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

History of Changes

Tracking Information

First Received Date ^ICMJE

August 4, 2009

Last Updated Date

January 24, 2012

Start Date ^ICMJE

April 2006

Primary Completion Date

May 2009 (final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

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.

Original Primary Outcome Measures ^ICMJE

Response will be measured by serum hepatic biochemical parameter assessment at the end of treatment [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]

Change History

Complete list of historical versions of study NCT00953615 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

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.

Original Secondary Outcome Measures ^ICMJE

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 ]

Current Other Outcome Measures ^ICMJE

Not Provided

Original Other Outcome Measures ^ICMJE

Not Provided

Descriptive Information

Brief Title ^ICMJE

Thalidomide for the Treatment of Primary Sclerosing Cholangitis (PSC)

Official Title ^ICMJE

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

Brief 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.

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 ^ICMJE

Interventional

Study Phase

Phase 2

Study Design ^ICMJE

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

Condition ^ICMJE

Primary Sclerosing Cholangitis

Intervention ^ICMJE

Drug: Thalidomide

Titrate to 400 mg daily for 6 months

Other Name: Thalomid

Study Arm (s)

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

Publications *

Not Provided

* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Terminated

Enrollment ^ICMJE

Completion Date

May 2009

Primary Completion Date

May 2009 (final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

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.

Gender

Both

Ages

18 Years to 72 Years

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact information is only displayed when the study is recruiting subjects

Location Countries ^ICMJE

United States

Administrative Information

NCT Number ^ICMJE

NCT00953615

Other Study ID Numbers ^ICMJE

342-06

Has Data Monitoring Committee

Yes

Responsible Party

Dr. K. Lindor, Mayo Clinic

Study Sponsor ^ICMJE

Mayo Clinic

Collaborators ^ICMJE

Celgene Corporation

Investigators ^ICMJE

Principal Investigator:

Keith D Lindor, MD

Mayo Clinic

Information Provided By

Mayo Clinic

Verification Date

January 2012

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

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