilirubin Levels | At enrollment visit (Week 0, Day 1) |
| Baseline Liver Function Tests: Albumin Levels | At enrollment visit (Week 0, Day 1) |
| Baseline Enhanced Liver Fibrosis (ELF) Test: Hyaluronic Acid (HA) Levels | At enrollment visit (Week 0, Day 1) |
| Baseline ELF Test: Procollagen Type III Amino-Terminal Propeptide (PIIINP) Levels | At enrollment visit (Week 0, Day 1) |
| Baseline ELF Test: Tissue Inhibitor of Metalloproteinase-1 (TIMP-1) Levels | At enrollment visit (Week 0, Day 1) |
| Baseline Liver Stiffness Measurement by Fibroscan | At enrollment visit (Week 0, Day 1) |
| PBC Worst Itch Numeric Rating Scale (PBC WI NRS) Score | Defined as the patient's score on the PBC WI NRS, assessing the severity of worst itch experienced during the study duration. | At weeks 0, 4, 8 and 12 |
| PBC-40 Itch Severity Score | Defined as the patient's score on the itch severity domain of the PBC-40 questionnaire, measuring the impact of itch on their quality of life. | At weeks 0, 4, 8 and 12 |
| Duration, degree, direction, disability, and distribution (5-D) Itch Scale | Defined as the patient's score on the 5-D Itch Scale, capturing dimensions of duration, degree, direction, disability, and distribution of itch. | At weeks 0, 4, 8 and 12 |
| PBC-40 Fatigue Severity Score | Defined as the patient's score on the fatigue severity domain of the PBC-40 questionnaire, quantifying the impact of fatigue on their daily life. | At weeks 0, 4, 8 and 12 |
| Sleep Disturbance Numeric Rating Scale (SD NRS) Score | Defined as the patient's score on the SD NRS, evaluating the severity of sleep disturbances experienced during the study duration. | At weeks 0, 4, 8 and 12 |
| Patient-Reported Outcomes Measurement Information System® (PROMIS®) Fatigue Short Form (SF)-7a Score | Defined as the patient's score on the PROMIS® Fatigue SF-7a, assessing fatigue levels and their effects on physical, mental, and social activities. | At weeks 0, 4, 8 and 12 |
| Percentage of Patient Responses to Sibel Health System Usability Survey (SUS) | Defined as the patients' feedback on the usability of the Sibel Health System, captured through the SUS administered during enrollment. This outcome will assess user experience and satisfaction with the system. | At week 12 (End of Study Survey) |
| Miami |
| Florida |
| 33136 |
| United States |
| UT Southwestern Medical Center, Department of Internal Medicine, Digestive and Liver Diseases | Dallas | Texas | 75390 | United States |
| D004066 |
| Digestive System Diseases |
| D008107 | Liver Diseases |
| D008103 | Liver Cirrhosis |
| D005355 | Fibrosis |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |