BRCA1 Mutation CarrierPhase 2NCT02314156
Transdermal or Oral Telapristone Acetate in Treating Patients Undergoing Mastectomy
A Phase 2 study testing Telapristone Acetate, Laboratory Biomarker Analysis and Questionnaire Administration in people with BRCA1 Mutation Carrier and BRCA2 Mutation Carrier, run by Northwestern University at 3 sites in United States.
Trial summary
This randomized trial studies transdermal or oral telapristone acetate in treating patients undergoing surgery to remove the breast (mastectomy). Telapristone acetate may help prevent breast cancer from forming in premenopausal women. Read more about this study
- Who is it for?
- Ages 18 years and older, women only
- Conditions the study is for
- BRCA1 Mutation Carrier, BRCA2 Mutation Carrier, Ductal Breast Carcinoma In Situ
- What is being tested?
- Telapristone Acetate, Laboratory Biomarker Analysis and Questionnaire Administration
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 67 participants
- Where
- 3 sitesUnited States
- Study dates
- Oct 2015to May 2021
- 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
- Women only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Women scheduled for unilateral or bilateral mastectomy for breast cancer therapy, pathology confirmed stage 0-II (including ductal carcinoma in situ), or prophylaxis (breast cancer, early onset [BRCA] mutation carriers, women with strong family history or lobular carcinoma in situ or other conditions where prophylactic mastectomy has been elected)
- Eastern Cooperative Oncology Group (ECOG) performance status =< 1 (Karnofsky >= 70%)
- Total bilirubin < 1.5 x upper limit of normal (ULN)
- 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
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- Alkaline phosphatase < 2.5 x ULN
- Blood urea nitrogen < 2 x ULN
- Willing to use non-hormonal contraception (adequate barrier-type contraception or intrauterine device [IUD]) from the time the pregnancy test is performed for the duration of study participation, and 30 days after study drug cessation (for women of childbearing potential only)
- Ability to understand and the willingness to sign a written informed consent document
- Willing and able to schedule mastectomy 4 weeks (+/- 7days) following start of study agent
- Willing to avoid exposing breast skin to natural or artificial sunlight (i.e. tanning beds) for the duration of study agent dosing
- Negative urine pregnancy test result, for participants of child bearing potential, within 5 days prior to first dose of study medication; 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; OR has had a menstrual period at any time in the preceding 12 consecutive months)
- Willing to use alcohol in moderation while taking study agent
Who cannot join
- The presence of skin invasion 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) on breast.
- A history of thromboembolic disorder or cerebral vascular disease
- Use of oral contraceptives or other hormonal treatments within eight weeks prior to the randomization or during the period of the study; women should not have used Depo-Provera in the preceding 6 months; use of hormone coated IUD like Mirena is allowed
- Participants may not have received any other investigational agents in the previous 3 months
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- History of allergic reactions attributed to compounds of similar chemical or biologic composition to telapristone (i.e. other progesterone antagonists)
- 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; previous unilateral radiation in women scheduled for mastectomy of the contralateral side is allowed
- Pregnant or breastfeeding
- Currently taking spironolactone
- Recent history (within 6 months) of alcoholism or drug abuse
- Known active infection with human immunodeficiency virus (HIV), hepatitis A, B, or C
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Northwestern University
Chicago, Illinois, 60611
Memorial Sloan-Kettering Cancer Center
New York, New York, 10065
Cedars-Sinai Medical Center
West Hollywood, California, 90048
About this study
Official title: Intra-mammary Distribution of Transdermal Telapristone Versus Oral Telapristone: A Randomized Window Trial in Women Undergoing Mastectomy
This randomized trial studies transdermal or oral telapristone acetate in treating patients undergoing surgery to remove the breast (mastectomy). Telapristone acetate may help prevent breast cancer from forming in premenopausal women. Giving telapristone acetate transdermally may be safer and have fewer side effects than oral administration.
Read the detailed description
PRIMARY OBJECTIVES:
- To demonstrate that mean levels of telapristone (telapristone acetate) in breast tissue following gel application will result in levels that are not more than 50% lower than those following oral administration.
SECONDARY OBJECTIVES:
- To assess whether plasma concentrations of telapristone are significantly lower with transdermal than oral therapy.
II. To compare within-breast variation of breast tissue concentration in transdermal and oral groups.
III. To measure changes in cell proliferation (marker of proliferation (Ki-67 labeling index).
IV. Explore changes in gene expression in breast tissue related to telapristone therapy.
- Assess change in serum progesterone associated with telapristone therapy. VI. Assess the safety and tolerability of oral and transdermal administration. VII. Assess symptom measurements using BESS Questionnaire
OUTLINE: Participants are randomized to 1 of 2 treatment arms.
ARM I (TRANSDERMAL TELAPRISTONE ACETATE): Patients receive telapristone acetate transdermally and placebo orally (PO) once daily (QD) for 4 weeks.
ARM II (ORAL TELAPRISTONE ACETATE): Patients receive placebo transdermally and telapristone acetate PO QD for 4 weeks.
After completion of study treatment, patients are followed up at day 60.
- Conditions
- BRCA1 Mutation Carrier, BRCA2 Mutation Carrier, Ductal Breast Carcinoma In Situ, Lobular Breast Carcinoma In Situ, Stage 0 Breast Cancer, Stage IA Breast Cancer
- Being tested
- Telapristone Acetate, Placebo, Laboratory Biomarker Analysis, Questionnaire Administration
- Medical subject headings
- Carcinoma, Intraductal, Noninfiltrating, Breast Carcinoma In Situ, Breast Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Telapristone AcetateDrug
Also called CDB-4124, Proellex, Progenta
Given transdermally
PlaceboOther
Also called PLCB
Given PO
Telapristone AcetateDrug
Also called CDB-4124, Proellex, Progenta
Given PO
PlaceboOther
Also called PLCB
Given transdermally
Laboratory Biomarker AnalysisOther
Correlative studies
Questionnaire AdministrationOther
Ancillary studies
| Study group | Role | What they receive |
|---|---|---|
| Arm I (transdermal telapristone acetate) | Experimental | Patients receive telapristone acetate transdermally and placebo PO QD for 4 weeks. Receives: Drug: Telapristone Acetate; Other: Placebo; Other: Laboratory Biomarker Analysis; Other: Questionnaire Administration |
| Arm II (oral telapristone acetate) | Active comparator | Patients receive placebo transdermally and telapristone acetate PO QD for 4 weeks. Receives: Drug: Telapristone Acetate; Other: Placebo; Other: Laboratory Biomarker Analysis; Other: Questionnaire Administration |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean Levels of Telapristone Acetate in Breast Tissue
Post-therapy mean levels of telapristone acetate in breast tissue.
Measured: At the time of mastectomy, up to 5 weeks from baseline
Secondary outcomes (7)
Plasma Concentrations of Telapristone Acetate
Post-therapy plasma concentrations of telapristone acetate.
Measured: At the time of mastectomy, up to 5 weeks from baseline
Within-breast Variation of Breast Tissue Concentration of Telapristone Acetate
Post-therapy concentrations of telapristone acetate in 5 locations within breast tissue.
Measured: At the time of mastectomy, up to 5 weeks from baseline
Changes in Cell Proliferation
Changes in cell proliferation (Ki67 labeling index) measured in percentage of positive cells from baseline to mastectomy by tumor status in ER positive tumors.
