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Metastatic Breast CancerPhase 3MACS2096NCT01626222

4EVER - Efficacy, Safety, Health Economics, Translational Research of Postmenopausal Women With Estrogen Receptor Positive Locally Advanced or Metastatic Breast Cancer

A Phase 3 study testing Exemestane and Everolimus (RAD001) in people with Metastatic Breast Cancer, run by Novartis Pharmaceuticals at 84 sites in Germany.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The present multi-center, open-label, single-arm study aims to evaluate the efficacy and safety, quality of life and health resources utilization in postmenopausal women with hormone receptor positive breast cancer progressing following prior therapy with non-steroidal aromatase inhibitors (NSAI) treated with the… Read more about this study

Who is it for?
Ages 18 years and older, women only
Conditions the study is for
Metastatic Breast Cancer
What is being tested?
Exemestane and Everolimus (RAD001)
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
301 participants
Where
84 sitesGermany
Study dates
Jun 2012to Nov 2013
Phase
Phase 3
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 years and older
  • Women only
  • No healthy volunteers
  • Adult, Older adult

Main Inclusion criteria:

Metastatic or locally advanced breast cancer not amenable to curative treatment by surgery or radiotherapy or any other non-systemic treatment.

Histological or cytological confirmation of estrogen receptor positive (ER+) and/or progesterone receptor positive (PgR+), human epidermal growth factor receptor 2 (HER2) negative breast cancer Postmenopausal women. Disease progression following prior therapy with non steroidal aromatase inhibitors (NSAI), defined as: Recurrence while on, or following completion of an adjuvant treatment with Letrozole or Anastrozole, or Progression while on or following completion of Letrozole or Anastrozole treatment for ABC/MBC.

Radiological evidence of recurrence or progression on last systemic therapy prior to enrollment.

Patients must have at least one lesion that can be accurately measured or bone lesions: lytic or mixed (lytic + sclerotic) in the absence of measurable disease.

Written informed consent obtained before any screening procedure and according to local guidelines.

Other protocol defined inclusion criteria apply.

Main Exclusion criteria:

HER2-overexpressing patients by local laboratory testing (IHC 3+ staining or in situ hybridization positive).

Patients with only non-measurable lesions other than bone metastasis (e.g. pleural effusion, ascites etc.).

Previous treatment with mTOR inhibitors or known hypersensitivity to mTOR inhibitors.

Symptomatic brain or other CNS metastases. Previously treated brain metastases are allowed provided the patient is free of symptoms, prior radiotherapy for brain metastasis was more than four weeks before enrollment and the dose of corticosteroids is low (i.e. ≤ 10 mg/d Prednisolone equivalent) and stable for at least two weeks prior to enrollment.

Patients with Hepatitis B or C or with a history of Hepatitis B or C. Patients unwilling to or unable to comply with the protocol. Other protocol defined exclusion criteria apply.

Trial locations and contacts

84 sites in 1 country.

84 sites in 1 country

Germany84 sites
  • Novartis Investigative Site

    Aachen, 52074

  • Novartis Investigative Site

    Amberg, 92224

  • Novartis Investigative Site

    Augsburg, 86150

  • Novartis Investigative Site

    Augsburg, 86156

  • Novartis Investigative Site

    Bergisch Gladbach, 51465

  • Novartis Investigative Site

    Berlin, Germany, 12203

  • Novartis Investigative Site

    Berlin, 10367

  • Novartis Investigative Site

    Berlin, 10707

  • Novartis Investigative Site

    Berlin, 12552

  • Novartis Investigative Site

    Berlin, 12683

  • Novartis Investigative Site

    Berlin, 14169

  • Novartis Investigative Site

    Berlin, 14195

  • Novartis Investigative Site

    Böblingen, 71032

  • Novartis Investigative Site

    Bochum, 44787

  • Novartis Investigative Site

    Bonn, 53105

  • Novartis Investigative Site

    Bonn, 53111

  • Novartis Investigative Site

    Bottrop, 46236

  • Novartis Investigative Site

    Braunschweig, 38100

  • Novartis Investigative Site

    Bremen, 28209

  • Novartis Investigative Site

    Chemnitz, 09113

  • Novartis Investigative Site

    Cologne, 50935

  • Novartis Investigative Site

    Cologne, 50937

  • Novartis Investigative Site

    Donauwörth, 86609

  • Novartis Investigative Site

    Dresden, 01127

  • Novartis Investigative Site

    Düsseldorf, 40225

  • Novartis Investigative Site

    Erlangen, 91054

  • Novartis Investigative Site

    Essen, 45136

  • Novartis Investigative Site

    Essen, 45147

  • Novartis Investigative Site

    Esslingen am Neckar, 73730

  • Novartis Investigative Site

    Eutin, 23701

  • Novartis Investigative Site

    Frankfurt, 60389

  • Novartis Investigative Site

    Frankfurt, 60590

  • Novartis Investigative Site

    Freiburg im Breisgau, 79106

  • Novartis Investigative Site

    Fulda, 36043

  • Novartis Investigative Site

    Fürstenwalde, 15517

  • Novartis Investigative Site

    Fürth, 90766

  • Novartis Investigative Site

    Gera, 07548

  • Novartis Investigative Site

    Gerlingen, 70839

  • Novartis Investigative Site

    Goslar, 38642

  • Novartis Investigative Site

    Gütersloh, 33332

  • Novartis Investigative Site

    Halle, 06110

  • Novartis Investigative Site

    Halle, 06120

  • Novartis Investigative Site

    Hamburg, 20249

  • Novartis Investigative Site

    Hanover, 30177

  • Novartis Investigative Site

    Heidelberg, 69120

  • Novartis Investigative Site

    Jena, 07740

  • Novartis Investigative Site

    Kassel, 34125

  • Novartis Investigative Site

    Kiel, 24103

  • Novartis Investigative Site

    Kiel, 24105

  • Novartis Investigative Site

    Langen, 63225

  • Novartis Investigative Site

    Lemgo, 32657

  • Novartis Investigative Site

    Lüneburg, 21339

  • Novartis Investigative Site

    Magdeburg, 39120

  • Novartis Investigative Site

    Mainz, Germany, 55131

  • Novartis Investigative Site

    Mannheim, 68165

  • Novartis Investigative Site

    Marburg, 35039

  • Novartis Investigative Site

    Memmingen, 87700

  • Novartis Investigative Site

    Mönchengladbach, 41061

  • Novartis Investigative Site

    Mühlhausen, 99974

  • Novartis Investigative Site

    Mülheim, 45468

  • Novartis Investigative Site

    München, 80637

  • Novartis Investigative Site

    München, 80638

  • Novartis Investigative Site

    München, 81241

  • Novartis Investigative Site

    München, 81377

  • Novartis Investigative Site

    Münster, 48149

  • Novartis Investigative Site

    Nuremberg, 90403

  • Novartis Investigative Site

    Oldenburg, 26121

  • Novartis Investigative Site

    Plauen-Kauschwitz, 08525

  • Novartis Investigative Site

    Ravensburg, 88214

  • Novartis Investigative Site

    Recklinghausen, 45657

  • Novartis Investigative Site

    Rosenheim, 83022

  • Novartis Investigative Site

    Rostock, 18057

  • Novartis Investigative Site

    Singen, 78224

  • Novartis Investigative Site

    Soest, 59494

  • Novartis Investigative Site

    Speyer, 67346

  • Novartis Investigative Site

    Stralsund, 18435

  • Novartis Investigative Site

    Stuttgart, 70178

  • Novartis Investigative Site

    Trier, 54290

  • Novartis Investigative Site

    Troisdorf, 53840

  • Novartis Investigative Site

    Tübingen, 72076

  • Novartis Investigative Site

    Velbert, 42551

  • Novartis Investigative Site

    Villingen-Schwenningen, 78052

  • Novartis Investigative Site

    Weißenfels, 06667

  • Novartis Investigative Site

    Wuppertal, 42105

Other trials in Germany

About this study

Official title: A Phase IIIB, Multi-Center, Open Label Study For Postmenopausal Women With Estrogen Receptor Positive Locally Advanced or Metastatic Breast Cancer Treated With Everolimus (RAD001) in Combination With Exemestane: 4EVER - Efficacy, Safety, Health Economics, Translational Research

The present multi-center, open-label, single-arm study aims to evaluate the efficacy and safety, quality of life and health resources utilization in postmenopausal women with hormone receptor positive breast cancer progressing following prior therapy with non-steroidal aromatase inhibitors (NSAI) treated with the combination of Everolimus and Exemestane.

Read the detailed description

In light of the need for new treatment options for postmenopausal, hormone receptor positive, HER2 negative women after failure of prior non-steroidal aromatase inhibitor (NSAI) therapy, the BOLERO-2 trial was performed and demonstrated significant efficacy of the combinatorial treatment of Everolimus and Exemestane compared to an Exemestane monotherapy in this setting.

In this randomized, double blind, placebo-controlled trial a statistically significant improvement in progression-free survival (PFS) by adding Everolimus to exemestane versus Exemestane alone was reported. Adding Everolimus determined a 2.4-fold prolongation in PFS from 3.2 up to 7.4 months and so lowered the risk of cancer progression by 56% for these women. These findings were confirmed by an independent assessment (4.1 vs. 11.0 months, risk reduction: 64%). The quality of life data shows positive trend in the Everolimus plus Exemestane treatment arm. (Baselga 2011, Hortobagyi 2011). Thus, the benefit of the combinatorial treatment versus Exemestane monotherapy was shown in a defined patient population under controlled conditions.

The primary objective of this trial to assess the Overall Response Rate (ORR) in postmenopausal women with hormone receptor positive breast cancer progressing following prior therapy with NSAIs treated with the combination of Everolimus and Exemestane. The secondary objectives include, Progression free survival (PFS), Overall survival (OS), Safety, Change in Quality of life scores over time, Health resource utilization. The exploratory objectives reflect scientific interest within the treatment of metastatic breast cancer and are to be modified, if applicable, according to the current scientific state of the art at the time of actual analysis. These include: the influence of age, performance status, cancer activity and inflammation on anxiety and depression; changes in serum bone-turnover biomarkers; Pharmacogenetics of Everolimus in patients with advanced breast cancer; presence and molecular characteristics of Circulating Tumor Cells; correlation of response to Exemestane/Everolimus with Proteomic analysis.

The present national, multi-center, open-label, single-arm study aims to evaluate the efficacy and safety, quality of life and health resources utilization of the combination of Everolimus and Exemestane in a broader patient population compared to BOLERO-2, i.e. without limitations as to the number of previous chemotherapy lines, the time point of progression after NSAI therapy, and the previous endocrine therapy as patients under Exemestane monotherapy may be enrolled. Since the combination was shown to significantly improve PFS in the previous BOLERO-2 trial, for ethical reasons no endocrine comparator drugs will be investigated in the present study, due to the low efficacy of Exemestane monotherapy (PFS 3.2 months).

Being tested
Exemestane, Everolimus (RAD001)
Keywords
Postmenopausal, Estrogen Receptor Positive, HER2 negative, Locally Advanced, Metastatic Breast Cancer, Everolimus (RAD001), Exemestane
Medical subject headings
Breast Neoplasms

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ExemestaneDrug

    Exemestane is supplied by Novartis until Everolimus is commercially available for the study setting. Afterwards the investigator will prescribe Exemestane according to the individual label. Commercially available Exemestane will be supplied as tablets of 25 mg strength for oral administration. Complete guidelines for management and administration of Exemestane can be found in the package insert. Exemestane will be dosed starting on treatment Day 1. Patients will be instructed to take 1 tablet of 25 mg Exemestane orally. Package insert instructions should be followed. On the first day of each cycle, patients will receive an adequate drug supply (before commercial availability) or a prescription (after commercial availability) for self-administration at home. The investigator must emphasize compliance and will instruct the patient to take Exemestane exactly as prescribed.

  • Everolimus (RAD001)Drug

    Everolimus (RAD001) is supplied by Novartis until Everolimus is commercially available for the study setting. Afterwards the investigator will prescribe Everolimus according to the individual label. Everolimus is formulated as tablets of 10 and 5 mg strength for oral administration. All study medication will be packaged into blister packs. The blisters should be opened only at the time of administration, as the drugs are both hygroscopic and light sensitive. Everolimus will be dosed starting on treatment Day 1. Patients will be instructed to take 1 tablet × 10 mg Everolimus orally with a large glass of water once daily at the same time each day with or without food. On the first day of each cycle, patients will receive an adequate drug supply (before commercial availability) or a prescription (after commercial availability) for self-administration at home. The investigator must emphasize compliance and will instruct the patient to take Everolimus exactly as prescribed.

Study groups in NCT01626222
Study groupRoleWhat they receive
Everolimus & ExemestaneExperimentalThis study will be performed in 300 postmenopausal women with hormone receptor positive locally advanced or metastatic breast cancer progressing following prior therapy with non-steroidal aromatase inhibitors (NSAI) as defined by: 1. Recurrence while on or after completion of an adjuvant treatment including Letrozole or Anastrozole, or 2. Progression while on or following the completion of Letrozole or Anastrozole treatment for locally advanced or metastatic breast cancer. Except for prior use of mTOR inhibitors, there are no restrictions as to the last anticancer treatment prior to enrollment. Patients must have documented evidence of recurrence or progression on last therapy prior to enrollment. Written informed consent must be obtained prior to any screening procedures. The investigator or designee must ensure that only patients who meet all the following inclusion and none of the exclusion criteria are offered enrollment in the study.
Receives: Drug: Exemestane; Drug: Everolimus (RAD001)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Overall Response Rate (ORR) after 24 weeks of treatment

    The Overall response rate (ORR) is the proportion of patients with a best overall response of confirmed complete (CR) or partial (PR) response by Week 24. The best overall response is determined from the sequence of investigator overall lesion responses according to RECIST 1.1. To be assigned a best overall response of CR at least two determinations of CR at least 4 weeks apart before progression are required. To be assigned a best overall response of PR at least two determinations of PR or better at least 4 weeks apart before progression (and not qualifying for a CR) are required.

    Measured: 24 weeks

Secondary outcomes (6)
  1. Progression free survival (PFS) after 48 weeks of treatment

    Progression-free survival (PFS) is the time from date of start of treatment to the date of event defined as the first documented progression or death due to any cause. If a patient has not had an event, progression-free survival is censored at the date of last adequate tumor assessment.

    Measured: 48 weeks

  2. Overall Response Rate (ORR) after 48 weeks of treatment

    The ORR by Week 48 will be derived from the sequence of overall lesion responses as described for the primary efficacy variable. The ORR by Week 48 will be summarized using frequency tables presenting absolute and relative frequencies together with appropriate confidence intervals

    Measured: 48 weeks

  3. Overall survival (OS) after 48 weeks of treatment

    Overall survival (OS) is defined as the time from date of start of treatment to date of death due to any cause. If a patient is not known to have died, survival will be censored at the date of last contact. OS will be summarized using the Kaplan-Meier method.

    Measured: 48 weeks

  4. Safety within 48 weeks of treatment

    The assessment of safety will be based mainly on the frequency of adverse events and on the number of laboratory values that fall outside of pre-determined ranges. Other safety data (e.g. vital signs, and special tests) will be considered as appropriate. For all safety analyses, the safety set will be used.

    Measured: 48 weeks

  5. Resource utilization

    Data relating to Resource Utilization will be used for the purpose of economic evaluation, which will be carried out and reported as a separate activity.

    The study population receiving RAD001 plus Exemestane will be compared to alternative cohorts (e.g., purely endocrine treatment with Fulvestrant monotherapy, Exemestane monotherapy or chemotherapy, e.g. Capecitabine) using a Markov model. For each alternative therapy option, median PFS, OS and health-related quality of life will be determined by a systematic review of literature or databases.

    Measured: 48 weeks

  6. Health-related quality of life

    Health-related quality of life (HRQoL) will be assessed using the EORTC QLQ-C30 and BR23 questionnaires and the EuroQoL EQ-5D questionnaire. The HADS D questionnaire will be used to assess anxiety and depression. Scoring will follow the instructions of the respective manuals.

    Measured: 48 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted22 Jun 2012 (estimated)
  2. Study startJun 2012
  3. Primary completionNov 2013
  4. Study completionNov 2013

Resources

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 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
301 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01626222
Sponsor's protocol ID
CRAD001JDE49
Acronym
MACS2096
Status last verified
Feb 2017
First submitted
20 Jun 2012
Last updated
23 Feb 2017
Results posted
No
Data monitoring committee
No

Publications

  • Hadji P, Stoetzer O, Decker T, Kurbacher CM, Marme F, Schneeweiss A, Mundhenke C, Distelrath A, Fasching PA, Lux MP, Luftner D, Janni W, Muth M, Kreuzeder J, Quiering C, Grischke EM, Tesch H. The impact of mammalian target of rapamycin inhibition on bone health in postmenopausal women with hormone receptor-positive advanced breast cancer receiving everolimus plus exemestane in the phase IIIb 4EVER trial. J Bone Oncol. 2018 Oct 2;14:010-10. doi: 10.1016/j.jbo.2018.09.010. eCollection 2019 Feb.

    PubMed 30515367
  • Tesch H, Stoetzer O, Decker T, Kurbacher CM, Marme F, Schneeweiss A, Mundhenke C, Distelrath A, Fasching PA, Lux MP, Luftner D, Hadji P, Janni W, Muth M, Kreuzeder J, Quiering C, Taran FA. Efficacy and safety of everolimus plus exemestane in postmenopausal women with hormone receptor-positive, human epidermal growth factor receptor 2-negative locally advanced or metastatic breast cancer: Results of the single-arm, phase IIIB 4EVER trial. Int J Cancer. 2019 Feb 15;144(4):877-885. doi: 10.1002/ijc.31738. Epub 2018 Oct 30.

    PubMed 29992557

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Exemestane and Everolimus (RAD001) in people with Metastatic Breast Cancer, run by Novartis Pharmaceuticals at 84 sites in Germany.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

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 23 Feb 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 84 sites in Germany. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

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 Response Rate (ORR) after 24 weeks of treatment (time frame: 24 weeks). 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 NCT01626222, which also lists 2 linked publications.

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 23 Feb 2017.

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