PAlbociclib and Circulating Tumor DNA for ESR1 Mutation Detection

NCT ID: NCT03079011

Last Updated: 2025-11-18

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

1017 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-03-22

Study Completion Date

2025-09-22

Brief Summary

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This study is a randomized, open-label, multicentric, phase III trial conducted in patients receiving aromatase inhibitor and palbociclib as first line therapy for estrogen receptor (ER)-positive HER2-negative metastatic breast cancer and which aims to evaluate, at the onset of ESR1 mutations in circulating tumor DNA, the efficacy of a change of the hormone therapy (aromatase inhibitor (AI) changed to fulvestrant) combined to palbociclib, together with the safety of hormone therapy and palbociclib combination in the overall population.

Detailed Description

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Conditions

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Metastatic Breast Cancer

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SEQUENTIAL

Step 1: 1000 patients screened for circulating blood ESR1 mutation detection at regular intervals will be treated with palbociclib 125 mg once daily for 21 days followed by 7 days off (28-day cycle) + AI (letrozole, anastrozole or exemestane) administered once daily in a continuous scheme until tumor progression (assessed by RECIST) or ESR1 mutation detection

Step 2: Up to 200 Patients with a rising circulating ESR1 mutation and without tumor progression will be randomized (1:1):

* ARM A: no change in therapy until tumor progression (RECIST) or possibility of a cross-over (step 3)
* ARM B: palbociclib 125 mg + fulvestrant 500 mg administered intramuscularly on D1,15 and 29 and once monthly thereafter until tumor progression (RECIST)
* Step 3 (cross over): up to 80 patients who have been randomized in arm A will be offered to be treated by fulvestrant + palbociclib, after having progressed under AI + palbociclib until tumor progression (RECIST) under fulvestrant + palbociclib.
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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A- palbociclib + AI

After randomization, the patient will be treated with palbociclib 125 mg once daily for 21 days followed by 7 days off to complete a 28-day cycle in combination with an aromatase inhibitor (letrozole, anastrozole or exemestane, according to physician's choice and according their respective summary product characteristics) administered once daily in a continuous scheme.

Group Type EXPERIMENTAL

Palbociclib 125mg

Intervention Type DRUG

Palbociclib 125 mg once daily for 21 days followed by 7 days off to complete a 28-day cycle

Aromatase Inhibitors

Intervention Type DRUG

Letrozole: 2.5 mg once daily on a continuous scheme (tablets, per os) Anastrozole:1 mg once daily on a continuous scheme (tablets , per os) Exemestane: 25 mg once daily on a continuous scheme (tablets, per os)

B- Palbociclib + fulvestrant

After randomization, the patient will be treated with palbociclib 125 mg once daily for 21 days followed by 7 days off to complete a 28-day cycle in combination with fulvestrant, a selective estrogen receptor down-regulator, 500 mg administered intramuscularly on Days 1, 15, and 29 and once monthly thereafter.

Group Type EXPERIMENTAL

Palbociclib 125mg

Intervention Type DRUG

Palbociclib 125 mg once daily for 21 days followed by 7 days off to complete a 28-day cycle

Fulvestrant Injectable Product

Intervention Type DRUG

500 mg by intramuscular injection on days 1 and 15 of cycle one and then on day one of each subsequent cycle (28 days)

Selection - Palbociclib + AI

All patients included into the study will be treated with palbociclib 125 mg once daily for 21 days followed by 7 days off to complete a 28-day cycle in combination with an aromatase inhibitor (letrozole, anastrozole or exemestane, according to physician's choice and according their respective summary product characteristics) administered once daily in a continuous scheme

Group Type EXPERIMENTAL

Palbociclib 125mg

Intervention Type DRUG

Palbociclib 125 mg once daily for 21 days followed by 7 days off to complete a 28-day cycle

Aromatase Inhibitors

Intervention Type DRUG

Letrozole: 2.5 mg once daily on a continuous scheme (tablets, per os) Anastrozole:1 mg once daily on a continuous scheme (tablets , per os) Exemestane: 25 mg once daily on a continuous scheme (tablets, per os)

Interventions

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Palbociclib 125mg

Palbociclib 125 mg once daily for 21 days followed by 7 days off to complete a 28-day cycle

Intervention Type DRUG

Aromatase Inhibitors

Letrozole: 2.5 mg once daily on a continuous scheme (tablets, per os) Anastrozole:1 mg once daily on a continuous scheme (tablets , per os) Exemestane: 25 mg once daily on a continuous scheme (tablets, per os)

Intervention Type DRUG

Fulvestrant Injectable Product

500 mg by intramuscular injection on days 1 and 15 of cycle one and then on day one of each subsequent cycle (28 days)

Intervention Type DRUG

Other Intervention Names

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Letrozole, Anastrozole or exemestane

Eligibility Criteria

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Inclusion Criteria

1. Women with proven loco-regionally recurrent or metastatic adenocarcinoma of the breast not amenable to curative therapy with disease considered potentially sensitive to aromatase inhibitors Note: patients relapsing while on adjuvant tamoxifen or other non-aromatase inhibitor adjuvant endocrine therapy and patients relapsing more than one year after the end of aromatase inhibitor adjuvant therapy are eligible for the present study;
2. Age ≥18 years;
3. Life expectancy \>3 months;
4. Eastern Cooperative Oncology Group (ECOG) performance status (PS) 0-2;
5. Estrogen Receptor (ER)-positive and HER2-negative breast cancer. Where available, assessment of Estrogen Receptor status should be based on the most recent tumor sample; to be considered as ER-positive, the most recent breast cancer tissue examined must display at least 10% of cancer cells with positive ER staining;
6. Tumor block (primary tumor or metastasis) available;
7. No prior systemic anti-cancer therapy for metastatic or advanced disease (chemotherapy, targeted therapy or hormone therapy); prior initiation of LHRH agonist or bone-directed agents is however allowed);
8. Menopausal patients or patients with suppressed ovarian function

* Women with bilateral oophorectomy
* Postmenopausal women, as defined by any of the following criteria:

* Age 60 or over;
* Age 50 to 59 years and meets one of the following criteria:

* Amenorrhea for ≥24 months and follicle-stimulating hormone within the postmenopausal range;
* patients with hysterectomy or chemotherapy-induced amenorrhea must display follicle-stimulating hormone within the postmenopausal range;
* Other women, provided they are being treated with monthly LHRH analogues (first injection performed ≥7 days before the treatment initiation) and are willing to continue to receive LHRH agonist therapy for the duration of the trial;
9. Patients may have measurable (according to Response Evaluation Criterion in Solid Tumors (RECIST v1.1) or not measurable disease

* Patients with only blastic bone lesions are not eligible;
* Patients with only pleural, cardiac or peritoneal effusion or meningeal carcinomatosis are not eligible;
10. Adequate organ and marrow function as defined below:

* Hemoglobin ≥90 g/L
* Absolute neutrophil count ≥1.5 g/L
* Platelet count ≥100 g/L
* Serum bilirubin ≤1.5 × upper limit of normal (ULN). This will not apply to patients with confirmed Gilbert's syndrome.
* ALT and AST ≤3 × ULN;
* Alkaline phosphatase ≤2.5 x ULN (≤5.0 x ULN if bone or liver metastases present)
* Serum creatinine ≤1.5 × ULN or calculated creatinine clearance ≥ 60 mL/min as determined by Cockcroft-Gault (using actual body weight) formula for females \[creatinine clearance =Weight (kg) × (140 - Age) × 0.85 (mL/min)/ (72 × serum creatinine (mg/dL))
11. Willingness and ability to comply with scheduled visits, treatment plan, laboratory tests, and any protocol-related procedures including screening evaluations;
12. Resolution of all acute toxic effects of prior anti-cancer therapy or surgical procedures to NCI CTCAE version 4.03 Grade 1 (except alopecia or other toxicities not considered a safety risk for the patient at investigator's discretion);
13. Written informed consent obtained prior to performing any protocol-related procedures including screening evaluations;
14. Patient affiliated to a social security system.

Exclusion Criteria

1. Locally advanced breast cancer or loco-regional relapse amenable for any treatment with curative intent;
2. Her2-positive or equivocal tumor status either on the primary or on the recurrent tumor, defined as IHC3+, Fish/Cish amplified or Fish/Cish equivocal according to the ASCO2015 criteria;
3. Prior endocrine therapy in the metastatic setting is not allowed;
4. Prior treatment with any CDK 4/6 inhibitor in the adjuvant or metastatic setting (neoadjuvant/preoperative treatment is allowed); however, prior therapy with another targeted treatment in the adjuvant setting is allowed;
5. Visceral crisis: Advanced, symptomatic, visceral spread that is at risk of life-threatening complication in the short term and that requires chemotherapy;
6. Any major surgery (defined as requiring general anaesthesia) or significant traumatic injury within 4 weeks of treatment initiation or patients that may require major surgery during the course of the study; however, surgical diagnostic procedure is allowed (even if performed under general anaesthesia);
7. Known, active bleeding diathesis;
8. Any serious known concomitant systemic disorder (e.g. known active infection including HIV, or cardiac disease) incompatible with the study (at the discretion of investigator), previous history of bleeding diathesis, or anti-coagulation treatment (the use of low molecular weight heparin is allowed);
9. Patients unable to swallow tablets;
10. History of mal-absorption syndrome or other condition that would interfere with enteral absorption;
11. Chronic daily treatment with corticosteroids with a dose of ≥10 mg/day methylprednisolone equivalent (excluding inhaled steroids);
12. Known active uncontrolled or symptomatic central nervous system (CNS) metastases, carcinomatous meningitis, or leptomeningeal disease as indicated by clinical symptoms, cerebral oedema, and/or progressive growth. Patients with a history of CNS metastases or cord compression are eligible if they have been definitively treated with local therapy (e.g., radiotherapy, stereotactic surgery) and are clinically stable and off anticonvulsants and steroids for at least 4 weeks before treatment start;
13. Known hypersensitivity to letrozole, anastrozole, exemestane, fulvestrant, palbociclib or any of their excipients;
14. Uncontrolled electrolyte disorders that can compound the effects of a QTc prolonging drug (e.g., hypocalcemia, hypokalemia, hypomagnesemia);
15. Patients treated within the last 7 days prior to treatment start in the trial with drug that are known to be CYP3A4 inhibitors, drugs that are known to be CIP3A4 inducers, who underwent a grapefruit cure;
16. Patients already included in another therapeutic trial evaluating an investigational medicinal product or having received an investigational medicinal product within 3 months;
17. History of previous:

* Any other stage II, III, IV cancer within 5 years preceding patient enrollment in the trial - however, multiple primary breast cancers (controlateral/ipsilateral cancers/local relapses) are allowed pending all tumor masses were ER+;
* Any history of hematological malignancy;
18. Persons deprived of their freedom or under guardianship or incapable of giving consent;
19. Pregnancy or lactation period. Women of childbearing potential must implement adequate non-hormonal contraceptive measures (barrier methods, intrauterine contraceptive devices, sterilization; LHRH agonist cannot be considered as an efficient contraceptive measure) during study treatment and for 90 days after discontinuation. A serum pregnancy test must be negative in premenopausal women or women with amenorrhea of less than 12 months.
Minimum Eligible Age

18 Years

Eligible Sex

FEMALE

Accepts Healthy Volunteers

No

Sponsors

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Pfizer

INDUSTRY

Sponsor Role collaborator

UNICANCER

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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François-Clément BIDARD, MD PhD

