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Pertuzumab in Platinum-Resistant Low Human Epidermal Growth Factor Receptor 3 (HER3) Messenger Ribonucleic Acid (mRNA) Epithelial Ovarian Cancer (PENELOPE)

Registration Number
NCT01684878
Lead Sponsor
Hoffmann-La Roche
Brief Summary

This two-part, multicenter study will evaluate the safety, tolerability and efficacy of pertuzumab in combination with standard chemotherapy in women with recurrent platinum-resistant epithelial ovarian cancer. In the non-randomized Part 1 safety run-in, participants will receive pertuzumab plus either topotecan or paclitaxel. In the randomized, double-blind Part 2 of the study, participants will receive either pertuzumab or placebo in combination with chemotherapy (topotecan, paclitaxel, or gemcitabine).

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
Female
Target Recruitment
208
Inclusion Criteria
  • Histologically or cytologically confirmed epithelial ovarian, primary peritoneal, and/or fallopian tube cancer that is platinum-resistant or refractory
  • Low Human epidermal growth factor receptor (HER) 3 messenger ribonucleic acid (mRNA) expression
  • At least one measurable and/or non-measurable lesion according to Response Evaluation Criteria in Solid Tumors (RECIST) version (V) 1.1
  • Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2
  • Left ventricular ejection fraction (LVEF) greater than or equal to (>/=) 50 percent (%)
  • Negative serum pregnancy test in women of childbearing potential
  • Women of childbearing potential must agree to use effective contraception as defined by protocol during and for at least 6 months post study treatment
Exclusion Criteria
  • Non-epithelial tumors
  • Ovarian tumors with low malignant potential (borderline tumors)
  • History of other malignancy of prognostic relevance within the last 5 years, except for carcinoma in situ of the cervix or basal cell carcinoma, or tumors with a negligible risk for metastasis or death, such as adequately controlled basal-cell carcinoma or squamous cell carcinoma of the skin or carcinoma in situ of the cervix or carcinoma in situ of the breast
  • Previous treatment with more than 2 chemotherapy regimens
  • Any prior radiotherapy to the pelvis or abdomen
  • History or evidence on physical/neurological examination of central nervous system disease unrelated to cancer (uncontrolled seizures), unless adequately treated with standard medical therapy
  • Pre-existing peripheral neuropathy >/= common toxicity criteria (CTC) grade 2 (applicable for paclitaxel cohort only)
  • Inadequate organ function
  • Uncontrolled hypertension or clinically significant cardiovascular disease
  • Current known infection with human immunodeficiency virus (HIV) or active infection with hepatitis B virus (HBV), or hepatitis C virus (HCV)
  • Current chronic daily treatment with corticosteroids (>/= 10 mg per day of methylprednisolone or equivalent), excluding inhaled steroids
  • History of receiving any investigational treatment within 28 days prior to first study drug administration
  • For Part 2 of the trial: prior enrollment into Part 1 of the trial
  • Concurrent participation in any therapeutic clinical trial

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Part 2: Pertuzumab+ChemotherapyPaclitaxel (Chemotherapy)Participants received pertuzumab and chemotherapy (paclitaxel or topotecan or gemcitabine) in cycles of 3 weeks until progressive disease as per investigator's assessment, unacceptable toxicity, withdrawal of consent, or death. Chemotherapy was administered as per investigators discretion.
Part 1: Pertuzumab + PaclitaxelPaclitaxel (Chemotherapy)Participants received pertuzumab and paclitaxel in cycles of 3 weeks until progressive disease as per investigator's assessment, unacceptable toxicity, withdrawal of consent, or death.
Part 1: Pertuzumab + TopotecanPaclitaxel (Chemotherapy)Participants received pertuzumab and topotecan in cycles of 3 weeks until progressive disease as per investigator's assessment, unacceptable toxicity, withdrawal of consent, or death.
Part 2: Placebo+ChemotherapyGemcitabine (Chemotherapy)Participants received pertuzumab matching placebo and chemotherapy (paclitaxel or topotecan or gemcitabine) in cycles of 3 weeks until progressive disease as per investigator's assessment, unacceptable toxicity, withdrawal of consent, or death. Chemotherapy was administered as per investigators discretion.
Part 1: Pertuzumab + TopotecanTopotecan (Chemotherapy)Participants received pertuzumab and topotecan in cycles of 3 weeks until progressive disease as per investigator's assessment, unacceptable toxicity, withdrawal of consent, or death.
Part 1: Pertuzumab + PaclitaxelPlaceboParticipants received pertuzumab and paclitaxel in cycles of 3 weeks until progressive disease as per investigator's assessment, unacceptable toxicity, withdrawal of consent, or death.
Part 2: Pertuzumab+ChemotherapyGemcitabine (Chemotherapy)Participants received pertuzumab and chemotherapy (paclitaxel or topotecan or gemcitabine) in cycles of 3 weeks until progressive disease as per investigator's assessment, unacceptable toxicity, withdrawal of consent, or death. Chemotherapy was administered as per investigators discretion.
Part 2: Pertuzumab+ChemotherapyTopotecan (Chemotherapy)Participants received pertuzumab and chemotherapy (paclitaxel or topotecan or gemcitabine) in cycles of 3 weeks until progressive disease as per investigator's assessment, unacceptable toxicity, withdrawal of consent, or death. Chemotherapy was administered as per investigators discretion.
Part 2: Placebo+ChemotherapyPlaceboParticipants received pertuzumab matching placebo and chemotherapy (paclitaxel or topotecan or gemcitabine) in cycles of 3 weeks until progressive disease as per investigator's assessment, unacceptable toxicity, withdrawal of consent, or death. Chemotherapy was administered as per investigators discretion.
Part 2: Placebo+ChemotherapyPaclitaxel (Chemotherapy)Participants received pertuzumab matching placebo and chemotherapy (paclitaxel or topotecan or gemcitabine) in cycles of 3 weeks until progressive disease as per investigator's assessment, unacceptable toxicity, withdrawal of consent, or death. Chemotherapy was administered as per investigators discretion.
Part 2: Placebo+ChemotherapyTopotecan (Chemotherapy)Participants received pertuzumab matching placebo and chemotherapy (paclitaxel or topotecan or gemcitabine) in cycles of 3 weeks until progressive disease as per investigator's assessment, unacceptable toxicity, withdrawal of consent, or death. Chemotherapy was administered as per investigators discretion.
Part 1: Pertuzumab + PaclitaxelPertuzumabParticipants received pertuzumab and paclitaxel in cycles of 3 weeks until progressive disease as per investigator's assessment, unacceptable toxicity, withdrawal of consent, or death.
Part 1: Pertuzumab + TopotecanPertuzumabParticipants received pertuzumab and topotecan in cycles of 3 weeks until progressive disease as per investigator's assessment, unacceptable toxicity, withdrawal of consent, or death.
Part 2: Pertuzumab+ChemotherapyPertuzumabParticipants received pertuzumab and chemotherapy (paclitaxel or topotecan or gemcitabine) in cycles of 3 weeks until progressive disease as per investigator's assessment, unacceptable toxicity, withdrawal of consent, or death. Chemotherapy was administered as per investigators discretion.
Primary Outcome Measures
NameTimeMethod
Part 1: Percentage of Participants With Adverse Events (AEs)Approximately 28 months (assessed at screening, baseline until 28 days after the last dose of study treatment)

