Could a skin gel help fight breast cancer? early trial tests endoxifen
NCT ID NCT03317405
First seen Jun 25, 2026 · Last updated Sep 03, 2026 · Updated 3 times
Summary
This early-phase trial is testing a gel form of the drug endoxifen, applied to the skin, in 32 women scheduled for breast surgery. The goal is to see if the gel is safe and how much of the drug gets into breast tissue and blood. If successful, it might offer a gentler way to treat or prevent breast cancer in the future.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- endoxifen hydrochloride gel
- What this could lead to
- If it works, this could lead to a topical treatment that lowers breast cancer risk or helps manage early-stage disease without the side effects of pills.
- What could go wrong
- This is a very early (Phase 1) trial with only 32 participants, so it primarily checks safety and dosing. It may not show any benefit against cancer, and skin irritation at the application site is a possible risk.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
-
32 people
The number who actually took part.
- Started
-
Oct 2018
- Expected to finish
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Aug 2027
An estimate. End dates often move.
- Lead sponsor
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A government research agency
The lead sponsor is the US National Institutes of Health.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Female participants only
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Women scheduled for unilateral or bilateral mastectomy for breast cancer therapy, pathology confirmed stage 0-III (including ductal carcinoma in situ), or prophylaxis (BRCA1/2 mutation carriers, women with strong family history or lobular carcinoma in situ or other conditions where prophylactic mastectomy has been elected) * Age \>= 18 years (since breast cancer is not a pediatric disease and no safety data are available for ENX use in children) * Eastern Cooperative Oncology Group (ECOG) performance status =\< 1 (Karnofsky \>= 70%) * Total bilirubin \< 1.5 x upper limit of normal (ULN) (in women with prior documented bilirubin elevations consistent with Gilbert's syndrome, total bilirubin up to 3 x ULN will be allowed) * Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase \[SGOT\]) \< 2.5 x ULN * Alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase \[SGPT\]) \< 2.5 x ULN * Creatinine \< 2 x ULN * Alkaline phosphatase \< 2.5 x ULN * Blood urea nitrogen \< 2 x ULN * Ability to understand and the willingness to sign a written informed consent document which includes a requirement to apply study agent to sensitive body parts daily * Willingness and ability to schedule mastectomy 21-28 days following start of study agent; women with breast implants may participate * Willingness to avoid exposing breast skin to natural or artificial sunlight (i.e. tanning beds) for the duration of study agent dosing * Negative urine or serum pregnancy test result, for participants of child bearing potential; female of child-bearing potential is any woman (regardless of sexual orientation, whether she has undergone a tubal ligation, or remains celibate by choice) who meets the following criteria: has not undergone a hysterectomy or bilateral oophorectomy; AND has had a menstrual period at any time in the preceding 12 consecutive months) * The effects of topical ENX gel on the developing human fetus are unknown; for this reason, women of child-bearing potential and their male partners must agree to use effective forms of birth control (abstinence is not an allowed method) prior to study entry and for the duration of study participation; should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her study physician immediately Exclusion Criteria: * The presence of gross skin invasion/ulceration by the breast cancer, or inflammatory changes with skin edema AND erythema; Note: Paget's disease is permitted * Women receiving a "nipple delay" procedure prior to mastectomy * Women with skin diseases (psoriasis, eczema) * A history of thromboembolic disorder * Endometrial intraepithelial neoplasia (also known as atypical hyperplasia) or a high risk of uterine cancer, defined here as known carriers of Lynch syndrome mutations (MLH1, MSH2, MSH6, PMS2) * Participants may not have received any other investigational agents in the previous 3 months * History of allergic reactions attributed to compounds of similar chemical or biologic composition to tamoxifen * Taken tamoxifen or other selective estrogen/progesterone receptor modulators (SERMs/SPRMs) within two years prior to entering study or been required to discontinue SERM therapy due to thromboembolic or uterine toxicity * Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements * History of prior breast cancer-specific therapy within the previous 2 years (chemotherapy, radiation, anti-HER2 agents, endocrine agents, everolimus, CDK4-6 inhibitors); previous unilateral radiation of the contralateral side in women scheduled for mastectomy is allowed; study gel will be applied to both breasts * History of prior mastectomy * Pregnant or breastfeeding * Patients receiving neoadjuvant chemotherapy with curative intent * Men are excluded from this study since breast cancer is men is rare and there are no data regarding skin penetration of topical breast cancer prevention agents through male chest wall skin (which is thicker and harrier than female chest wall skin) * Current users of other topical medications on the breast skin must be willing and able to discontinue use for the duration of participation; body lotion and other non-medicinal topical compounds may be applied \> 4 hours after study gel application
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Cedars-Sinai Medical Center
Los Angeles, California, 90048, United States
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Memorial Sloan Kettering Cancer Center
New York, New York, 10065, United States
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Northwestern University
Chicago, Illinois, 60611, United States
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Other studies related to the condition(s) this trial covers.
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