Primary Sclerosing Cholangitis
Conditions
Brief summary
The purpose of this study is to determine whether fenofibrate is safe and effective in the treatment primary sclerosing cholangitis (PSC).
Interventions
DRUGfenofibrate
160 mg po daily
Sponsors
University of Florida
University of Miami
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
* Male and female patients of 18 to 75 years old * Confirmed diagnosis of PSC including typical findings of stricturing and dilatations of the intra and/or extrahepatic biliary ducts in radiographic exam, (endoscopic retrograde cholangiopancreatography -ERCP, percutaneous cholangiogram - PTC or magnetic resonance cholangiopancreatography- MRCP) * Serum alkaline phosphatase levels elevated to at least 1.5 times the upper limit of normal.
Exclusion criteria
* Hypersensitivity to fenofibrate * Prisoners and institutionalized subjects * Pregnant or nursing women * Anticipated need for liver transplantation in one year * Recipients of liver transplantation * Recurrent variceal hemorrhage, uncontrolled encephalopathy or refractory ascites * Co-existing liver diseases including auto-immune and viral hepatitis * Acute or chronic renal failure, defined as glomerular filtration rate (GFR)\< 60 ml/min, GFR calculated using the Modification of Diet in Renal Disease (MDRF) GFR calculator * Known cholecystitis * Current use of statins * Current use of coumadin anticoagulant therapy * Previous history of, or known high risk for, venous thromboembolism,
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Serum Alkaline Phosphatase | 6 months | Serum alkaline phosphatase will be measured at entry and end of study |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Mayo Risk Score for Primary Sclerosing Cholangitis | 6 months | The Mayo risk score (MRS), which is a composite of several variables (age, bilirubin, albumin, aspartate aminotransferase\[AST\] and h/o variceal bleeding), will be measured at entry and end of study. The MRS is a mathematically calculated risk score. MRS does not have a theoretical lower/upper bound (that is, no theoretical minimum and maximum values). Mayo risk score \<=0 indicates low risk of death. MRS between 0 and 2 indicates intermediate risk, and greater than 2 indicates high risk. There is no known range for this score. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Fenofibrate fenofibrate 160 mg po daily | 8 |
| Total | 8 |
Baseline characteristics
| Characteristic | Fenofibrate |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 8 Participants |
| Age, Continuous | 43.7 years STANDARD_DEVIATION 8 |
| Region of Enrollment United States | 8 participants |
| Sex: Female, Male Female | 2 Participants |
| Sex: Female, Male Male | 6 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 1 / 8 |
| serious Total, serious adverse events | 0 / 8 |
Outcome results
Primary
Serum Alkaline Phosphatase
Serum alkaline phosphatase will be measured at entry and end of study
Time frame: 6 months
| Arm | Measure | Value (MEDIAN) | Dispersion |
|---|---|---|---|
| Baseline | Serum Alkaline Phosphatase | 290 U/L | Standard Error 76 |
| At 6 Months | Serum Alkaline Phosphatase | 165 U/L | Standard Error 19 |
p-value: 0.008Matched pairs
Secondary
Mayo Risk Score for Primary Sclerosing Cholangitis
The Mayo risk score (MRS), which is a composite of several variables (age, bilirubin, albumin, aspartate aminotransferase\[AST\] and h/o variceal bleeding), will be measured at entry and end of study. The MRS is a mathematically calculated risk score. MRS does not have a theoretical lower/upper bound (that is, no theoretical minimum and maximum values). Mayo risk score \<=0 indicates low risk of death. MRS between 0 and 2 indicates intermediate risk, and greater than 2 indicates high risk. There is no known range for this score.
Time frame: 6 months
Population: Median baseline MRS was compared to Median MRS after 6 months of study drug
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Baseline | Mayo Risk Score for Primary Sclerosing Cholangitis | Baseline | -0.41 score |
| Baseline | Mayo Risk Score for Primary Sclerosing Cholangitis | After 6 months | -0.46 score |
p-value: 0.46Wilcoxon (Mann-Whitney)