Basal Cell Carcinoma (BCC)Phase 2NCT01108094
Pilot Biomarker Trial to Evaluate the Efficacy of Itraconazole in Patients w/ Basal Cell Carcinomas
A Phase 2 study testing Itraconazole in people with Basal Cell Carcinoma (BCC) and Skin Cancer, run by Stanford University at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Basal Cell Carcinoma (BCC), Skin Cancer
- What is being tested?
- Itraconazole
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 29 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2010to Feb 2012
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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)
Show all 10Show fewer
- Receiving immunosuppressive drugs
- Pregnant
- Lactating
- Any female actively trying to become pregnant
Trial locations and contacts
1 site in 1 country.
United States1 site
Stanford University School of Medicine
Stanford, California, 94305
About this study
Official title: Pilot Biomarker Trial to Evaluate the Efficacy of Itraconazole in Patients With Basal Cell Carcinomas
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.
Read the 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
- Being tested
- Itraconazole
- Medical subject headings
- Carcinoma, Basal Cell, Skin Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ItraconazoleDrug
Also called Sporanox
- 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
| Study group | Role | What they receive |
|---|---|---|
| Cohort A - Itraconazole 400 mg | Experimental | Oral itraconazole 400 mg as 200 mg twice daily, for 1 month, stratified by prior vismodegib history Receives: Drug: Itraconazole |
| Cohort B - Itraconazole 200 mg | Experimental | Oral itraconazole 200 mg as 100 mg twice daily, for up to 3 months Receives: Drug: Itraconazole |
| Untreated Control | No intervention | Patients otherwise eligible but unwilling to take itraconazole were enrolled onto the control arm of the study and received no treatment |
What is being measured
The primary outcome is the main question the study is designed to answer.
Ki67 Tumor Proliferation Biomarker
Percent change in Ki67 tumor proliferation biomarker was assessed at baseline and after 1 month of treatment, for Cohort A1 (vismodegib-naïve participants receiving 400 mg as 200 mg twice daily) vs control patients. The outcome is expressed as the % change from baseline of cells with a positive signal after staining for Ki67.
- Paired analysis of tumors shows percent change between baseline (prior to treatment) and post itraconazole treatment in individual patients, \& is reported as the mean of the changes observed for those lesions for which both baseline and treated valued are available.
- Unpaired analysis shows percent change between individual tumors from control patients and itraconazole treated patients, and is reported as the change in mean of the group of baseline basal cell carcinoma (BCC) lesion measurements and the group of treated BCC lesion measurements.
Measured: 1 month
Secondary outcomes (3)
Change of GLI1 Tumor Biomarker
Tumor biomarker GLI1 (glioma-associated oncogene 1), part of the Hedgehog (HH) pathway, was assessed in vismodegib-naïve participants at baseline and after 1 month of treatment by quantitative polymerase chain reaction (qPCR). The relative expression of the biomarker was measured as the fold increase of GLI1 expression compared to that of housekeeping gene hypoxanthine-guanine phosphoribosyltransferase (HPRT), and the outcome was assessed as the percent change from the mean of the pre-treatment measurements to the mean of the post-treatment measurements. A negative mean indicates an overall reduction in GLI1 expression.
Measured: 1 month
Tumor Size
Tumor size was assessed by caliper measurement of the longest perpendicular diameters before and after itraconazole treatment, and determination of tumor area by multiplication of the measurements for each tumor. The outcome is expressed as the mean percent change in tumor area from baseline, with standard deviation. A negative value indicates a reduction in size.
Measured: Up to 3 months
Tumor Response
The following criteria for basal cell carcinoma (BCC) tumor response were used.
- Complete response (CR) means no visible evidence of any lesion consistent with BCC
- Partial response (PR) means less than CR, but there was a visible decrease in BCC tumor size
- No response (NR) / Stable Disease (SD) means no visible decrease in BCC tumor size
- Progressive disease (PD) means an increase in size or number of BCC tumor lesions
Treatment assessment was conducted on the basis of lesion photographs by a dermatologist investigator who was blinded to the assigned treatment.
Measured: End of treatment period: 1 month (Cohort A) or 2.3 months (mean for Cohort B)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Apr 2010 (estimated)
- Study startApr 2010
- Primary completionMar 2011
- Study completionFeb 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Basal Cell Carcinoma (BCC)Studies of the same condition, open and under way.
- More trials run by Stanford UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 29 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stanford University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jean Yuh Tang, Stanford University
- Investigators
- Jean Y Tang, MD, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01108094
- Sponsor's protocol ID
- IRB-17365
- Other IDs
- SU-04162010-5722, SKIN0004-TX
- Status last verified
- Nov 2018
- First submitted
- 19 Apr 2010
- Last updated
- 13 Nov 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 24493717
Study at a glance
From the registry record, last updated by the sponsor on 13 Nov 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Biomarker Trial to Evaluate the Efficacy of Itraconazole in Patients With Basal Cell Carcinomas
- ClinicalTrials.gov ID
- NCT01108094
- Acronym / sponsor ID
- IRB-17365
- Conditions
- Basal Cell Carcinoma (BCC), Skin Cancer
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 29 participants (actual)
- Lead sponsor
- Stanford UniversityUniversity, hospital or other
- Study start
- Apr 2010
- Primary completion
- Mar 2011 (actual)
- Study completion
- Feb 2012 (actual)
- First posted
- 21 Apr 2010 (estimated)
- Results
- Posted 13 Nov 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cohort A - Itraconazole 400 mg | Experimental | Drug: ItraconazoleOral itraconazole 400 mg as 200 mg twice daily, for 1 month, stratified by prior vismodegib history |
| Cohort B - Itraconazole 200 mg | Experimental | Drug: ItraconazoleOral itraconazole 200 mg as 100 mg twice daily, for up to 3 months |
| Untreated Control | No intervention | –Patients otherwise eligible but unwilling to take itraconazole were enrolled onto the control arm of the study and received no treatment |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (10 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ItraconazoleAlso: Sporanox* 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 | Drug | Cohort A - Itraconazole 400 mg, Cohort B - Itraconazole 200 mg |
Primary outcome
Ki67 Tumor Proliferation Biomarker
Time frame: 1 month
Percent change in Ki67 tumor proliferation biomarker was assessed at baseline and after 1 month of treatment, for Cohort A1 (vismodegib-naïve participants receiving 400 mg as 200 mg twice daily) vs control patients. The outcome is expressed as the % change from baseline of cells with a positive signal after staining for Ki67. * Paired analysis of tumors shows percent change between baseline (prior to treatment) and post itraconazole treatment in individual patients, \& is reported as the mean of the changes observed for those lesions for which both baseline and treated valued are available. * Unpaired analysis shows percent change between individual tumors from control patients and itraconazole treated patients, and is reported as the change in mean of the group of baseline basal cell carcinoma (BCC) lesion measurements and the group of treated BCC lesion measurements.
3 secondary outcomes
Change of GLI1 Tumor Biomarker
Time frame: 1 month
Tumor Size
Time frame: Up to 3 months
Tumor Response
Time frame: End of treatment period: 1 month (Cohort A) or 2.3 months (mean for Cohort B)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Nov 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 24493717Kim 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jean Y Tang, MD principal investigator
Stanford University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Itraconazole in people with Basal Cell Carcinoma (BCC) and Skin Cancer, run by Stanford University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 Nov 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 13 Nov 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Ki67 Tumor Proliferation Biomarker (time frame: 1 month). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01108094, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Nov 2018.