Open Label, Phase II Investigation of Thalidomide for the Treatment of Primary Sclerosing Cholangitis
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.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Alkaline Phosphatase, Aspartate Aminotransferase, Alanine Aminotransferase
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.
6 months, baseline
Keith D Lindor, MD
United States: Food and Drug Administration
|Mayo Clinic||Rochester, Minnesota 55905|