SARO.19.003 part B
13 evidence topics · 1 source
Objective
Pharmacokinetic and pharmacodynamic assessment
Location and study date
Study design
Open-label multiple-dose study
Eligibility criteria
PBC-cirrhosis population
Treatment
Study outcomes
Rationale for using pharmacokinetic parameters as the primary endpoint
Not applicable.
Statistical analysis description
Number of participants (Planned and analyzed)
Enrollment including replacement
| Population | Participants |
|---|---|
| Enrolled | “19” |
Description of analysis sets
Pharmacokinetic analysis
Results
Participant disposition
Replacement after withdrawal
Baseline characteristics
Efficacy results
Moderate hepatic impairment: exposure and clearance
ALP reduction after four weeks in participants with abnormal baseline ALP
| Outcome | Reported reduction |
|---|---|
| ALP reduction | “17–40%” |
Health-related quality of life and patient-reported outcomes
No evidence found.
Safety results
Serious event in a participant receiving 2 mg
Study limitations
Summary
The study was open-label and lasted four weeks. It characterizes short-term pharmacokinetics rather than comparative clinical efficacy. Increased exposure in moderate hepatic impairment and the small cohort restrict extrapolation to longer treatment or broader cirrhotic populations.