Trial Outcomes & Findings for Pilot Study of Fenofibrate for PSC (NCT NCT01142323)

Results Overview

Serum alkaline phosphatase will be measured at entry and end of study

Recruitment status

TERMINATED

Study phase

PHASE1/PHASE2

Target enrollment

8 participants

Primary outcome timeframe

6 months

Results posted on

2018-02-06

Participant Flow

Participant milestones

Participant milestones
Measure	Fenofibrate fenofibrate 160 mg po daily
Overall Study STARTED	8
Overall Study COMPLETED	8
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Pilot Study of Fenofibrate for PSC

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Fenofibrate n=8 Participants fenofibrate 160 mg po daily
Age, Categorical <=18 years	0 Participants n=93 Participants
Age, Categorical Between 18 and 65 years	8 Participants n=93 Participants
Age, Categorical >=65 years	0 Participants n=93 Participants
Age, Continuous	43.7 years STANDARD_DEVIATION 8 • n=93 Participants
Sex: Female, Male Female	2 Participants n=93 Participants
Sex: Female, Male Male	6 Participants n=93 Participants
Region of Enrollment United States	8 participants n=93 Participants

PRIMARY outcome

Timeframe: 6 months

Serum alkaline phosphatase will be measured at entry and end of study

Outcome measures

Outcome measures
Measure	Baseline n=8 Participants Prior to drug intervention	At 6 Months n=8 Participants fenofibrate 160 mg/day
Serum Alkaline Phosphatase	290 U/L Standard Error 76	165 U/L Standard Error 19

SECONDARY outcome

Timeframe: 6 months

Population: Median baseline MRS was compared to Median MRS after 6 months of study drug

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.

Outcome measures

Outcome measures
Measure	Baseline n=8 Participants Prior to drug intervention	At 6 Months fenofibrate 160 mg/day
Mayo Risk Score for Primary Sclerosing Cholangitis Baseline	-0.41 score Interval -0.71 to 1.4	—
Mayo Risk Score for Primary Sclerosing Cholangitis After 6 months	-0.46 score Interval -0.96 to 1.8	—

Adverse Events

Fenofibrate

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Fenofibrate n=8 participants at risk fenofibrate 160 mg po daily
Skin and subcutaneous tissue disorders worsening of psoriasis	100.0% 1/1 • Number of events 1

Additional Information

Dr. Cynthia Levy

University of Miami

Phone: 305-243-2147

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place