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

Phase 2
18 Years
72 Years
Not Enrolling
Primary Sclerosing Cholangitis

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Trial Information

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.

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.®)

Exclusion Criteria:

- Pregnant and/or lactating female

- Inability or unwillingness to practice contraceptive measures for the prevention of

- 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.

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Alkaline Phosphatase, Aspartate Aminotransferase, Alanine Aminotransferase

Outcome Description:

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 Time Frame:

6 months, baseline

Safety Issue:


Principal Investigator

Keith D Lindor, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Mayo Clinic


United States: Food and Drug Administration

Study ID:




Start Date:

April 2006

Completion Date:

May 2009

Related Keywords:

  • Primary Sclerosing Cholangitis
  • PSC
  • Thalidomide
  • Cholangitis
  • Cholangitis, Sclerosing



Mayo Clinic Rochester, Minnesota  55905