Clinical trial NCT03921866: UK Ibrance Patient Program (IPP) Study
Official title: Observational Cohort Study of Patients With Hormone Receptor-positive Metastatic Breast Cancer Treated With Palbociclib (Ibrance(Registered)) as Part of the United Kingdom Ibrance (Registered) Patient Program (IPP); the Real Outcomes Ibrance (Registered) Study (ROIS)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: HR+/HER2- Locally Advanced, Metastatic Breast Cancer
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2019-04-19
Enrollment: 191
Sex: FEMALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Women over the age of 18 who entered into the UK IPP

Brief summary: What are the real-world treatment patterns, patients' characteristics, clinical outcomes and healthcare resource utilisation associated with palbociclib treatment in the 3 years following initiation in United Kingdom patients with hormone receptor-positive, human epidermal growth factor 2-negative metastatic breast cancer treated as part of the IPP?

Detailed description: Hormone receptor positive (HR+) breast cancer (BC) represents the largest therapeutic subtype of the disease, accounting for 60 to 65% of all malignant neoplasms of the breast. Palbociclib (Ibrance®) is a small-molecule inhibitor of cyclin-dependent kinases 4 and 6 (CDK4/6) which in clinical trial settings has been shown to increase progression-free survival (PFS) for patients with HR+, human epidermal growth factor 2-negative (HER2-) metastatic breast cancer (MBC). Palbociclib first received a European Union (EU) marketing authorisation in September 2016, to be commercialised as Ibrance® by Pfizer. Palbociclib was recommended for use with an aromatase inhibitor in patients with HR+/HER2- locally advanced and MBC in the National Health Service (NHS) in England by the National Institute for Health and Care Excellence (NICE) in November 2017 and by the Scottish Medicines Consortium (SMC) in December 2017. In order to provide access to palbociclib in the United Kingdom (UK) during the NICE/SMC appraisal period, the Ibrance® Patient Program (IPP) was initiated and run by Pfizer between April 2017 until a positive NICE/SMC appraisal in November 2017 (for England and Wales) or December 2017 (for Scotland).

Pfizer are interested in the opportunity to collect data from patients who received palbociclib as part of the UK IPP, to better understand patients' characteristics in a routine care setting, treatment persistence and dose management, clinical outcomes, and healthcare resource utilisation. This study will provide real-world evidence on patients' clinical progression and experience of treatment with palbociclib in routine clinical settings in a UK context.

Research question:

What are the real-world treatment patterns, patients' characteristics, clinical outcomes and healthcare resource utilisation associated with palbociclib treatment in the 3 years following initiation in United Kingdom patients with HR+/HER2- MBC treated as part of the IPP?

Eligibility criteria: Inclusion criteria:

All patients meeting the following eligibility criteria will be included in the study:

* Patients enrolled into the IPP at one of the selected hospitals (see Annex 1 for IPP enrolment letter).
* Patients who received ≥1 dose of palbociclib as part of the IPP at one of the selected sites.
* For sites where data collection is performed by pH Associates, written informed consent will be required from living patients to access their medical records.
* Patient aged ≥18 years old at enrollment into the IPP