Role: PRINCIPAL_INVESTIGATOR

Institut Curie

Suzette DELALOGE, MD PhD

Role: PRINCIPAL_INVESTIGATOR

Gustave Roussy, Cancer Campus, Grand Paris

Anne-Claire HARDY BESSARD, MD

Role: PRINCIPAL_INVESTIGATOR

Centre Armoricain d'Oncologie

Locations

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Centre Antoine Lacassagne

Nice, , France

Site Status

CHP Saint Grégoire

Saint-Grégoire, , France

Site Status

Centre ONCOGARD - Institut de Cancérologie du Gard

Nîmes, , France

Site Status

Institut Curie

Paris, , France

Site Status

Hopital Diaconesses-Croix Saint Simon

Paris, , France

Site Status

Hopital Européen Georges Pompidou

Paris, , France

Site Status

Saint Louis Hospital

Paris, , France

Site Status

Centre Hospitalier de Pau

Pau, , France

Site Status

Centre Catalan d'Oncologie

Perpignan, , France

Site Status

Polyclinique Francheville

Périgueux, , France

Site Status

Centre Hospitalier Lyon Sud

Pierre-Bénite, , France

Site Status

CARIO - Centre Armoricain Radiothérapie Imagerie Médicale et Oncologi

Plérin, , France

Site Status

Centre Hospitalier de Cornouaille

Quimper, , France

Site Status

Clinique de la Croix du Sud

Quint-Fonsegrives, , France

Site Status

Centre Eugène Marquis

Rennes, , France

Site Status

Centre de radiothérapie et d'oncologie médicale LE-CROME

Ris-Orangis, , France

Site Status

Centre Henri Becquerel

Rouen, , France

Site Status

Centre Hospitalier Saint-Brieuc

Saint-Brieuc, , France

Site Status

Institut Curie - Hôpital René Huguenin

Saint-Cloud, , France

Site Status

Centre Hospitalier Universitaire

Amiens, , France

Site Status

Clinique de l'Europe

Amiens, , France

Site Status

Institut de Cancérologie de l'Ouest - Site Paul Papin

Angers, , France

Site Status

Centre Hospitalier d'Auxerre

Auxerre, , France

Site Status

Institut Sainte Catherine

Avignon, , France

Site Status

Centre Hospitalier de Beauvais

Beauvais, , France

Site Status

Centre Hospitalier de Blois

Blois, , France

Site Status

Clinique Tivoli Ducos

Bordeaux, , France

Site Status

Institut Bergonié

Bordeaux, , France

Site Status

Polyclinique Bordeaux Nord Aquitaine

Bordeaux, , France

Site Status

Centre Hospitalier de boulogne sur Mer

Boulogne-sur-Mer, , France

Site Status

Centre Hospitalier Fleyriat

Bourg-en-Bresse, , France

Site Status

CHRU Morvan

Brest, , France

Site Status

Clinique PASTEUR-CFRO

Brest, , France

Site Status

Centre Francois Baclesse

Caen, , France

Site Status

Centre Hospitalier René Dubos

Cergy-Pontoise, , France

Site Status

Medipole de Savoie

Challes-les-Eaux, , France

Site Status

Centre Hôpital Sainte Marie

Chalon-sur-Saône, , France

Site Status

CH William Morey

Chalon-sur-Saône, , France

Site Status

Centre Hospitalier Métropole de Savoie

Chambéry, , France

Site Status

Centre Hospitalier de Cholet

Cholet, , France

Site Status

Centre Jean Perrin

Clermont-Ferrand, , France

Site Status

Pôle Santé République

Clermont-Ferrand, , France

Site Status

Centre Georges François Leclerc

Dijon, , France

Site Status

CHI Fréjus St-Raphaël

Fréjus, , France

Site Status

Institut Daniel Hollard - Groupe Hospitalier Mutualiste de Grenoble

Grenoble, , France

Site Status

Centre Hospitalier Départemental de Vendée

La Roche-sur-Yon, , France

Site Status

Clinique du Cap d'Or

La Seyne-sur-Mer, , France

Site Status

Centre Hospitalier de Versailles

Le Chesnay, , France

Site Status

Centre Hospitalier le Mans

Le Mans, , France

Site Status

Institut Hospitalier Franco-Britannique

Levallois-Perret, , France

Site Status

CHU Dupuytren

Limoges, , France

Site Status

Clinique François Chénieux

Limoges, , France

Site Status

Centre Hospitalier Bretagne Sud

Lorient, , France

Site Status

Centre Leon Berard

Lyon, , France

Site Status

Clinique de la Sauvegarde

Lyon, , France

Site Status

Hôpital privé Jean Mermoz

Lyon, , France

Site Status

Hôpital Européen Marseille

Marseille, , France

Site Status

Hôpital Saint Joseph

Marseille, , France

Site Status

Institut Paoli Calmettes

Marseille, , France

Site Status

Centre d'Oncologie radiothérapie de Macon

Mâcon, , France

Site Status

Clinique Claude Bernard

Metz, , France

Site Status

Centre Hospitalier ANNECY GENEVOIS

Metz-Tessy, , France

Site Status

CH Mont de Marsan

Mont-de-Marsan, , France

Site Status

Centre Hospitalier Montceau les mines

Montceau-les-Mines, , France

Site Status

ICM - Val d'Aurelle

Montpellier, , France

Site Status

Centre d'Oncologie de Gentilly

Nancy, , France

Site Status

Hopital Privé du Confluent

Nantes, , France

Site Status

Institut de Cancérologie de l'Ouest - Site René Gauducheau

Saint-Herblain, , France

Site Status

Clinique Mutualiste de l'Estuaire

Saint-Nazaire, , France

Site Status

Institut de cancérologie lucien Neuwith

Saint-Priest-en-Jarez, , France

Site Status

Centre Hospitalier de Broussais

St-Malo, , France

Site Status

Centre Paul Stauss

Strasbourg, , France

Site Status

Clinique de l'Orangerie

Strasbourg, , France

Site Status

Clinique Sainte Anne

Strasbourg, , France

Site Status

Hôpitaux Universitaires de Strasbourg

Strasbourg, , France

Site Status

Hôpitaux du Léman

Thonon-les-Bains, , France

Site Status

Clinique Pasteur

Toulouse, , France

Site Status

Institut Claudius Regaud

Toulouse, , France

Site Status

Centre Hospitalier de Tours - Hopital Bretonneau

Tours, , France

Site Status

Centre Hospitalier Bretagne Atlantique

Vannes, , France

Site Status

UNEOS Site Hôpital Robert Schuman

Vantoux, , France

Site Status

Gustave Roussy

Villejuif, , France

Site Status

Countries

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France

References

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Bidard FC, Hardy-Bessard AC, Dalenc F, Bachelot T, Pierga JY, de la Motte Rouge T, Sabatier R, Dubot C, Frenel JS, Ferrero JM, Ladoire S, Levy C, Mouret-Reynier MA, Lortholary A, Grenier J, Chakiba C, Stefani L, Plaza JE, Clatot F, Teixeira L, D'Hondt V, Vegas H, Derbel O, Garnier-Tixidre C, Canon JL, Pistilli B, Andre F, Arnould L, Pradines A, Bieche I, Callens C, Lemonnier J, Berger F, Delaloge S; PADA-1 investigators. Switch to fulvestrant and palbociclib versus no switch in advanced breast cancer with rising ESR1 mutation during aromatase inhibitor and palbociclib therapy (PADA-1): a randomised, open-label, multicentre, phase 3 trial. Lancet Oncol. 2022 Nov;23(11):1367-1377. doi: 10.1016/S1470-2045(22)00555-1. Epub 2022 Sep 29.

Reference Type DERIVED
PMID: 36183733 (View on PubMed)

Berger F, Marce M, Delaloge S, Hardy-Bessard AC, Bachelot T, Bieche I, Pradines A, De La Motte Rouge T, Canon JL, Andre F, Arnould L, Clatot F, Lemonnier J, Marques S, Bidard FC; PADA-1 investigators. Randomised, open-label, multicentric phase III trial to evaluate the safety and efficacy of palbociclib in combination with endocrine therapy, guided by ESR1 mutation monitoring in oestrogen receptor-positive, HER2-negative metastatic breast cancer patients: study design of PADA-1. BMJ Open. 2022 Mar 3;12(3):e055821. doi: 10.1136/bmjopen-2021-055821.

Reference Type DERIVED
PMID: 35241469 (View on PubMed)

Other Identifiers

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2016-004360-18

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

UC-0140/1615 - UCBG3-05

Identifier Type: -

Identifier Source: org_study_id

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