Clinical Trial: Thalidomide for the Treatment of Primary Sclerosing Cholangitis (PSC)

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

Official Title: Open Label, Phase II Investigation of Thalidomide for the Treatment of Primary Sclerosing Cholangitis

Brief Summary: 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.

Detailed Summary: 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.
Sponsor: Mayo Clinic

Current Primary Outcome: Alkaline Phosphatase, Aspartate Aminotransferase, Alanine Aminotransferase [ Time Frame: 6 months, baseline ]

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.


Original Primary Outcome: Response will be measured by serum hepatic biochemical parameter assessment at the end of treatment [ Time Frame: 6 months ]

Current Secondary Outcome:

  • Overall Toxicity and Tolerability [ Time Frame: 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.
  • Mayo Risk Score [ Time Frame: 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.
  • Soluble Tumor Necrosis Factor - Alpha [ Time Frame: 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.


Original Secondary Outcome:

  • Overall Toxicity and Tolerability [ Time Frame: 6 months ]
  • Mayo Risk Score [ Time Frame: 6 months ]
  • Health-related quality of life assessed by validated instruments [ Time Frame: 6 months ]
  • Soluble tumor necrosis factoe - alpha levels I [ Time Frame: 6 months ]


Information By: Mayo Clinic

Dates:
Date Received: August 4, 2009
Date Started: April 2006
Date Completion:
Last Updated: January 24, 2012
Last Verified: January 2012