Measured: Baseline to mastectomy (up to 5 weeks)
Changes in Serum Sex Hormone Concentrations: Estradiol
Change in estradiol in premenopausal women from baseline to post-intervention compared between treatment groups
Measured: Baseline to mastectomy, up to 5 weeks post-intervention
Change in Symptoms as Captured in the Breast Cancer Prevention Trial (BCPT) Eight Symptom Scale (BESS) Questionnaire
Mean change in symptoms as captured using the Breast Cancer Prevention Trial (BCPT) Eight Symptom Scale (BESS) questionnaire. A patient reported outcome, scores range from 0 (Not at All) to 4 (Extremely) when asked about experiencing symptoms. A positive change in scores indicates an increase in symptoms experienced and a negative change in scores indicates a decrease in symptoms experienced
Measured: Baseline to mastectomy (up to 5 weeks)
Changes in Serum Sex Hormone Concentrations: Progesterone
Change in progesterone in premenopausal women from baseline to post-intervention compared between treatment groups
Measured: Baseline to mastectomy (up to 5 weeks)
Changes in Serum Sex Hormone Concentrations: FSH
Change in FSH in premenopausal women from baseline to post-intervention compared between treatment groups
Measured: Baseline to mastectomy (up to 5 weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Dec 2014 (estimated)
- Study startOct 2015
- Primary completionJan 2018
- Study completionMay 2021
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.
- Other trials for BRCA1 Mutation CarrierStudies of the same condition, open and under way.
- More trials run by Northwestern 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
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 67 participants (actual)
Sponsor and investigators
- Lead sponsor
- Northwestern University
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Seema Khan, Principal investigator, Northwestern University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02314156
- Sponsor's protocol ID
- NCI 2013-01-03
- Other IDs
- NCI-2014-02412, N01-CN-2012-00035, NCI 2013-01-03, NWU2013-01-03, HHSN26100003
- Status last verified
- Jan 2023
- First submitted
- 9 Dec 2014
- Last updated
- 22 Feb 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 22 Feb 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Intra-mammary Distribution of Transdermal Telapristone Versus Oral Telapristone: A Randomized Window Trial in Women Undergoing Mastectomy
- ClinicalTrials.gov ID
- NCT02314156
- Acronym / sponsor ID
- NCI 2013-01-03
- Conditions
- BRCA1 Mutation Carrier, BRCA2 Mutation Carrier, Ductal Breast Carcinoma In Situ, Lobular Breast Carcinoma In Situ, Stage 0 Breast Cancer, Stage IA Breast Cancer
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 67 participants (actual)
- Lead sponsor
- Northwestern UniversityUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- Oct 2015 (actual)
- Primary completion
- Jan 2018 (actual)
- Study completion
- May 2021 (actual)
- First posted
- 11 Dec 2014 (estimated)
- Results
- Posted 22 Feb 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This randomized trial studies transdermal or oral telapristone acetate in treating patients undergoing surgery to remove the breast (mastectomy). Telapristone acetate may help prevent breast cancer from forming in premenopausal women. Giving telapristone acetate transdermally may be safer and have fewer side effects than oral administration.
Detailed description
PRIMARY OBJECTIVES:
- To demonstrate that mean levels of telapristone (telapristone acetate) in breast tissue following gel application will result in levels that are not more than 50% lower than those following oral administration.
SECONDARY OBJECTIVES:
- To assess whether plasma concentrations of telapristone are significantly lower with transdermal than oral therapy.
II. To compare within-breast variation of breast tissue concentration in transdermal and oral groups.
III. To measure changes in cell proliferation (marker of proliferation (Ki-67 labeling index).
IV. Explore changes in gene expression in breast tissue related to telapristone therapy.
- Assess change in serum progesterone associated with telapristone therapy. VI. Assess the safety and tolerability of oral and transdermal administration. VII. Assess symptom measurements using BESS Questionnaire
OUTLINE: Participants are randomized to 1 of 2 treatment arms.
ARM I (TRANSDERMAL TELAPRISTONE ACETATE): Patients receive telapristone acetate transdermally and placebo orally (PO) once daily (QD) for 4 weeks.
ARM II (ORAL TELAPRISTONE ACETATE): Patients receive placebo transdermally and telapristone acetate PO QD for 4 weeks.
After completion of study treatment, patients are followed up at day 60.
