Clinical trial NCT03025711: Outcomes Of The Spanish Cohort Of Early Access To Pertuzumab And Trastuzumab Emtansine
Official title: Use Of Pertuzumab And Trastuzumab Emtansine In Adult Patients With Her2-Positive Metastatic Or Locally Recurrent Unresectable Breast Cancer
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Breast Neoplasms
Keywords: Advanced HER2+ breast cancer
Pertuzumab
Trastuzumab emtansine
Compassionate use
Early access program
Effectiveness
Safety
Lead sponsor: Puerta de Hierro University Hospital
Sponsor class: OTHER
First posted: 2017-01-19
Enrollment: 220
Sex: ALL
Minimum age (years): 18
Maximum age (years): 100
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: This study will include a cohort of approximately 700 adult patients from the Spain with HER2-positive metastatic or locally recurrent unresectable breast cancer and who are treated with Trastuzumab emtansine (T-DM1) or Pertuzumab under compassionate use or early access program. The decision to initiate use of Trastuzumab emtansine (T-DM1) and Pertuzumab is made independently by the participant and their health care provider and is not mandated by the study design or protocol.

Brief summary: The overall study objective is to evaluate the effectiveness and safety of Trastuzumab emtansine (T-DM1) and Pertuzumab under real-world disease conditions in the Spain, and specifically in patients treated under compassionate use or early access program

Detailed description: This is a retrospective, non-interventional, non-comparative, observational cohort study / registry in the Spain. The study design will reflect real-life clinical management of patients with HER2-positive MBC. Type and frequency of actual patient visits and all evaluations will be done as for routine clinical practice.

The analysis of the efficacy and safety results obtained in patients receiving pertuzumab or TDM1 in those early access systems is of utmost importance. These real-world patients with advanced breast cancer may have different characteristics than those enrolled in clinical trials and clinicians must often extrapolate into therapeutic decisions not fully supported by a robust evidence.

Eligibility criteria: Inclusion Criteria:

* Adult patients (age ≥ 18 years at enrolment) with HER2-positive metastatic or locally recurrent unresectable breast cancer and who are treated with Trastuzumab emtansine (T-DM1) or Pertuzumab.
* Patients who initiate Trastuzumab emtansine (T-DM1) and Pertuzumab under Spanish compassionate use or early access program.

Exclusion Criteria:

* Given the characteristics of the study there are no exclusion criteria.

Primary outcomes:
measure: Overall survival.
timeFrame: Through study completion (from date of start of treatment until the date of death from any cause, assessed up to 48 months).
Secondary outcomes:
measure: Progression free survival.
timeFrame: Through study completion (from date of start of treatment until the date of first documented progression assessed up to 48 months)
measure: Best overall response rate
timeFrame: Through study completion, an average of 4 year
measure: Duration of response (DOR)
timeFrame: Through study completion, an average of 4 year
measure: Time to treatment failure
timeFrame: Through study completion (from date of start of treatment until the date of treatment failure, assessed up to 48 months)
measure: Time to Objective Response
timeFrame: Through study completion (from date of start of treatment until the date of the first confirmed response, assessed up to 48 months)
measure: Time to change treatment
timeFrame: Through study completion (from date of start of treatment until the date of change treatment, assessed up to 48 months)
measure: Time to next treatment
timeFrame: Through study completion (from date of start of treatment until the date of start other treatment, assessed up to 48 months)
measure: All suspected Grade 3/4/5 adverse reactions
timeFrame: Through study completion, an average of 4 year
measure: Adverse events of special interest to anti HER2 Mab (AESI)
timeFrame: Through study completion, an average of 4 year
measure: AEs of scientific interest
timeFrame: Through study completion, an average of 4 year
Other outcomes:
Locations:
city: Madrid
country: Spain
facility: Puerta de Hierro University Hospital
Study officials:
affiliation: Department of Clinical Pharmacology, University Hospital Puerta de Hierro Majadahonda, Madrid, Spain
name: Belén Ruiz-Antorán, PhD
role: PRINCIPAL_INVESTIGATOR
References: