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

Stanford University

Status and phase: Completed; Phase 2

Conditions: Skin Cancer; Basal Cell Carcinoma (BCC)

Treatments: Drug: Itraconazole

Study type: Interventional

Funder types: Other

Identifiers

NCT01108094

IRB-17365

SKIN0004-TX (Other Identifier)

SU-04162010-5722 (Other Identifier)

Details and patient eligibility

About

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.

Full 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.

Enrollment: 29 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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29 participants in 3 patient groups

Cohort A - Itraconazole 400 mg

Experimental group

Description:

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

Treatment:

Drug: Itraconazole

Cohort B - Itraconazole 200 mg

Experimental group

Description:

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

Treatment:

Drug: Itraconazole

Untreated Control

No Intervention group

Description:

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