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm I (transdermal telapristone acetate) | Experimental | Drug: Telapristone Acetate, Other: Placebo, Other: Laboratory Biomarker Analysis, Other: Questionnaire AdministrationPatients receive telapristone acetate transdermally and placebo PO QD for 4 weeks. |
| Arm II (oral telapristone acetate) | Active comparator | Drug: Telapristone Acetate, Other: Placebo, Other: Laboratory Biomarker Analysis, Other: Questionnaire AdministrationPatients receive placebo transdermally and telapristone acetate PO QD for 4 weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- Women scheduled for unilateral or bilateral mastectomy for breast cancer therapy, pathology confirmed stage 0-II (including ductal carcinoma in situ), or prophylaxis (breast cancer, early onset [BRCA] mutation carriers, women with strong family history or lobular carcinoma in situ or other conditions where prophylactic mastectomy has been elected)
- Eastern Cooperative Oncology Group (ECOG) performance status =< 1 (Karnofsky >= 70%)
- Total bilirubin < 1.5 x upper limit of normal (ULN)
- Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase [SGOT]) < 2.5 x ULN
- Alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT]) < 2.5 x ULN
Key exclusion criteria (14 listed)
- The presence of skin invasion 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) on breast.
- A history of thromboembolic disorder or cerebral vascular disease
- Use of oral contraceptives or other hormonal treatments within eight weeks prior to the randomization or during the period of the study; women should not have used Depo-Provera in the preceding 6 months; use of hormone coated IUD like Mirena is allowed
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 |
|---|---|---|
| Telapristone AcetateAlso: CDB-4124, Proellex, ProgentaGiven transdermally | Drug | Arm I (transdermal telapristone acetate) |
| PlaceboAlso: PLCBGiven PO | Other | Arm I (transdermal telapristone acetate) |
| Telapristone AcetateAlso: CDB-4124, Proellex, ProgentaGiven PO | Drug | Arm II (oral telapristone acetate) |
| PlaceboAlso: PLCBGiven transdermally | Other | Arm II (oral telapristone acetate) |
| Laboratory Biomarker AnalysisCorrelative studies | Other | Arm I (transdermal telapristone acetate), Arm II (oral telapristone acetate) |
| Questionnaire AdministrationAncillary studies | Other | Arm I (transdermal telapristone acetate), Arm II (oral telapristone acetate) |
Primary outcome
Mean Levels of Telapristone Acetate in Breast Tissue
Time frame: At the time of mastectomy, up to 5 weeks from baseline
Post-therapy mean levels of telapristone acetate in breast tissue.
7 secondary outcomes
Plasma Concentrations of Telapristone Acetate
Time frame: At the time of mastectomy, up to 5 weeks from baseline
Within-breast Variation of Breast Tissue Concentration of Telapristone Acetate
Time frame: At the time of mastectomy, up to 5 weeks from baseline
Changes in Cell Proliferation
Time frame: Baseline to mastectomy (up to 5 weeks)
Changes in Serum Sex Hormone Concentrations: Estradiol
Time frame: Baseline to mastectomy, up to 5 weeks post-intervention
Change in Symptoms as Captured in the Breast Cancer Prevention Trial (BCPT) Eight Symptom Scale (BESS) Questionnaire
Time frame: Baseline to mastectomy (up to 5 weeks)
Changes in Serum Sex Hormone Concentrations: Progesterone
Time frame: Baseline to mastectomy (up to 5 weeks)
Changes in Serum Sex Hormone Concentrations: FSH
Time frame: Baseline to mastectomy (up to 5 weeks)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Feb 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Seema Khan principal investigator
Northwestern University
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Telapristone Acetate, Laboratory Biomarker Analysis and Questionnaire Administration in people with BRCA1 Mutation Carrier and BRCA2 Mutation Carrier, run by Northwestern University at 3 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 14 inclusion and 14 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 22 Feb 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 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. 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 22 Feb 2023). 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?
Mean Levels of Telapristone Acetate in Breast Tissue (time frame: At the time of mastectomy, up to 5 weeks from baseline). 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 22 Feb 2023.