Thalidomide for the Treatment of Primary Sclerosing Cholangitis (PSC)

NCT00953615 | Terminated Phase 2 Results DMC

• 1 other identifier: 342-06
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

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.

Conditions

Primary Sclerosing Cholangitis

Keywords

PSC; Thalidomide

Outcome Measures

Primary Outcomes (1)

• Alkaline Phosphatase, Aspartate Aminotransferase, Alanine Aminotransferase

  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 Outcomes (3)

• Overall Toxicity and Tolerability

  6 months

• Mayo Risk Score

  6 months

• Soluble Tumor Necrosis Factor - Alpha

  6 months, baseline

Study Arms (1)

Thalidomide

EXPERIMENTAL

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

Interventions

Thalidomide DRUG

Titrate to 400 mg daily for 6 months

Also known as: Thalomid

Thalidomide

Eligibility Criteria

• Age: 18 Years - 72 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Mayo Clinic: lead
• Celgene Corporation: collaborator

Study Sites (1)

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

MeSH Terms

Conditions

Cholangitis, Sclerosing

Interventions

Thalidomide

Condition Hierarchy (Ancestors)

Cholangitis; Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases

Intervention Hierarchy (Ancestors)

Phthalimides; Phthalic Acids; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Piperidones; Piperidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Isoindoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Limitations and Caveats

Only one subject was enrolled, so study was terminated.

Results Point of Contact

• Title: Keith D. Lindor, M.D.
• Organization: Mayo Clinic

lindor.keith@mayo.edu

507-284-2969

Study Officials

• Keith D Lindor, MD

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: August 4, 2009
• First Posted: August 6, 2009
• Study Start: April 1, 2006
• Primary Completion: May 1, 2009
• Study Completion: May 1, 2009
• Last Updated: February 27, 2012
• Results First Posted: February 27, 2012

Record last verified: 2012-01

Locations

US (1)