Trial Outcomes & Findings for Real-World Data Study to Evaluate the Effectiveness of OCA on Hepatic Outcomes in PBC Patients (NCT NCT05292872)
NCT ID: NCT05292872
Last Updated: 2025-01-24
Results Overview
The primary analysis outcome was assessed with hazard ratio (HR) comparing hazard of first event of the composite endpoint among OCA-treated participants and SMR-weighted non-OCA-treated PBC participants indexes. It included all-cause death, liver transplant, hospitalization for hepatic decompensation based on first occurrence of: variceal bleed, ascites (including hepatic hydrothorax and spontaneous bacterial peritonitis) and hepatic encephalopathy. OCA-treated indexes were censored 90 days after OCA discontinuation, or if fibrates were initiated. Control indexes were censored if a participant-initiated OCA therapy, initiated fibrate therapy, reinitiated UDCA for participants who had discontinued UDCA for \>6 months, or end of study period (31 Dec 2021), whichever came first. The 2.5th and 97.5th percentile of nonparametric bootstrap samples were used to estimate 95% CI for HR and to perform a test of hypothesis. Risk is presented using the number of composite event and components.
COMPLETED
4577 participants
Up to 67 months
2025-01-24
Participant Flow
This study was conducted in the United States (US) and data was analyzed from Komodo Health claims database between 01 Jun 2015 to 31 Dec 2021.
Participants were included if, between 01Jun2015 and 31Dec2021, they met criteria for PBC diagnosis: At least 1 inpatient claim with an associated PBC International Classification of Diseases (ICD)-10 code, or at least 2 outpatient claims separated by \>1 day with a PBC ICD-9 or ICD-10 code. Patient records (indexes) were selected from a healthcare database. The unit of analysis were indexes, not participants. Analysis was conducted using weighted index methods.
Unit of analysis: Indexes
Participant milestones
| Measure |
OCA-treated
PBC participants with a history of Ursodeoxycholic acid (UDCA) failure (inadequate response, intolerance, or discontinuation) who initiated Obeticholic acid (OCA) in the study window were included.
|
Non-OCA Treated
PBC participants with a history of UDCA failure who were eligible but not treated with OCA for the study. This was a real-world study, and it was conducted in a population of participants selected from a database using weighted index methods.
|
|---|---|---|
|
Overall Study
STARTED
|
403 403
|
4174 405
|
|
Overall Study
COMPLETED
|
403 403
|
4174 405
|
|
Overall Study
NOT COMPLETED
|
0 0
|
0 0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Race and Ethnicity were not collected from any participant.
Baseline characteristics by cohort
| Measure |
OCA-treated
n=403 Indexes
PBC participants with a history of UDCA failure (inadequate response, intolerance, or discontinuation) who initiated OCA in the study window were included.
|
Non-OCA Treated
n=405 Indexes
PBC participants with a history of UDCA failure who were eligible but not treated with OCA for the study. This was a real-world study, and it was conducted in a population of participants selected from a database using weighted index methods.
|
Total
n=808 Indexes
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
56.213 Years
STANDARD_DEVIATION 10.580 • n=403 Indexes
|
55.859 Years
STANDARD_DEVIATION 12.599 • n=405 Indexes
|
56.036 Years
STANDARD_DEVIATION 11.638 • n=808 Indexes
|
|
Sex: Female, Male
Female
|
369 Indexes
n=403 Indexes
|
369 Indexes
n=405 Indexes
|
738 Indexes
n=808 Indexes
|
|
Sex: Female, Male
Male
|
34 Indexes
n=403 Indexes
|
36 Indexes
n=405 Indexes
|
70 Indexes
n=808 Indexes
|
|
Race and Ethnicity Not Collected
|
—
|
—
|
0 Participants
Race and Ethnicity were not collected from any participant.
|
|
Region
Northeast
|
82 Indexes
n=403 Indexes
|
84 Indexes
n=405 Indexes
|
166 Indexes
n=808 Indexes
|
|
Region
Midwest
|
38 Indexes
n=403 Indexes
|
38 Indexes
n=405 Indexes
|
76 Indexes
n=808 Indexes
|
|
Region
West
|
97 Indexes
n=403 Indexes
|
106 Indexes
n=405 Indexes
|
203 Indexes
n=808 Indexes
|
|
Region
South
|
186 Indexes
n=403 Indexes
|
175 Indexes
n=405 Indexes
|
361 Indexes
n=808 Indexes
|
|
Region
Territory
|
0 Indexes
n=403 Indexes
|
1 Indexes
n=405 Indexes
|
1 Indexes
n=808 Indexes
|
|
Region
Unknown
|
0 Indexes
n=403 Indexes
|
1 Indexes
n=405 Indexes
|
1 Indexes
n=808 Indexes
|
PRIMARY outcome
Timeframe: Up to 67 monthsPopulation: Weighted index population. The unit of analysis is Indexes.
The primary analysis outcome was assessed with hazard ratio (HR) comparing hazard of first event of the composite endpoint among OCA-treated participants and SMR-weighted non-OCA-treated PBC participants indexes. It included all-cause death, liver transplant, hospitalization for hepatic decompensation based on first occurrence of: variceal bleed, ascites (including hepatic hydrothorax and spontaneous bacterial peritonitis) and hepatic encephalopathy. OCA-treated indexes were censored 90 days after OCA discontinuation, or if fibrates were initiated. Control indexes were censored if a participant-initiated OCA therapy, initiated fibrate therapy, reinitiated UDCA for participants who had discontinued UDCA for \>6 months, or end of study period (31 Dec 2021), whichever came first. The 2.5th and 97.5th percentile of nonparametric bootstrap samples were used to estimate 95% CI for HR and to perform a test of hypothesis. Risk is presented using the number of composite event and components.
Outcome measures
| Measure |
OCA-treated
n=403 Indexes
PBC participants with a history of UDCA failure (inadequate response, intolerance, or discontinuation) who initiated OCA in the study window were included.
|
Non-OCA Treated
n=405 Indexes
PBC participants with a history of UDCA failure who were eligible but not treated with OCA for the study. This was a real-world study, and it was conducted in a population of participants selected from a database using weighted index methods.
|
|---|---|---|
|
Risk of the First Event of the Composite Events
|
8 Events during the study period
|
32 Events during the study period
|
SECONDARY outcome
Timeframe: Up to 67 monthsPopulation: Weighted index population. The unit of analysis is Indexes.
The primary source for death was the Social Security Death Index (SSDI) along with obituary search, which was compared to Komodo Health claims and LabCorp/Quest laboratory data. The secondary objectives were to estimate the effect of OCA treatment versus non-OCA treatment on each component of the composite endpoint, with the same censoring rule applied as in the primary composite endpoint. Risk is presented using the the number of all-cause death within the risk period (prior to censoring).
Outcome measures
| Measure |
OCA-treated
n=403 Indexes
PBC participants with a history of UDCA failure (inadequate response, intolerance, or discontinuation) who initiated OCA in the study window were included.
|
Non-OCA Treated
n=405 Indexes
PBC participants with a history of UDCA failure who were eligible but not treated with OCA for the study. This was a real-world study, and it was conducted in a population of participants selected from a database using weighted index methods.
|
|---|---|---|
|
Risk of Death
|
2 Events during the study period
|
9 Events during the study period
|
SECONDARY outcome
Timeframe: Up to 67 monthsPopulation: Weighted index population. The unit of analysis is Indexes.
The primary source for liver transplant was the Organ Transplant Network (OPTN) transplant registry. Risk is presented using the number of liver transplantation.
Outcome measures
| Measure |
OCA-treated
n=403 Indexes
PBC participants with a history of UDCA failure (inadequate response, intolerance, or discontinuation) who initiated OCA in the study window were included.
|
Non-OCA Treated
n=405 Indexes
PBC participants with a history of UDCA failure who were eligible but not treated with OCA for the study. This was a real-world study, and it was conducted in a population of participants selected from a database using weighted index methods.
|
|---|---|---|
|
Risk of Liver Transplantation
|
2 Events during the study period
|
12 Events during the study period
|
SECONDARY outcome
Timeframe: Up to 67 monthsPopulation: Weighted index population. The unit of analysis is Indexes.
The primary source for hospitalization for hepatic decompensation were Komodo Health claims. Risk is presented using the number of hospitalization for hepatic decompensation.
Outcome measures
| Measure |
OCA-treated
n=403 Indexes
PBC participants with a history of UDCA failure (inadequate response, intolerance, or discontinuation) who initiated OCA in the study window were included.
|
Non-OCA Treated
n=405 Indexes
PBC participants with a history of UDCA failure who were eligible but not treated with OCA for the study. This was a real-world study, and it was conducted in a population of participants selected from a database using weighted index methods.
|
|---|---|---|
|
Risk of Hospitalization for Hepatic Decompensation
|
6 Events during the study period
|
23 Events during the study period
|
Adverse Events
OCA-treated
Non-OCA Treated
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place