Thalidomide for the Treatment of Primary Sclerosing Cholangitis (PSC)

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT00953615
• Date of registration: 04/08/2009
• Prospective Registration: No
• Primary sponsor: Mayo Clinic
• Public title: Thalidomide for the Treatment of Primary Sclerosing Cholangitis (PSC)
• Scientific title: Open Label, Phase II Investigation of Thalidomide for the Treatment of Primary Sclerosing Cholangitis
• Date of first enrolment: April 2006
• Target sample size: 1
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT00953615
• Study type: Interventional
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Keith D Lindor, MD
• Affiliation: Mayo Clinic

Key inclusion & exclusion 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.



Age minimum: 18 Years
Age maximum: 72 Years
Gender: All

Health Condition(s) or Problem(s) studied

Primary Sclerosing Cholangitis

Intervention(s)

Drug: Thalidomide

Primary Outcome(s)

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

Secondary Outcome(s)

Overall Toxicity and Tolerability [Time Frame: 6 months]

Soluble Tumor Necrosis Factor - Alpha [Time Frame: 6 months, baseline]

Mayo Risk Score [Time Frame: 6 months]

Secondary ID(s)

342-06

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Celgene Corporation

Results

• Results available: Yes
• Date Posted: 27/02/2012
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00953615