Clinical trial NCT04524728: Patterns of Treatment and Outcome of Palbociclib Plus Endocrine Therapy
Official title: Patterns of Treatment and Outcome of Palbociclib Plus Endocrine Therapy in Hormone Receptor-positive (HR+)/HER2 Receptor-negative (HER2-) Metastatic Breast Cancer (MBC): a Real World Multicentre Italian Study
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Breast
Neoplasm Malignant
Female
Keywords: endocrine therapy
metastatic breast cancer
palbociclib
real world
visceral involvement
Lead sponsor: Istituti Clinici Scientifici Maugeri SpA
Sponsor class: OTHER
First posted: 2020-08-24
Enrollment: 191
Sex: FEMALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: One hundred-ninety-one patients (92 in cohort A, 99 in cohort B) were enrolled and treated and 182 were evaluable for the analysis. Median age was 62 years (range 47-79)

Brief summary: This is a multicentre real-world experience aimed at verifying the outcome of palbociclib plus ET in an unselected population of MBC patients. The primary endpoint is the clinical benefit rate (CBR); secondary aims are the median PFS (mPFS), overall survival (OS) and safety.

Detailed description: This is an open-label, longitudinal, prospective, multicentre cohort study. Eligible patients are pre- and postmenopausal women with a histologically proven HR+ MBC, candidate to receive palbociclib plus endocrine therapy (ET) as first or subsequent line of therapy according to their contingent clinical situation. Additional inclusion criteria are HER2- disease (immunohistochemistry (IHC) 0-1+ or IHC 2+, confirmed as fluorescence in situ hybridization \[FISH\] negative), presence of measurable or evaluable lesions and life expectancy of at least 4 months. They need to have adequate bone marrow, hepatic and renal function, according to clinical practice guidelines for antineoplastic drug administration. Previous chemotherapy or ET for metastatic disease is allowed. Patients receive palbociclib 125 mg daily, 3 weeks on/1 week off in a 28-day cycle, combined with letrozole 2.5 mg administered orally on a continuous daily dosing schedule (cohort A) or fulvestrant at the dose of 500 mg intramuscular on days 1, 14, 28, then every 4 weeks thereafter (cohort B). Premenopausal women receive a GnRH analogue in combination with ET and palbociclib. Treatment is administered until documented disease progression (PD), unacceptable toxicity or patient refusal. The tumour assessment is performed approximately every 16 weeks. Treatment efficacy is evaluated by Response Evaluation Criteria In Solid Tumors (RECIST version 1.1). A complete blood count and organ function test is performed before each cycle, through study completion, an avarange of 1 year. No pre-specified treatment modifications are planned; dose reductions, delay or discontinuations of palbociclib are performed according to observed side effects. AEs are recorded and graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) (version 5.0).

The primary aim of the study is to analyse the activity of palbociclib plus ET in terms of clinical benefit rate that is defined as the percentage of patients experiencing complete response (CR), partial response (PR), or stable disease (SD) lasting 6 months or more. Secondary aims include the evaluation of the safety of the treatments, progression-free survival and overall survival.

Eligibility criteria: Inclusion Criteria:

* Pre- and postmenopausal women with a histologically proven HR+MBC, candidate to receive palbociclib plus ET as first or subsequent line of therapy according to their contingent clinical situation.
* HER2- disease (IHC 0-1 or IHC 2, confirmed as FISH negative), presence of measurable or evaluable lesions and life expectancy of at least 4 months.
* Adequate bone marrow, hepatic and renal function, according to clinical practice guidelines for antineoplastic drug administration

Exclusion Criteria:

* ER- PgR- disease
* HER2+ disease (IHC 3 or IHC 2, confirmed as FISH positive)
* Any cardiovascular, renal or hepatic condition that would compromise conditions in the opinion of the investigator

Primary outcomes:
measure: Clinical Benefit Rate (CBR)
timeFrame: From the date of randomization through study completion, assessed up to 16 weeks
Secondary outcomes:
measure: Median PFS
timeFrame: From date of randomization until the date of first documented progression, assessed up to 16 weeks through study completion
measure: Overall survival
timeFrame: from the date of randomization until the date of death from any cause or lost of follow-up, whichever came first, assessed up to 100 months.
measure: Drug safety and tolerability
timeFrame: at day 1 of any cycle from the date of the start of therapy through study completion, an avarange of 1 year
Other outcomes:
Locations:
city: Pavia
country: Italy
facility: Istituti Clinici Scientifici Maugeri IRCCS
Study officials:
affiliation: ICS Maugeri, IRCCS, Department of Medical Oncology, Pavia, italy
name: Raffaella Palumbo, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: