Breast NeoplasmsKNOWHERNCT03025711
Outcomes Of The Spanish Cohort Of Early Access To Pertuzumab And Trastuzumab Emtansine
An observational study of Breast Neoplasms, run by BELEN RUIZ-ANTORAN at 1 site in Spain.
Trial 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 Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Breast Neoplasms
- How many people
- 220 participants
- Where
- 1 siteSpain
- Study dates
- 1 Dec 2017to 31 Jan 2020
- Phase
- Observational
- Are there results?
- None posted in the registry
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 to 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
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.
Who may be able to join
- 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.
Who cannot join
- Given the characteristics of the study there are no exclusion criteria.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Use Of Pertuzumab And Trastuzumab Emtansine In Adult Patients With Her2-Positive Metastatic Or Locally Recurrent Unresectable Breast Cancer
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
Read the 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.
- Conditions
- Breast Neoplasms
- Keywords
- Advanced HER2+ breast cancer, Pertuzumab, Trastuzumab emtansine, Compassionate use, Early access program, Effectiveness, Safety
- Medical subject headings
- Breast Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Pertuzumab | Patients with HER2-positive metastatic or locally recurrent unresectable breast cancer and who are treated with Pertuzumab under Spanish compassionate use or early access program | |
| Trastuzumab emtansine | Patients with HER2-positive metastatic or locally recurrent unresectable breast cancer and who are treated with Trastuzumab emtansine (T-DM1) under Spanish compassionate use or early access program |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall survival.
The time between the date of start of treatment and the date of death. For subjects without documentation of death, OS will be censored on the last date the subject was known to be alive
Measured: Through study completion (from date of start of treatment until the date of death from any cause, assessed up to 48 months).
Secondary outcomes (10)
Progression free survival.
The time from start of treatment to the date of the first documented tumour progression as determined by the clinician (may be based on clinical examination or radiographic or laboratory features).
Measured: Through study completion (from date of start of treatment until the date of first documented progression assessed up to 48 months)
Best overall response rate
Response rate is defined as the proportion of patients with complete response (CR) or partial response (PR) based on their best overall response as written in the medical record
Measured: Through study completion, an average of 4 year
Duration of response (DOR)
The time between the date of first confirmed response to the date of the first documented tumour progression, or death due to any cause, whichever occurs first. At the time of the analysis, several limitations should be taken into consideration for this retrospective study: DOR is only appraisable if measurable disease and DOR data availability in the medical records (ideally assessed with the RECIST criteria) could be incomplete.
Measured: Through study completion, an average of 4 year
Time to treatment failure
Measured: Through study completion (from date of start of treatment until the date of treatment failure, assessed up to 48 months)
Time to Objective Response
The time from start of treatment to the date of the first confirmed response (evaluated for responders only)
Measured: Through study completion (from date of start of treatment until the date of the first confirmed response, assessed up to 48 months)
Time to change treatment
Measured: Through study completion (from date of start of treatment until the date of change treatment, assessed up to 48 months)
Time to next treatment
Measured: Through study completion (from date of start of treatment until the date of start other treatment, assessed up to 48 months)
All suspected Grade 3/4/5 adverse reactions
Measured: Through study completion, an average of 4 year
Adverse events of special interest to anti HER2 Mab (AESI)
- AESIs regarding treatment with T-DM1: Hepatic disorder (specific analytical alteration)
- AESIs regarding treatment with Pertuzumab: Interstitial Lung Disease
Measured: Through study completion, an average of 4 year
AEs of scientific interest
- An asymptomatic decline in LVEF requiring treatment or leading to discontinuation of study treatment (regarding treatment with T-DM1 and Pertuzumab)
- Other AEs leading to treatment discontinuation
Measured: Through study completion, an average of 4 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jan 2017 (estimated)
- Study start1 Dec 2017
- Primary completion1 Oct 2018
- Study completion31 Jan 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Breast NeoplasmsStudies of the same condition, open and under way.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 220 participants (actual)
Sponsor and investigators
- Lead sponsor
- BELEN RUIZ-ANTORAN
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, BELEN RUIZ-ANTORAN, Puerta de Hierro University Hospital
- Investigators
- Belén Ruiz-Antorán, PhD, Principal investigator, Department of Clinical Pharmacology, University Hospital Puerta de Hierro Majadahonda, Madrid, Spain
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03025711
- Sponsor's protocol ID
- ML29844
- Acronym
- KNOWHER
- Status last verified
- Mar 2020
- First submitted
- 11 Jan 2017
- Last updated
- 5 Mar 2020
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Use Of Pertuzumab And Trastuzumab Emtansine In Adult Patients With Her2-Positive Metastatic Or Locally Recurrent Unresectable Breast Cancer
- ClinicalTrials.gov ID
- NCT03025711
- Acronym / sponsor ID
- KNOWHER / ML29844
- Conditions
- Breast Neoplasms
- Keywords
- Advanced HER2+ breast cancer, Pertuzumab, Trastuzumab emtansine, Compassionate use, Early access program, Effectiveness, Safety
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 220 participants (actual)
- Lead sponsor
- BELEN RUIZ-ANTORANUniversity, hospital or other
- Study start
- 1 Dec 2017 (actual)
- Primary completion
- 1 Oct 2018 (actual)
- Study completion
- 31 Jan 2020 (actual)
- First posted
- 19 Jan 2017 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Pertuzumab | – | –Patients with HER2-positive metastatic or locally recurrent unresectable breast cancer and who are treated with Pertuzumab under Spanish compassionate use or early access program |
| Trastuzumab emtansine | – | –Patients with HER2-positive metastatic or locally recurrent unresectable breast cancer and who are treated with Trastuzumab emtansine (T-DM1) under Spanish compassionate use or early access program |
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- 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.
Key inclusion criteria (2 listed)
- 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.
Key exclusion criteria (1 listed)
- Given the characteristics of the study there are no exclusion criteria.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Overall survival.
Time frame: Through study completion (from date of start of treatment until the date of death from any cause, assessed up to 48 months).
The time between the date of start of treatment and the date of death. For subjects without documentation of death, OS will be censored on the last date the subject was known to be alive
10 secondary outcomes
Progression free survival.
Time frame: Through study completion (from date of start of treatment until the date of first documented progression assessed up to 48 months)
Best overall response rate
Time frame: Through study completion, an average of 4 year
Duration of response (DOR)
Time frame: Through study completion, an average of 4 year
Time to treatment failure
Time frame: Through study completion (from date of start of treatment until the date of treatment failure, assessed up to 48 months)
Time to Objective Response
Time frame: Through study completion (from date of start of treatment until the date of the first confirmed response, assessed up to 48 months)
Time to change treatment
Time frame: Through study completion (from date of start of treatment until the date of change treatment, assessed up to 48 months)
Time to next treatment
Time frame: Through study completion (from date of start of treatment until the date of start other treatment, assessed up to 48 months)
All suspected Grade 3/4/5 adverse reactions
Time frame: Through study completion, an average of 4 year
Adverse events of special interest to anti HER2 Mab (AESI)
Time frame: Through study completion, an average of 4 year
AEs of scientific interest
Time frame: Through study completion, an average of 4 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Mar 2020.
- 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.
Belén Ruiz-Antorán, PhD principal investigator
Department of Clinical Pharmacology, University Hospital Puerta de Hierro Majadahonda, Madrid, Spain
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Breast Neoplasms, run by BELEN RUIZ-ANTORAN at 1 site in Spain.
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 2 inclusion and 1 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 5 Mar 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Spain. 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Overall survival. (time frame: Through study completion (from date of start of treatment until the date of death from any cause, assessed up to 48 months).). 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 5 Mar 2020.