Phase I Trial of Endoxifen Gel Versus Placebo in Women Undergoing Breast Surgery
6 other identifiers
interventional
32
1 country
3
Brief Summary
This randomized phase I trial studies the side effects and best dose of endoxifen hydrochloride in treating participants who are undergoing breast surgery. Endoxifen hydrochloride may treat or reduce the risk of breast cancer.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_1
Started Oct 2018
Longer than P75 for phase_1
3 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
October 20, 2017
CompletedFirst Posted
Study publicly available on registry
October 23, 2017
CompletedStudy Start
First participant enrolled
October 31, 2018
CompletedPrimary Completion
Last participant's last visit for primary outcome
February 22, 2021
CompletedResults Posted
Study results publicly available
June 13, 2023
CompletedStudy Completion
Last participant's last visit for all outcomes
August 12, 2026
ExpectedMay 4, 2026
February 1, 2026
2.3 years
October 20, 2017
January 31, 2023
May 1, 2026
Conditions
Outcome Measures
Primary Outcomes (1)
Number of Participants With Dermal Toxicity on Breast Skin at the Application Site
Number of participants with dermal toxicity on breast skin at the transdermal gel application site. Will be assessed by Common Terminology Criteria for Adverse Events version 4.
Up to 60 days
Secondary Outcomes (4)
Drug Concentration in Tissue
up to 28 days
Drug Concentration in Plasma
baseline and up to 28 days
Change in Plasma Estrogenic and Coagulation Parameters
baseline and up to 28 days
Change in Pre-therapy and Post-therapy Symptoms Captured by BESS Questionnaire
baseline and up to 60 days
Study Arms (2)
Cohort I (endoxifen hydrochloride)
EXPERIMENTALParticipants apply endoxifen hydrochloride gel to both breast skin and keep it untouched over at least 4 hours once daily for 21-28 days before breast surgery.
Cohort II (placebo)
PLACEBO COMPARATORParticipants apply placebo to both breast skin and keep it untouched over at least 4 hours once daily for 21-28 days before breast surgery.
Interventions
Apply to the skin
Ancillary studies
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (3)
Cedars-Sinai Medical Center
Los Angeles, California, 90048, United States
Northwestern University
Chicago, Illinois, 60611, United States
Memorial Sloan Kettering Cancer Center
New York, New York, 10065, United States
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Seema A. Khan
- Organization
- Northwestern University
Study Officials
- PRINCIPAL INVESTIGATOR
Seema A Khan
Northwestern University
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- LTE60
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- RANDOMIZED
- Masking
- TRIPLE
- Who Masked
- PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- PREVENTION
- Intervention Model
- PARALLEL
- Sponsor Type
- NIH
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 20, 2017
First Posted
October 23, 2017
Study Start
October 31, 2018
Primary Completion
February 22, 2021
Study Completion (Estimated)
August 12, 2026
Last Updated
May 4, 2026
Results First Posted
June 13, 2023
Record last verified: 2026-02