Primary outcomes:
measure: Percentage of Participants According to Treatment Lines
timeFrame: At baseline
measure: Time From Letrozole to Palbociclib Initiation
timeFrame: At baseline
measure: Number of Participants With Menopausal Status
timeFrame: At baseline
measure: Percentage of Participants According to Disease Free Interval at Palbociclib Initiation
timeFrame: At baseline
measure: Percentage of Participants With Primary or Recurrent Metastatic Breast Cancer Diagnosis
timeFrame: At baseline
measure: Percentage of Participants With Lymph Nodes Involvement
timeFrame: At baseline
measure: Number of Lymph Nodes Involved
timeFrame: At baseline
measure: Number of Participants With Estrogen, Progesterone and Human Epidermal Growth Factor 2 (HER2) Receptor Status
timeFrame: At baseline
measure: Percentage of Participants Who Had Rebiopsy After Metastatic Disease Diagnosis
timeFrame: At baseline
measure: Percentage of Participants According to Tumor Stage
timeFrame: At baseline
measure: Percentage of Participants According to Nodal Status
timeFrame: At baseline
measure: Percentage of Participants According to Metastasis
timeFrame: At baseline
measure: Tumor Size at Palbociclib Initiation
timeFrame: At baseline
measure: Percentage of Participants According to Tumor Grade
timeFrame: At baseline
measure: Percentage of Participants With Ki-67 Protein Proliferation Index Recorded
timeFrame: At baseline
measure: Ki-67 Protein Proliferation Index
timeFrame: At baseline
measure: Percentage of Participants With Eastern Cooperative Oncology Group Performance Score (ECOG PS)
timeFrame: At baseline
measure: Percentage of Participants With Recurrence Type
timeFrame: At baseline
measure: Percentage of Participants According to Number of Metastatic Sites
timeFrame: At baseline
measure: Percentage of Participants According to Location of Metastases
timeFrame: At baseline
measure: Percentage of Participants With Non-Visceral Location of Metastases
timeFrame: At baseline
measure: Number of Participants According to Metastatic Sites With Locoregional Recurrence
timeFrame: At baseline
measure: Duration of Disease at Initiation of Palbociclib
timeFrame: At baseline
measure: Percentage of Participants Who Received Chemotherapy in Adjuvant or Neoadjuvant Setting
timeFrame: At baseline
measure: Percentage of Participants Who Received Chemotherapy in Advanced, Disease Modifying or Metastatic Setting
timeFrame: At baseline
measure: Number of Lines of Prior Chemotherapy for Metastatic Disease
timeFrame: At baseline
measure: Percentage of Participants Who Received Luteinizing Hormone Releasing Hormone (LHRH) or Chemotherapy
timeFrame: At baseline
measure: Percentage of Participants Who Received Endocrine Therapy in Adjuvant or Neoadjuvant Setting
timeFrame: At baseline
measure: Number of Participants With Types of Endocrine Therapy in Adjuvant or Neoadjuvant Setting
timeFrame: At baseline
measure: Percentage of Participants Who Received Endocrine Therapy in Advanced, Disease Modifying or Metastatic Setting
timeFrame: At baseline
measure: Percentage of Participants With Type of Endocrine Therapy in Advanced, Disease Modifying or Metastatic Setting
timeFrame: At baseline
measure: Number of Lines of Prior Endocrine Therapy for Metastatic Disease
timeFrame: At baseline
measure: Number of Participants Who Received Radiotherapy in Advanced, Disease Modifying or Metastatic Setting
timeFrame: At baseline
measure: Percentage of Participants Who Received Concomitant Medications
timeFrame: At baseline
measure: Number of Participants With Concomitant Medications Prescribed Along With Goserelin
timeFrame: At baseline
measure: Number of Participants According to Number of Prior Treatments in Metastatic Setting
timeFrame: At baseline
measure: Number of Participants According to Number of Prior Chemotherapy and Hormone Therapy in Metastatic Setting
timeFrame: At baseline
measure: Number of Participants According to Number of Prior Chemotherapies in Metastatic Setting
timeFrame: At baseline
measure: Number of Participants According to Number of Prior Hormone Therapies in Metastatic Setting
timeFrame: At baseline
Secondary outcomes:
measure: Percentage of Participants With Their Starting Dose of Palbociclib
timeFrame: Data collected at index date (for a maximum period of 3 years)
measure: Percentage of Participants Who Received Endocrine Therapy Along With Palbociclib
timeFrame: Data collected from index date until lost to follow up or end of follow up, whichever occurred first (for a maximum period of 3 years)
measure: Percentage of Participants With Dose Reductions and Treatment Discontinuation
timeFrame: Data collected from index date until lost to follow up or end of follow up, whichever occurred first (for a maximum period of 3 years)
measure: Number of Participants With Reasons for Palbociclib Discontinuation
timeFrame: Data collected from index date until lost to follow up or end of follow up, whichever occurred first (for a maximum period of 3 years)
measure: Percentage of Participants With Temporary Discontinuation
timeFrame: Data collected from index date until lost to follow up or end of follow up, whichever occurred first (for a maximum period of 3 years)
measure: Percentage of Participants According to Time to Dose Reduction in First Line Therapy
timeFrame: Data collected from index date until lost to follow up or end of follow up, whichever occurred first (for a maximum period of 3 years)
measure: Time to Palbociclib Discontinuation
timeFrame: Data collected from index date until lost to follow up or end of follow up, whichever occurred first (for a maximum period of 3 years)
measure: Number of Participants With First 3 Lines of Treatment After Progression
timeFrame: Data collected from date of progression until lost to follow up or end of follow up period, whichever occurred first (for a maximum period of 3 years)
measure: Doses Prescribed for First 3 Lines of Treatment After Progression
timeFrame: Data collected from date of progression until lost to follow up or end of follow up period, whichever occurred first (for a maximum period of 3 years)
measure: Duration of First 3 Lines of Treatment After Progression
timeFrame: Data collected from date of progression until lost to follow up or end of follow up period, whichever occurred first (for a maximum period of 3 years)
measure: Number of Completed Cycles of Palbociclib
timeFrame: Data collected from index date until lost to follow up or end of follow up, whichever occurred first (for a maximum period of 3 years)
measure: Percentage of Participants Who Received Letrozole and Fulvestrant With Palbociclib
timeFrame: Data collected from index date until lost to follow up or end of follow up, whichever occurred first (for a maximum period of 3 years)
measure: Percentage of Participants With Progression Free Survival Following Palbociclib Initiation
timeFrame: Data collected from index date until lost to follow up or end of follow up, whichever occurred first (for a maximum period of 3 years)
measure: Percentage of Participants Alive Following Palbociclib Initiation
timeFrame: Data collected from index date until lost to follow up or end of follow up, whichever occurred first (for a maximum period of 3 years)
measure: Percentage of Participants With Partial Response (PR) and Complete Response (CR) Following Palbociclib Initiation
timeFrame: Data collected from index date until lost to follow up or end of follow up, whichever occurred first (for a maximum period of 3 years)
measure: Percentage of Participants With Stable Disease (SD) Following Palbociclib Initiation
timeFrame: Data collected from index date until lost to follow up or end of follow up, whichever occurred first (for a maximum period of 3 years)
measure: Progression Free Survival (PFS)
timeFrame: From index date to PD or death whichever occurred first (for a maximum period of 3 years)
measure: Overall Survival (OS)
timeFrame: From index date until date of death or date of censoring (for a maximum period of 3 years)
measure: Time to Achieving Best Overall Response (BOR)
timeFrame: From index date till date of BOR or date of censoring (for a maximum period of 3 years)
measure: Duration of Follow-up Period
timeFrame: Data collected from index date until lost to follow up or end of follow up, whichever occurred first (for a maximum period of 3 years)
measure: Percentage of Participants With Best Response (BR), Progressive Disease (PD) and Stable Disease (SD) to Palbociclib
timeFrame: From index date till BR (CR or PR, whichever occurred first), SD, PD or date of censoring (for a maximum period of 3 years)
measure: Time to Best Response (BR)
timeFrame: From index date till BR or date of censoring (for a maximum period of 3 years)
measure: Time to First Response
timeFrame: From index date till first documented CR or PR or date of censoring (for a maximum period of 3 years)
measure: Percentage of Participants With Neutropenia Post-Palbociclib Initiation
timeFrame: Data collected from index date up to 6 months post-palbociclib initiation (for a maximum period of 3 years)
measure: Percentage of Participants With Neutropenia Post-Palbociclib Initiation as Recorded and Inferred From Clinical Notes
timeFrame: Data collected from index date up to 6 months post-palbociclib initiation (for a maximum period of 3 years)
measure: Percentage of Participants With Neutropenia Post-Palbociclib Initiation as Recorded and Inferred From ANC Measurements
timeFrame: Data collected from index date up to 6 months post-palbociclib initiation (for a maximum period of 3 years)
measure: Percentage of Participants According to the Severe Grade of Neutropenia
timeFrame: Data collected from index date up to 3 months post-palbociclib initiation (for a maximum period of 3 years)
measure: Percentage of Participants With Febrile Neutropenia Post-Palbociclib Initiation
timeFrame: Data collected from index date up to 3 months post-palbociclib initiation (for a maximum period of 3 years)
measure: Percentage of Participants With Gastro-Intestinal Toxicities Post-Palbociclib Initiation
timeFrame: Data collected from index date up to 1-year of follow up period (for a maximum period of 3 years)
measure: Percentage of Participants With Adverse Events During Follow-up
timeFrame: Data collected from index date up to 1-year of follow up period (for a maximum period of 3 years)
measure: Absolute Values for Hematology Parameter in First 6 Months Following Palbociclib Initiation: Hemoglobin
timeFrame: From index date up to 6 months after palbociclib initiation (for a maximum period of 3 years)
measure: Absolute Values for Hematology Parameters in First 6 Months Following Palbociclib Initiation: White Blood Cell, Absolute Neutrophil Counts and Platelet Counts
timeFrame: Data collected from index date up to 6 months after palbociclib initiation (for a maximum period of 3 years)
measure: Absolute Values for Liver Function Parameters in First 6 Months Following Palbociclib Initiation: Aspartate Aminotransferase, Alanine Aminotransferase and Alkaline Phosphatase
timeFrame: Data collected from index date up to 6 months after palbociclib initiation (for a maximum period of 3 years)
measure: Absolute Values for Liver Function Parameter in First 6 Months Following Palbociclib Initiation: Albumin
timeFrame: Data collected from index date up to 6 months after palbociclib initiation (for a maximum period of 3 years)
measure: Absolute Values for Liver Function Parameter in First 6 Months Following Palbociclib Initiation: Bilirubin
timeFrame: Data collected from index date up to 6 months after palbociclib initiation (for a maximum period of 3 years)
measure: Absolute Values for Bone Profile Parameters in First 6 Months Following Palbociclib Initiation: Calcium and Phosphate
timeFrame: Data collected from index date up to 6 months after palbociclib initiation (for a maximum period of 3 years)
measure: Absolute Values for Clinical Chemistry Parameters in First 6 Months Following Palbociclib Initiation: Potassium, Sodium and Urea
timeFrame: Data collected from index date up to 6 months after palbociclib initiation (for a maximum period of 3 years)
measure: Absolute Values for Clinical Chemistry Parameter in First 6 Months Following Palbociclib Initiation: Creatinine
timeFrame: Data collected from index date up to 6 months after palbociclib initiation (for a maximum period of 3 years)
measure: Percentage of Participants With Inpatient Admissions and Outpatient Visits
timeFrame: Data collected from index date up to 1-year of follow up period (for a maximum period of 3 years)
measure: Number of Inpatient Admissions Per Participant
timeFrame: Data collected from index date up to 1-year of follow up period (for a maximum period of 3 years)
measure: Number of Outpatient Visits Per Participant
timeFrame: Data collected from index date up to 1-year of follow up period (for a maximum period of 3 years)
measure: Percentage of Participants With Type of Hospital Admission
timeFrame: Data collected from index date up to 1-year of follow up period (for a maximum period of 3 years)
measure: Percentage of Participants With Reasons for Hospital Admission
timeFrame: Data collected from index date up to 1-year of follow up period (for a maximum period of 3 years)
measure: Duration of Inpatient Hospital Stay
timeFrame: Data collected from index date up to 1-year of follow up period (for a maximum period of 3 years)
measure: Reasons of Outpatient Visit
timeFrame: Data collected from index date up to 1-year of follow up period (for a maximum period of 3 years)
measure: Type of Health Care Professional Consultations During Outpatient Visit
timeFrame: Data collected from index date up to 1-year of follow up period (for a maximum period of 3 years)
measure: Percentage of Participants Contacted Cancer National Service (CNS) and Acute Oncology Service (AOS) During First Year After Palbociclib Initiation
timeFrame: Data collected from index date up to 1-year of follow up period (for a maximum period of 3 years)
measure: Percentage of Participants According to Number of CNS Interactions
timeFrame: Data collected from index date up to 1-year of follow up period (for a maximum period of 3 years)
measure: Number of AOS Interactions Per Participant
timeFrame: Data collected from index date up to 1-year of follow up period (for a maximum period of 3 years)
measure: Type of CNS and AOS Interactions
timeFrame: Data collected from index date up to 1-year of follow up period (for a maximum period of 3 years)
measure: Reasons for CNS and AOS Interaction
timeFrame: Data collected from index date up to 1-year of follow up period (for a maximum period of 3 years)
Other outcomes:
Locations:
city: Truro
country: United Kingdom
facility: Royal Cornwall Hospital
state: Cornwall
city: Brighton
country: United Kingdom
facility: Brighton Sussex Cancer Centre
city: Glasgow
country: United Kingdom
facility: Beatson West of Scotland Cancer Centre
city: London
country: United Kingdom
facility: Guys and St Thomas' NHS Trust
city: Maidstone
country: United Kingdom
facility: Maidstone Hospital
city: Manchester
country: United Kingdom
facility: The Christie NHS Foundation Trust
city: Newcastle upon Tyne
country: United Kingdom
facility: Newcastle Freeman Hospital
city: Wirral
country: United Kingdom
facility: Clatterbridge
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: