Thalidomide for the Treatment of Primary Sclerosing Cholangitis (PSC)

January 24, 2012 updated by: Mayo Clinic

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.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Detailed Description

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.

Study Type

Interventional

Enrollment (Actual)

1

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Mayo Clinic

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 72 years (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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.
Drug: Thalidomide
Titrate to 400 mg daily for 6 months
Other Names:
  • Thalomid

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
6 months, baseline

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
6 months
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.
6 months
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.
6 months, baseline

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Mayo Clinic

Collaborators

Celgene Corporation

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

April 1, 2006

Primary Completion (ACTUAL)

May 1, 2009

Study Completion (ACTUAL)

May 1, 2009

Study Registration Dates

First Submitted

August 4, 2009

First Submitted That Met QC Criteria

August 5, 2009

First Posted (ESTIMATE)

August 6, 2009

Study Record Updates

Last Update Posted (ESTIMATE)

February 27, 2012

Last Update Submitted That Met QC Criteria

January 24, 2012

Last Verified

January 1, 2012

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 342-06

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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