Thalidomide for the Treatment of Primary Sclerosing Cholangitis (PSC)
Condition: Primary Sclerosing Cholangitis
MeSH terms: Cholangitis; Cholangitis, Sclerosing
Intervention: Thalidomide
Intervention description: Titrate to 400 mg daily for 6 months
Intervention MeSH terms: Thalidomide
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 1.0
Sex: All
Minimum age: 18 Years
Maximum age: 72 Years
Healthy volunteers: No
Keywords: PSC; Thalidomide

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.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Previous diagnosis of primary sclerosing cholangitis as defined by: serum alkaline
             phosphatase level greater than or equal to 1.5 times the upper limit of normal,
             negative serum antimitochondrial antibody test, cholangiography diagnostic of PSC
             without other etiology for biliary obstruction, and liver histology consistent with or
             diagnostic of PSC

          -  Patients must give written informed consent.

          -  Patients must be willing and able to comply with the most recent version of the
             FDA-mandated System for Thalidomide Education and Prescribing Safety (S.T.E.P.S.®)
             program.

        Exclusion Criteria:

          -  Pregnant and/or lactating female

          -  Inability or unwillingness to practice contraceptive measures for the prevention of
             pregnancy

          -  History of hypersensitivity reaction to thalidomide

          -  Inability to provide consent

          -  Findings suggestive of liver disease of other etiology such as primary biliary
             cirrhosis, chronic alcoholic liver disease, chronic hepatitis B and C infection,
             hemochromatosis, Wilson's disease, alpha-1-antitrypsin deficiency, autoimmune
             hepatitis, and cryptogenic liver disease

          -  Anticipated need for liver transplantation in one year from decompensated chronic
             liver disease or recurrent variceal bleeding, spontaneous hepatic encephalopathy, or
             refractory ascites

          -  Treatment with tacrolimus, cyclosporine, sirolimus, ursodeoxycholic acid,
             corticosteroids, colchicine, methotrexate, azathioprine, cyclosporine, chlorambucil,
             budesonide, pentoxifylline, nicotine, silymarin, vitamin E or pirfenidone in the
             preceding three months

          -  History of peripheral neuropathy

          -  Use of medications with significant drug-drug interactions with thalidomide

          -  History of Human Immunodeficiency Virus (HIV) positive status or Acquired
             Immunodeficiency Syndrome (AIDS)

          -  History of coexistent advanced malignancy

          -  History of coexistent severe cardiovascular disease

          -  History of coexistent severe renal disease

          -  History of current excessive or recent (within 6 months) alcohol use

          -  Any condition that, in the opinion of the investigators, would interfere with the
             patient's ability to complete the study safely or successfully

          -  History of thrombolytic events. Combination use with corticosteroids increases risk of
             deep vein thrombosis.