Thalidomide for the Treatment of Primary Sclerosing Cholangitis (PSC)
The purpose of this study is to determine the safety and benefit of Thalidomide with primary sclerosing cholangitis (PSC). This is a six month study.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Open Label, Phase II Investigation of Thalidomide for the Treatment of Primary Sclerosing Cholangitis|
- Alkaline Phosphatase, Aspartate Aminotransferase, Alanine Aminotransferase [ Time Frame: 6 months, baseline ] [ Designated as safety issue: No ]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.
- Overall Toxicity and Tolerability [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]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.
- Mayo Risk Score [ Time Frame: 6 months ] [ Designated as safety issue: No ]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.
- Soluble Tumor Necrosis Factor - Alpha [ Time Frame: 6 months, baseline ] [ Designated as safety issue: No ]Assessment of effect from thalidomide on soluble tumor necrosis factor - alpha compared to baseline values were to be performed at study conclusion.
|Study Start Date:||April 2006|
|Study Completion Date:||May 2009|
|Primary Completion Date:||May 2009 (Final data collection date for primary outcome measure)|
Participants will be treated with Thalidomide, starting at a dose of 100 mg per day, increasing the dose by 100 mg every 14 days to a maximum of 400 mg per day.
Titrate to 400 mg daily for 6 months
Other Name: Thalomid
At entry, patients will have a complete history and physical, blood tests, ultrasound, and will complete questionnaires. Eligible patients will take Thalidomide 400 mg once a day in the evening. Patients will start a dose of 100 mg per day for two weeks, increasing by 100 mg per day every two weeks to a maximum dose of 400 mg per day for 6 months. Patients will return at 6 months for an evaluation, blood tests and completion of questionnaires. Blood tests will be performed by mailed-in kits at 3 months. Patients will receive weekly phone calls for the first 2 months and bi-monthly thereafter.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00953615
|United States, Minnesota|
|Rochester, Minnesota, United States, 55905|
|Principal Investigator:||Keith D Lindor, MD||Mayo Clinic|