Trial Outcomes & Findings for Thalidomide for the Treatment of Primary Sclerosing Cholangitis (PSC) (NCT NCT00953615)

Results Overview

The primary outcome was the change in serum liver biochemical parameter levels after 6 months of thalidomide when compared to baseline values. This was to be analyzed using the nonparametric Wilcoxon signed rank test of significance. This was based on the non-normal distribution of serum hepatic biochemical parameters among patients with PSC and the continuous nature of these variables.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

1 participants

Primary outcome timeframe

6 months, baseline

Results posted on

2012-02-27

Participant Flow

Participants were recruited between 4/6/2006 and 5/7/2009 at Mayo Clinic outpatient area.

Participant milestones

Participant milestones
Measure	Thalidomide Patients with primary sclerosing cholangitis (PSC) who met with enrollment criteria were treated with 400 mg in the evening for a duration of 6 months
Overall Study STARTED	1
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Thalidomide Patients with primary sclerosing cholangitis (PSC) who met with enrollment criteria were treated with 400 mg in the evening for a duration of 6 months
Overall Study Adverse Event	1

Baseline Characteristics

Thalidomide for the Treatment of Primary Sclerosing Cholangitis (PSC)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Thalidomide n=1 Participants Patients with primary sclerosing cholangitis (PSC) who met with enrollment criteria were treated with 400 mg in the evening for a duration of 6 months
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants
Region of Enrollment United States	1 participants n=5 Participants

PRIMARY outcome

Timeframe: 6 months, baseline

Population: Analysis was not performed because participant did not complete the study due to adverse events.

The primary outcome was the change in serum liver biochemical parameter levels after 6 months of thalidomide when compared to baseline values. This was to be analyzed using the nonparametric Wilcoxon signed rank test of significance. This was based on the non-normal distribution of serum hepatic biochemical parameters among patients with PSC and the continuous nature of these variables.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 6 months

Overall toxicity and tolerability were to be measured by the number of patients with development of neuropathy, increased liver biochemistries, drowsiness, dizziness and orthostatic hypotension.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 6 months

The Mayo Risk Score estimates the survival probability of a patient with primary sclerosing cholangitis based on the following variables: age, bilirubin, albumin, AST and history of variceal bleeding.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 6 months, baseline

Assessment of effect from thalidomide on soluble tumor necrosis factor - alpha compared to baseline values were to be performed at study conclusion.

Outcome measures

Outcome data not reported

Adverse Events

Thalidomide

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	Thalidomide n=1 participants at risk Patients with primary sclerosing cholangitis (PSC) who met with enrollment criteria were treated with 400 mg in the evening for a duration of 6 months
Nervous system disorders hand pain	100.0% 1/1 • Number of events 1 • 28 weeks
Gastrointestinal disorders loose stools	100.0% 1/1 • Number of events 1 • 28 weeks
Gastrointestinal disorders decreased appetite	100.0% 1/1 • Number of events 1 • 28 weeks
Gastrointestinal disorders aphthous stomatitis	100.0% 1/1 • Number of events 1 • 28 weeks
General disorders fatigue	100.0% 1/1 • Number of events 1 • 28 weeks
General disorders weight loss	100.0% 1/1 • Number of events 1 • 28 weeks
Cardiac disorders low blood pressure	100.0% 1/1 • Number of events 1 • 28 weeks

Additional Information

Keith D. Lindor, M.D.

Mayo Clinic

Phone: 507-284-2969

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place