Malignant NeoplasmEarly Phase 1NCT03471364
Ketoconazole in Treating Participants With Ongoing EGFR Inhibitor-Induced Rash
A Early Phase 1 study testing Ketoconazole, Laboratory Biomarker Analysis and Quality-of-Life Assessment in people with Malignant Neoplasm, run by Mayo Clinic at 6 sites in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Malignant Neoplasm
- What is being tested?
- Ketoconazole, Laboratory Biomarker Analysis and Quality-of-Life Assessment
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 58 participants
- Where
- 6 sitesUnited States
- Study dates
- 25 Sep 2018to 15 Sep 2024
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
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
- Patient has developed a rash or symptoms of a rash (cutaneous burning) characteristic of an EGFR inhibitor (health-care provider report of the rash with no other documentation is permitted)
- Patient is anticipated to continue for at least 28 days with an EGFR inhibitor or restart =< 14 days of registration and continue for at least 28 days
- Mayo only: Patient is willing to provide a skin biopsy for correlative research; Note: Can be waived with permission of study chair (documentation such as an email must be provided)
- Patient must complete baseline quality of life (QOL) packet
Who cannot join
- Patient has a prior allergy or intolerance of ketoconazole
- Patient has an allergy or intolerance to sulfites
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
Carle on Vermilion
Danville, Illinois, 61832
Mayo Clinic in Rochester
Rochester, Minnesota, 55905
University of Rochester
Rochester, New York, 14642
Park Nicollet Frauenshuh Cancer Center
Saint Louis Park, Minnesota, 55426
Regions Hospital
Saint Paul, Minnesota, 55101
Carle Cancer Center
Urbana, Illinois, 61801
About this study
Official title: Double-Blinded, Placebo-Controlled Trial to Explore the Anti-Androgen, Ketoconazole, for Treating Patients With an Ongoing Epidermal Growth Factor Receptor (EGFR) Inhibitor-Induced Rash
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.
Read the detailed description
PRIMARY OBJECTIVES:
- 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.
- Conditions
- Malignant Neoplasm
- Being tested
- Ketoconazole, Laboratory Biomarker Analysis, Placebo Administration, Quality-of-Life Assessment, Questionnaire Administration
- Medical subject headings
- Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KetoconazoleDrug
Also called Fungarest, Fungoral, Ketoderm, Ketoisdin
Applied topically
Laboratory Biomarker AnalysisOther
Correlative studies
Placebo AdministrationOther
Applied topically
Quality-of-Life AssessmentOther
Also called Quality of Life Assessment
Ancillary studies
Questionnaire AdministrationOther
Ancillary studies
| Study group | Role | What they receive |
|---|---|---|
| Arm I (ketoconazole) | Experimental | Participants apply ketoconazole topically BID on days 1-28. Receives: Drug: Ketoconazole; Other: Laboratory Biomarker Analysis; Other: Quality-of-Life Assessment; Other: Questionnaire Administration |
| Arm II (placebo) | Placebo comparator | Participants apply placebo topically BID on days 1-28. Receives: Other: Laboratory Biomarker Analysis; Other: Placebo Administration; Other: Quality-of-Life Assessment; Other: Questionnaire Administration |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Up to 4 weeks
Secondary outcomes (3)
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.
Measured: 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.
Measured: 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.
Measured: Up to 4 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Mar 2018
- Study start25 Sep 2018
- Primary completion15 Sep 2024
- Study completion15 Sep 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Malignant NeoplasmStudies of the same condition, open and under way.
- More trials run by Mayo ClinicThe 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
- Early Phase 1
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 58 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Aminah Jatoi, M.D., Principal investigator, Mayo ClinicElizabeth J. Cathcart-Rake, M.D., Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03471364
- Sponsor's protocol ID
- MC17C1
- Other IDs
- NCI-2018-00355, MC17C1, R01CA207183, 17-007786
- Status last verified
- Oct 2025
- First submitted
- 6 Mar 2018
- Last updated
- 21 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Double-Blinded, Placebo-Controlled Trial to Explore the Anti-Androgen, Ketoconazole, for Treating Patients With an Ongoing Epidermal Growth Factor Receptor (EGFR) Inhibitor-Induced Rash
- ClinicalTrials.gov ID
- NCT03471364
- Acronym / sponsor ID
- MC17C1
- Conditions
- Malignant Neoplasm
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 58 participants (actual)
- Lead sponsor
- Mayo ClinicUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- 25 Sep 2018 (actual)
- Primary completion
- 15 Sep 2024 (actual)
- Study completion
- 15 Sep 2024 (actual)
- First posted
- 20 Mar 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
PRIMARY OBJECTIVES:
- 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.
Study design
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm I (ketoconazole) | Experimental | Drug: Ketoconazole, Other: Laboratory Biomarker Analysis, Other: Quality-of-Life Assessment, Other: Questionnaire AdministrationParticipants apply ketoconazole topically BID on days 1-28. |
| Arm II (placebo) | Placebo comparator | Other: Laboratory Biomarker Analysis, Other: Placebo Administration, Other: Quality-of-Life Assessment, Other: Questionnaire AdministrationParticipants apply placebo topically BID on days 1-28. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Patient has developed a rash or symptoms of a rash (cutaneous burning) characteristic of an EGFR inhibitor (health-care provider report of the rash with no other documentation is permitted)
- Patient is anticipated to continue for at least 28 days with an EGFR inhibitor or restart =< 14 days of registration and continue for at least 28 days
- Mayo only: Patient is willing to provide a skin biopsy for correlative research; Note: Can be waived with permission of study chair (documentation such as an email must be provided)
- Patient must complete baseline quality of life (QOL) packet
Key exclusion criteria (2 listed)
- Patient has a prior allergy or intolerance of ketoconazole
- Patient has an allergy or intolerance to sulfites
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 |
|---|---|---|
| KetoconazoleAlso: Fungarest, Fungoral, Ketoderm, KetoisdinApplied topically | Drug | Arm I (ketoconazole) |
| Laboratory Biomarker AnalysisCorrelative studies | Other | Arm I (ketoconazole), Arm II (placebo) |
| Placebo AdministrationApplied topically | Other | Arm II (placebo) |
| Quality-of-Life AssessmentAlso: Quality of Life AssessmentAncillary studies | Other | Arm I (ketoconazole), Arm II (placebo) |
| Questionnaire AdministrationAncillary studies | Other | Arm I (ketoconazole), Arm II (placebo) |
Primary outcome
Proportion of patients who report an improvement in skin rash
Time frame: Up to 4 weeks
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.
3 secondary outcomes
Incidence of skin toxicity
Time frame: Up to 4 weeks
Incidence of skin toxicity
Time frame: Up to 4 weeks
Incidence of adverse events for ketoconazole
Time frame: Up to 4 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Sep 2026.
- Mayo Clinic Clinical TrialsLink provided by the sponsor.
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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.
Aminah Jatoi, M.D. principal investigator
Mayo Clinic
Elizabeth J. Cathcart-Rake, M.D. principal investigator
Mayo Clinic
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Ketoconazole, Laboratory Biomarker Analysis and Quality-of-Life Assessment in people with Malignant Neoplasm, run by Mayo Clinic at 6 sites 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 2 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 21 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites 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. Some participants in this study may receive a placebo. 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Proportion of patients who report an improvement in skin rash (time frame: Up to 4 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 21 Sep 2026.