Pilot Biomarker Trial to Evaluate the Efficacy of Itraconazole in Patients w/ Basal Cell Carcinomas

NCT ID: NCT01108094

Last Updated: 2018-11-13

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 29 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2010-04-30
• Study Completion Date: 2012-02-29

Brief Summary

Basal cell carcinomas (BCCs) are the most common human cancer in the US and affect over 1 million people. There is no effective drug to prevent basal cell carcinomas of the skin.

We hope to learn if an oral anti-fungal drug, itraconazole, might inhibit a marker of proliferation and a biomarker (tumor signaling pathway) of BCC development.

Itraconazole is an FDA-approved drug for the treatment of fungal infections of the skin, and has been used for the past 25 years with relatively few side effects. It has been shown in mice to reduce a BCC biomarker and to reduce growth of BCCs.

Thus, it may reduce BCC growth in humans.

Detailed Description

Participants with at least one BCC tumor measuring 4 mm or greater in diameter will be enrolled onto 1 of 2 treatment cohorts to receive oral itraconazole.

• Cohort A - 400 mg itraconazole (as 200 mg twice daily for 30 days), stratified by:

• Cohort A1 - Participants are vismodegib-naive.
• Cohort A2 - Participants had received prior vismodegib treatment.
• Cohort B - 200 mg itraconazole (as 100 mg twice daily, for up to 4 months). The objective of this cohort is to assess the anti-cancer efficacy of lower-dose extended treatment.
• Control Group - Tumors from untreated participants.

Conditions

Basal Cell Carcinoma (BCC); Skin Cancer

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Cohort A - Itraconazole 400 mg

Oral itraconazole 400 mg as 200 mg twice daily, for 1 month, stratified by prior vismodegib history

Group Type: EXPERIMENTAL

Itraconazole

Intervention Type: DRUG

• Cohort A: oral itraconazole 400 mg as 200 mg twice daily; for 1 month
• Cohort B: oral itraconazole 200 mg as 100 mg twice daily; for up to 3 months

Cohort B - Itraconazole 200 mg

Oral itraconazole 200 mg as 100 mg twice daily, for up to 3 months

Group Type: EXPERIMENTAL

Itraconazole

Intervention Type: DRUG

• Cohort A: oral itraconazole 400 mg as 200 mg twice daily; for 1 month
• Cohort B: oral itraconazole 200 mg as 100 mg twice daily; for up to 3 months

Untreated Control

Patients otherwise eligible but unwilling to take itraconazole were enrolled onto the control arm of the study and received no treatment

Group Type: NO_INTERVENTION

No interventions assigned to this group

Interventions

Itraconazole

• Cohort A: oral itraconazole 400 mg as 200 mg twice daily; for 1 month
• Cohort B: oral itraconazole 200 mg as 100 mg twice daily; for up to 3 months

Intervention Type: DRUG

Other Intervention Names

Sporanox

Eligibility Criteria

Inclusion Criteria

• At least one BCC tumor (greater than 4 mm in diameter) at any skin location, to be biopsied and surgically removed.
• Had at least one liver function test [eg, aspartate aminotransferase (AST), alanine aminotransferase (ALT)] with normal results in the last year.
• Consent to research use of their BCC tissue.
• Cohort A or B: Willing to take itraconazole during the 2 to 3 weeks between biopsy and surgical removal of BCC

Exclusion Criteria

• History or current hepatitis or other liver disease.
• Currently taking systemic medications that would affect BCC tumors (oral retinoids) or metabolism of itraconazole (anti-convulsants, corticosteroids)
• History or current evidence of malabsorption or liver disease within the one year prior to enrollment.
• History or current evidence of hyperthyroidism increasing metabolism of itraconazole
• Unable to attend to 2nd study visit at Stanford for Mohs surgical excision
• Current immunosuppression disease (cancer, autoimmune disease)
• Receiving immunosuppressive drugs
• Pregnant
• Lactating
• Any female actively trying to become pregnant

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Stanford University

OTHER

Sponsor Role: lead

Responsible Party

Jean Yuh Tang

Associate Professor of Dermatology

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Jean Y Tang, MD

Role: PRINCIPAL_INVESTIGATOR

Stanford University

Locations

Stanford University School of Medicine

Stanford, California, United States

Countries

United States

References

Kim DJ, Kim J, Spaunhurst K, Montoya J, Khodosh R, Chandra K, Fu T, Gilliam A, Molgo M, Beachy PA, Tang JY. Open-label, exploratory phase II trial of oral itraconazole for the treatment of basal cell carcinoma. J Clin Oncol. 2014 Mar 10;32(8):745-51. doi: 10.1200/JCO.2013.49.9525. Epub 2014 Feb 3.

Reference Type: RESULT

PMID: 24493717 (View on PubMed)

Other Identifiers

SU-04162010-5722

Identifier Type: OTHER

Identifier Source: secondary_id

SKIN0004-TX

Identifier Type: OTHER

Identifier Source: secondary_id

IRB-17365

Identifier Type: -

Identifier Source: org_study_id