Breast CancerPhase 2NCT02040857
Palbociclib in Combination With Adjuvant Endocrine Therapy for Hormone Receptor Positive, HER2 Negative Invasive Breast Cancer
A Phase 2 study testing Palbociclib and Aromatase Inhibitor in people with Breast Cancer, run by Dana-Farber Cancer Institute at 10 sites in United States.
Trial summary
This research study is evaluating a drug called Palbociclib in combination with endocrine therapy as a possible treatment for hormone receptor positive breast cancer. Palbociclib is a drug that may stop cancer cells from growing. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Breast Cancer
- What is being tested?
- Palbociclib and Aromatase Inhibitor
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 162 participants
- Where
- 10 sitesUnited States
- Study dates
- Jan 2014to 27 Dec 2024
- 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
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participants must have histologically confirmed hormone receptor positive (HR+) HER2 negative stage II (except T2N0) or stage III invasive breast cancer. Evaluation for metastatic disease is not required in the absence of symptoms.
- Men and both pre- and postmenopausal women are eligible.
- Prior Treatment:
- Participants may have received (neo)adjuvant chemotherapy, but must be at least 30 days after last dose, with no more than grade 1 residual toxicity at time of screening.
- Participants may have received adjuvant radiotherapy, but must be at least 30 days after last dose , with no more than grade 1 residual toxicity at the time of screening.
- If most recent therapy was surgery, participants must be at least 30 days out from definitive surgery with no active wound healing complications.
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- Participants must have demonstrated ability to tolerate endocrine therapy by prior successful completion of at least 1 month of tamoxifen or aromatase inhibitor (AI) therapy without significant adverse events, and in the opinion of the treating physician any ongoing toxicity does not preclude ability to continue on tamoxifen or AI for at least a projected 2 year continuous duration. Ongoing use of any endocrine therapy, including tamoxifen, letrozole, anastrozole, or exemestane, is allowed. Patients may enroll within 2 years of beginning endocrine therapy, as long as there is a plan for at least 2 more years of adjuvant endocrine therapy.
- ECOG performance status 0-1
- Age ≥18 years.
- Normal organ and marrow function
- Baseline QTc ≤ 480 ms
- The effects of palbociclib on the developing human fetus are unknown. Women who might become pregnant must use adequate contraception
- Ability to understand and the willingness to sign a written informed consent document
Who cannot join
- Concurrent therapy with other investigational agents.
- Prior therapy with any CDK4/6 inhibitor.
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to palbociclib.
- Participants receiving any medications or substances that are strong inhibitors or inducers of CYP3A isoenzymes are ineligible.
- Current use of drugs that are known to prolong the QT interval
- Subjects with organ allograft requiring immunosuppression.
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- 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.
- Pregnant women are excluded from this study. Breastfeeding should be discontinued prior to entry onto the study.
- Individuals with a history of a different malignancy are ineligible except for the following circumstances. Individuals with a history of other malignancies are eligible if they have been disease-free for at least 5 years and are deemed by the investigator to be at low risk for recurrence of that malignancy. Individuals with the following cancers are eligible if diagnosed and treated within the past 5 years: ductal carcinoma in situ of the breast, cervical cancer in situ, and basal cell or squamous cell carcinoma of the skin.
- No ongoing combination antiretroviral therapy
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
United States10 sites
Massachusetts General Hospital
Boston, Massachusetts, 02214
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
MGH/North Shore Cancer Center
Danvers, Massachusetts, 01923
Indiana University Health Hospital
Indianapolis, Indiana, 46202
Dana-Farber/New Hampshire Oncology-Hematology
Londonderry, New Hampshire, 03053
DF/DWCC at Milford Regional Cancer Center
Milford, Massachusetts, 01757
University of Pennsylvania-Abramson Cancer Center
Philadelphia, Pennsylvania, 19104
University of California, San Francisco
San Francisco, California, 94115
South Shore Hospital
Weymouth, Massachusetts, 02190
About this study
Official title: A Phase 2 Pilot Feasibility Study of Palbociclib in Combination With Adjuvant Endocrine Therapy for Hormone Receptor Positive Invasive Breast Carcinoma
This research study is evaluating a drug called Palbociclib in combination with endocrine therapy as a possible treatment for hormone receptor positive breast cancer.
- Palbociclib is a drug that may stop cancer cells from growing. Palbociclib blocks activity of two closely related enzymes (proteins that help chemical reactions in the body occur), called Cyclin D Kinases 4 and 6 (CDK 4/6). These proteins are part of a pathway, or a sequence of steps which is known to regulate cell growth. Laboratory testing has suggested palbociclib may stop the growth of hormone receptor positive breast cancer.
