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Thalidomide for the Treatment of Primary Sclerosing Cholangitis (PSC)

This study has been terminated.
(Lack of enrollment)
Sponsor:
Collaborator:
Celgene Corporation
Information provided by:
Mayo Clinic
ClinicalTrials.gov Identifier:
NCT00953615
First received: August 4, 2009
Last updated: January 24, 2012
Last verified: January 2012

August 4, 2009
January 24, 2012
April 2006
May 2009   (final data collection date for primary outcome measure)
Alkaline Phosphatase, Aspartate Aminotransferase, Alanine Aminotransferase [ Time Frame: 6 months, baseline ] [ Designated as safety issue: No ]
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.
Response will be measured by serum hepatic biochemical parameter assessment at the end of treatment [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
Complete list of historical versions of study NCT00953615 on ClinicalTrials.gov Archive Site
  • Overall Toxicity and Tolerability [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    Assessment of effect from thalidomide on soluble tumor necrosis factor - alpha compared to baseline values were to be performed at study conclusion.
  • Overall Toxicity and tolerability [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
  • Mayo Risk Score [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
  • Health-related quality of life assessed by validated instruments [ Time Frame: 6 months ] [ Designated as safety issue: No ]
  • Soluble tumor necrosis factoe - alpha levels I [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
Not Provided
Not Provided
 
Thalidomide for the Treatment of Primary Sclerosing Cholangitis (PSC)
Open Label, Phase II Investigation of Thalidomide for the Treatment of Primary Sclerosing Cholangitis

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.

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.

Interventional
Phase 2
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Primary Sclerosing Cholangitis
Drug: Thalidomide
Titrate to 400 mg daily for 6 months
Other Name: Thalomid
Experimental: Thalidomide
Participants will be treated with Thalidomide, starting at a dose of 100 mg per day, increasing the dose by 100 mg every 14 days to a maximum of 400 mg per day.
Intervention: Drug: Thalidomide
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Terminated
1
May 2009
May 2009   (final data collection date for primary outcome measure)

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.
Both
18 Years to 72 Years
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT00953615
342-06
Yes
Dr. K. Lindor, Mayo Clinic
Mayo Clinic
Celgene Corporation
Principal Investigator: Keith D Lindor, MD Mayo Clinic
Mayo Clinic
January 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP