An Early Phase 1 interventional study of Ketoconazole and Laboratory Biomarker Analysis in Malignant Neoplasm, sponsored by Mayo Clinic. Completed at 6 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-09-21.
Sponsored by Mayo Clinic · Early Phase 1, Interventional, and Supportive care
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This early phase I trial studies the side effects of ketoconazole and how well it works in treating participants with ongoing EGFR inhibitor-induced rash. Ketoconazole may reduce the symptoms related to EGFR inhibitor therapy and improve EGFR inhibitor-induced rash.
PRIMARY OBJECTIVES:
I. To demonstrate that topical ketoconazole, an anti-androgen, palliates EGFR inhibitor-induced rash within a group of racially diverse cancer patients.
II. To explore the role of ribonucleic acid (RNA) sequencing to identify other targets that might be used at a later date for rash palliation.
III. To evaluate toxicities associated with topical ketoconazole.
OUTLINE: Participants are randomized to 1 of 2 arms.
ARM I: Participants apply ketoconazole topically twice daily (BID) on days 1-28.
ARM II: Participants apply placebo topically BID on days 1-28.
After completion of study treatment, participants are followed up at 1 week.
10,824 studies on the registry are indexed under Neoplasms; 2,325 are open to participants now.
This study's enrollment of 58 is close to the median of 60 across 7,706 interventional studies indexed under Neoplasms.
Browse Neoplasms studies →Mayo Clinic is the lead sponsor of 3,271 studies on the registry; 672 are open to participants now.
Of its 452 completed or terminated interventional studies of FDA-regulated products, 321 (71%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants apply ketoconazole topically BID on days 1-28.
Drug: Ketoconazole · Other: Laboratory Biomarker Analysis · Other: Quality-of-Life Assessment · Other: Questionnaire Administration
Participants apply placebo topically BID on days 1-28.
Other: Laboratory Biomarker Analysis · Other: Placebo Administration · Other: Quality-of-Life Assessment · Other: Questionnaire Administration
Applied topically
Also known as: Fungarest, Fungoral, Ketoderm, Ketoisdin, Nizoral, Orifungal M, Panfungol, R-41400, Xolegel
Correlative studies
Applied topically
Ancillary studies
Also known as: Quality of Life Assessment
Ancillary studies
Proportion of patients who report an improvement in skin rash
Assessed by Skindex-16. Will be estimated using the cumulative incidence function with time to improvement defined as the time from randomization to the first of the two consecutive weeks of improved symptom. The cumulative incidence of rash improvement after 4 weeks of treatment will be summarized separately by treatment arm. The difference in rash improvement incidences will be estimated and will be compared using two-sample Z-test.
Time frame: Up to 4 weeks
Incidence of skin toxicity
As measured by the Skindex-16. Responses to the Skindex-16 will be categorized into three subscales: symptom, emotional, and functional. Analysis of the total scales and subscales of the Skindex-16 will involve a t-test and Wilcoxon rank sum procedures (as appropriate) at each time point as well as linear mixed modeling. Descriptive factors will be used as covariates in the modeling analysis. The change from baseline in the total score and subscales of the Skindex-16 will be compared between two arms by a two-sample, two-sided t-test. If there is evidence of non-normality (via Shapiro-Wilk testing), a non-parametric procedure such as Wilcoxon rank sum will be used.
Time frame: Up to 4 weeks
Incidence of skin toxicity
As measured by the Skin Toxicity Assessment Tool (STAT). Responses to the STAT will be categorized into three subscales: symptom, emotional, and functional. Analysis of the total scales and subscales of the STAT will involve a t-test and Wilcoxon rank sum procedures (as appropriate) at each time point as well as linear mixed modeling. Descriptive factors will be used as covariates in the modeling analysis. The change from baseline in the total score and subscales of the STAT will be compared between two arms by a two-sample, two-sided t-test. If there is evidence of non-normality (via Shapiro-Wilk testing), a non-parametric procedure such as Wilcoxon rank sum will be used.
Time frame: Up to 4 weeks
Incidence of adverse events for ketoconazole
Adverse events will be tabulated by treatment arm. Frequencies of various types of adverse events (AEs) will be compared using Fisher's exact test. Will explore the difference in reliability of the direct versus (vs.) indirect AE attribution approaches in the placebo arm.
Time frame: Up to 4 weeks
Change in PLA2G4D, PLOD2, and SALL4
Assessed by ribonucleic acid (RNA) sequencing. Will explore the association between baseline/change in PLA2G4D, PLOD2, and SALL4 with rash improvement. Baseline gene expression level and change from baseline in gene expression level for the androgen-related genes such as PLA2G4D, PLOD2, and SALL4 will be compared between arms by t-test and Wilcoxon rank sum procedures (as appropriate). Mixed Models for Logistic Regression will also be implemented to explore the association between PLA2G4D, PLOD2, and SALL4 with rash improvement by adjusting the baseline gene expression level and change from baseline in gene expression levels for each gene. Descriptive factors will be used as covariates in the modeling analysis.
Time frame: Baseline up to 4 weeks
Ran from Sep 2018 to primary completion in Sep 2024, 71 months.
Of 227 non-industry phase 1 interventional studies under Neoplasms, half reached primary completion within 48 months of starting (the median), counting those still running; a quarter within 28 and three quarters within 88. This study's 71 months is above that.
Counting every sponsor, half of 904 studies reached it within 40 months; a quarter within 23 and three quarters within 64.
Neoplasms covers many different conditions, not one, so the studies compared here vary widely. Read these figures as a rough guide.
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This study is completed, as verified in Oct 2025. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Ketoconazole