- Endocrine therapy prevents breast cancer cell growth by blocking estrogen stimulation. During this study endocrine therapy will either be tamoxifen or an aromatase inhibitor. It is standard of care for premenopausal women to take tamoxifen and for postmenopausal women to take either an aromatase inhibitor or tamoxifen after a diagnosis of hormone receptor positive breast cancer.
Read the detailed description
After the screening procedures confirms that the participant is able to participate in the study. The participant will be given a dosing diary for each treatment cycle. Each treatment cycle lasts 28 days, during which time the participant will take Palbociclib once a day on days 1-21 of each 28 day cycle and the aromatase inhibitor that the participant is already taking once a day every day. The diary will also include special instructions for taking the study drug(s).
All participants participating in the research study will receive the same dose of Palbociclib.
While participating in the research study the participant will have the following tests and procedures:
- Clinical Exams: The participant will have a physical exam on the first day of the month for first three months of therapy. After that, the participant will have a physical exam every other month until the end of the first year of therapy. After the first year, the participant will have a physical exam every 3 months during the second year of therapy. During the physical exam, the participant will be asked questions about their general health, questions about any problems that they may be experiencing, and any medications they are taking.
- Blood tests: The participant will have blood drawn on the first and fifteenth days of the first three months of therapy. After that, the participant will have blood drawn every month for the remainder of the first year of treatment and the every other month until the end of therapy. These tests will be used to determine if the participant is having any side effects related to the study drug.
- Electrocardiograms (EKG): The participant will have an EKG performed on the first day of the first three months of therapy. After that, the participant will have an EKG every other month until the end of the first year of therapy. After the first year of therapy, the participant will have an EKG every 3 months during the second year of therapy.
- Conditions
- Breast Cancer
- Being tested
- Palbociclib, Aromatase Inhibitor
- Keywords
- Breast Cancer
- Medical subject headings
- Breast Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PalbociclibDrug
Also called PD 0332991-0054, PD-0332991, PD-332991, PF-00080665-73
CDK 4/6 inhibitor
Aromatase InhibitorDrug
Also called Femara, Letrozole, Anastrozole, Arimidex
Endocrine Therapy
| Study group | Role | What they receive |
|---|---|---|
| Palbociclib With Adjuvant Endocrine Therapy | Experimental | * Palbociclib 125 mg PO qd 21 days on, 7 days off
* Endocrine Therapy: Tamoxifen 20mg, Letrozole 2.5mg, Anastrozole 1mg, or Exemestane 25mg PO qd Receives: Drug: Palbociclib; Drug: Aromatase Inhibitor |
What is being measured
The primary outcome is the main question the study is designed to answer.
2-Year Treatment Discontinuation Rate
The 2-year treatment discontinuation rate is the percentage of participants who do not complete the palbociclib treatment per protocol for reasons due to toxicity, withdrawal of consent to be treated, or other events related to tolerability in uncensored participants. Participants who discontinued palbociclib early for reasons that were not treatment-related were censored.
Measured: Evaluate upon completion of palbociclib, up to 2 years of treatment completion.
Secondary outcomes (4)
2-year Treatment Discontinuation Rate by Aromatase Inhibitor and Tamoxifen-based Therapy Subgroup
The 2-year treatment discontinuation rate is the percentage of participants who do not complete the palbociclib treatment per protocol for reason due to toxicity, withdrawal of consent to be treated, or other events related to tolerability of all enrolled participants.
Measured: Evaluate upon completion of palbociclib, up to 2 years of treatment completion.
Grade 3-4 Treatment-Related Neutropenia Toxicity Rate
Grade 3-4 treatment-related neutropenia toxicity rate is the percentage of participants experiencing at least one grade 3-4 neutropenia AE with treatment attribution of possible, probable or definite based on NCI Common Toxicity Criteria for Adverse Events version 4 (CTCAEv4) during the time of observation on treatment as reported on case report forms.
Measured: AE data collected every cycle from the time of the first dose of study treatment, through the study and until 30 days after removal from study or death, whichever occurs first. Therefore, AEs were observed up to 2 years plus 30 days.
All Grade Treatment-Related Fatigue Toxicity Rate
All grade treatment-related fatigue toxicity rate is the percentage of participants experiencing at least one grade 1-4 fatigue AE with treatment attribution of possible, probable or definite based on NCI Common Toxicity Criteria for Adverse Events version 4 (CTCAEv4) during the time of observation on treatment as reported on case report forms.
Measured: AE data collected every cycle from the time of the first dose of study treatment, through the study and until 30 days after removal from study or death, whichever occurs first. Therefore, AEs were observed up to 2 years plus 30 days.
All GradeTreatment-Related Alopecia Toxicity Rate
All grade treatment-related alopecia toxicity rate is the percentage of participants experiencing at least one grade 1-4 alopecia AE with treatment attribution of possible, probable or definite based on NCI Common Toxicity Criteria for Adverse Events version 4 (CTCAEv4) during the time of observation on treatment as reported on case report forms.
Measured: AE data collected every cycle from the time of the first dose of study treatment, through the study and until 30 days after removal from study or death, whichever occurs first. Therefore, AEs were observed up to 2 years plus 30 days.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jan 2014 (estimated)
- Study startJan 2014
- Primary completionMay 2018
- Study completion27 Dec 2024
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.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Breast CancerStudies of the same condition, open and under way.
- More trials run by Dana-Farber Cancer InstituteThe 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
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 162 participants (actual)
Sponsor and investigators
- Lead sponsor
- Dana-Farber Cancer Institute
- Sponsor type
- University, hospital or other
- Collaborators
- Pfizer
- Responsible party
- Principal investigator, Erica Mayer, MD, MPH, Dana-Farber Cancer Institute
- Investigators
- Erica Mayer, MD, MPH, Principal investigator, Dana-Farber Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02040857
- Sponsor's protocol ID
- 13-559
- Status last verified
- Apr 2025
- First submitted
- 13 Jan 2014
- Last updated
- 29 Apr 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
Mayer EL, DeMichele A, Rugo HS, Miller K, Waks AG, Come SE, Mulvey T, Jeselsohn R, Overmoyer B, Guo H, Barry WT, Huang Bartlett C, Koehler M, Winer EP, Burstein HJ. A phase II feasibility study of palbociclib in combination with adjuvant endocrine therapy for hormone receptor-positive invasive breast carcinoma. Ann Oncol. 2019 Sep 1;30(9):1514-1520. doi: 10.1093/annonc/mdz198.
PubMed 31250880
Study at a glance
From the registry record, last updated by the sponsor on 29 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Pilot Feasibility Study of Palbociclib in Combination With Adjuvant Endocrine Therapy for Hormone Receptor Positive Invasive Breast Carcinoma
- ClinicalTrials.gov ID
- NCT02040857
- Acronym / sponsor ID
- 13-559
- Conditions
- Breast Cancer
- Keywords
- Breast Cancer
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 162 participants (actual)
- Lead sponsor
- Dana-Farber Cancer InstituteUniversity, hospital or other
- Collaborators
- Pfizer
- Study start
- Jan 2014
- Primary completion
- May 2018 (actual)
- Study completion
- 27 Dec 2024 (actual)
- First posted
- 20 Jan 2014 (estimated)
- Results
- Posted 23 Dec 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This research study is evaluating a drug called Palbociclib in combination with endocrine therapy as a possible treatment for hormone receptor positive breast cancer.
- Palbociclib is a drug that may stop cancer cells from growing. Palbociclib blocks activity of two closely related enzymes (proteins that help chemical reactions in the body occur), called Cyclin D Kinases 4 and 6 (CDK 4/6). These proteins are part of a pathway, or a sequence of steps which is known to regulate cell growth. Laboratory testing has suggested palbociclib may stop the growth of hormone receptor positive breast cancer.
- Endocrine therapy prevents breast cancer cell growth by blocking estrogen stimulation. During this study endocrine therapy will either be tamoxifen or an aromatase inhibitor. It is standard of care for premenopausal women to take tamoxifen and for postmenopausal women to take either an aromatase inhibitor or tamoxifen after a diagnosis of hormone receptor positive breast cancer.
Detailed description
After the screening procedures confirms that the participant is able to participate in the study. The participant will be given a dosing diary for each treatment cycle. Each treatment cycle lasts 28 days, during which time the participant will take Palbociclib once a day on days 1-21 of each 28 day cycle and the aromatase inhibitor that the participant is already taking once a day every day. The diary will also include special instructions for taking the study drug(s).
All participants participating in the research study will receive the same dose of Palbociclib.
While participating in the research study the participant will have the following tests and procedures:
- Clinical Exams: The participant will have a physical exam on the first day of the month for first three months of therapy. After that, the participant will have a physical exam every other month until the end of the first year of therapy. After the first year, the participant will have a physical exam every 3 months during the second year of therapy. During the physical exam, the participant will be asked questions about their general health, questions about any problems that they may be experiencing, and any medications they are taking.
- Blood tests: The participant will have blood drawn on the first and fifteenth days of the first three months of therapy. After that, the participant will have blood drawn every month for the remainder of the first year of treatment and the every other month until the end of therapy. These tests will be used to determine if the participant is having any side effects related to the study drug.
- Electrocardiograms (EKG): The participant will have an EKG performed on the first day of the first three months of therapy. After that, the participant will have an EKG every other month until the end of the first year of therapy. After the first year of therapy, the participant will have an EKG every 3 months during the second year of therapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Palbociclib With Adjuvant Endocrine Therapy | Experimental | Drug: Palbociclib, Drug: Aromatase Inhibitor* Palbociclib 125 mg PO qd 21 days on, 7 days off * Endocrine Therapy: Tamoxifen 20mg, Letrozole 2.5mg, Anastrozole 1mg, or Exemestane 25mg PO qd |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- Participants must have histologically confirmed hormone receptor positive (HR+) HER2 negative stage II (except T2N0) or stage III invasive breast cancer. Evaluation for metastatic disease is not required in the absence of symptoms.
- Men and both pre- and postmenopausal women are eligible.
- Prior Treatment:
- Participants must have demonstrated ability to tolerate endocrine therapy by prior successful completion of at least 1 month of tamoxifen or aromatase inhibitor (AI) therapy without significant adverse events, and in the opinion of the treating physician any ongoing toxicity does not preclude ability to continue on tamoxifen or AI for at least a projected 2 year continuous duration. Ongoing use of any endocrine therapy, including tamoxifen, letrozole, anastrozole, or exemestane, is allowed. Patients may enroll within 2 years of beginning endocrine therapy, as long as there is a plan for at least 2 more years of adjuvant endocrine therapy.
- ECOG performance status 0-1
Key exclusion criteria (10 listed)
- Concurrent therapy with other investigational agents.
- Prior therapy with any CDK4/6 inhibitor.
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to palbociclib.
- Participants receiving any medications or substances that are strong inhibitors or inducers of CYP3A isoenzymes are ineligible.
- Current use of drugs that are known to prolong the QT interval
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 |
|---|---|---|
| PalbociclibAlso: PD 0332991-0054, PD-0332991, PD-332991, PF-00080665-73CDK 4/6 inhibitor | Drug | Palbociclib With Adjuvant Endocrine Therapy |
| Aromatase InhibitorAlso: Femara, Letrozole, Anastrozole, ArimidexEndocrine Therapy | Drug | Palbociclib With Adjuvant Endocrine Therapy |
Primary outcome
2-Year Treatment Discontinuation Rate
Time frame: Evaluate upon completion of palbociclib, up to 2 years of treatment completion.
The 2-year treatment discontinuation rate is the percentage of participants who do not complete the palbociclib treatment per protocol for reasons due to toxicity, withdrawal of consent to be treated, or other events related to tolerability in uncensored participants. Participants who discontinued palbociclib early for reasons that were not treatment-related were censored.
4 secondary outcomes
2-year Treatment Discontinuation Rate by Aromatase Inhibitor and Tamoxifen-based Therapy Subgroup
Time frame: Evaluate upon completion of palbociclib, up to 2 years of treatment completion.
Grade 3-4 Treatment-Related Neutropenia Toxicity Rate
Time frame: AE data collected every cycle from the time of the first dose of study treatment, through the study and until 30 days after removal from study or death, whichever occurs first. Therefore, AEs were observed up to 2 years plus 30 days.
All Grade Treatment-Related Fatigue Toxicity Rate
Time frame: AE data collected every cycle from the time of the first dose of study treatment, through the study and until 30 days after removal from study or death, whichever occurs first. Therefore, AEs were observed up to 2 years plus 30 days.
All GradeTreatment-Related Alopecia Toxicity Rate
Time frame: AE data collected every cycle from the time of the first dose of study treatment, through the study and until 30 days after removal from study or death, whichever occurs first. Therefore, AEs were observed up to 2 years plus 30 days.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Apr 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 31250880Mayer EL, DeMichele A, Rugo HS, Miller K, Waks AG, Come SE, Mulvey T, Jeselsohn R, Overmoyer B, Guo H, Barry WT, Huang Bartlett C, Koehler M, Winer EP, Burstein HJ. A phase II feasibility study of palbociclib in combination with adjuvant endocrine therapy for hormone receptor-positive invasive breast carcinoma. Ann Oncol. 2019 Sep 1;30(9):1514-1520. doi: 10.1093/annonc/mdz198.
- Individual participant dataSharing planned: No
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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.
Erica Mayer, MD, MPH principal investigator
Dana-Farber Cancer Institute
10 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 Palbociclib and Aromatase Inhibitor in people with Breast Cancer, run by Dana-Farber Cancer Institute at 10 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 13 inclusion and 10 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 29 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 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 23 Dec 2020). 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?
2-Year Treatment Discontinuation Rate (time frame: Evaluate upon completion of palbociclib, up to 2 years of treatment completion.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02040857, which also lists 1 linked publication.
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 29 Apr 2025.