An AE can be any unfavorable and unintended sign (including an abnormality laboratory finding), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product.

Part 2: Progression Free Survival (PFS) as Assessed by a Blinded Independent Review Committee (IRC) Including Malignant Bowel Obstruction (MBO)Approximately 44 months (assessed at screening and every 9 weeks from randomization until disease progression)

PFS (IRC-Assessed) was defined as the time from randomization into Part 2 of the trial until progressive disease (PD), MBO or death from any cause, whichever occurred first per Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1. PD could base on symptom deterioration or was defined as at least a 20% increase in the sum of the longest diameter of target lesions, taking as reference the smallest sum longest diameter recorded since treatment started or the appearance of one or more new lesions and/or the unequivocal progression of existing non-target lesions.

Secondary Outcome Measures
NameTimeMethod
Part 2: Overall SurvivalApproximately 44 months (assessed at screening and every 9 weeks from randomization until disease progression)

Overall survival was defined as the time from randomization into Part 2 of the trial until death from any cause

Part 1- Objective Response Rate (ORR)Approximately 28 months (assessed at baseline and every 9 weeks from randomization until disease progression)

ORR was defined as the number of participants with best overall response (BOR) of complete response (CR) or partial response (PR) recorded from the start of treatment, until the end of treatment. BOR documented as confirmed CR: disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to less than (\<)10 millimeter (mm). PR: At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters.

Part 2: Progression-free Survival (PFS) Assessed by the InvestigatorApproximately 44 months (assessed at screening and every 9 weeks from randomization until disease progression)

PFS (Investigator-assessed) is defined as the time from randomization, until disease progression according to RECIST v1.1 including death or MBO, whichever occurs first. Censoring is based on the last tumor assessment. If no tumor assessment post baseline, then censoring is at day 1. PD could base on symptom deterioration or was defined as at least a 20% increase in the sum of the longest diameter of target lesions, taking as reference the smallest sum longest diameter recorded since treatment started or the appearance of one or more new lesions and/or the unequivocal progression of existing non-target lesions.

Part 2- Objective Response Rate (ORR)Approximately 44 months (assessed at screening and every 9 weeks from randomization until disease progression)

ORR was defined as the number of participants with BOR of CR or PR recorded from the start of treatment, until the end of treatment. BOR documented as confirmed CR: disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to less than (\<)10 millimeter (mm). PR: At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters.

Part 1: PFS Assessed by the InvestigatorApproximately 28 months (assessed at screening and every 9 weeks from randomization until disease progression)

PFS as assessed by Investigator was defined as the time from first dose of pertuzumab or chemotherapy in Part 1 of the trial, until disease progression according to RECIST version 1.1, symptomatic deterioration or death from any cause, whichever occurs first. PD could base on symptom deterioration or was defined as at least a 20% increase in the sum of the longest diameter of target lesions, taking as reference the smallest sum longest diameter recorded since treatment started or the appearance of one or more new lesions and/or the unequivocal progression of existing non-target lesions. Participants were censored at the last tumor assessment. Participants who have no tumor assessments after baseline and who were still alive will be censored at 1 day.

Part 2: European Organization for Research and Treatment of Cancer (EORTC) Quality of Life (QoL) Questionnaire (QLQ) of Core 30 (C30) ScoreBaseline (assessed at baseline and every 9 weeks from randomization until disease progression)

EORTC QLQ-C30: included functional scales (physical, role, cognitive, emotional, and social), global health status, symptom scales (fatigue, pain, nausea/vomiting) and single items (dyspnoea, appetite loss, insomnia, constipation/diarrhea and financial difficulties). Most questions used 4-point scale (1 'Not at all' to 4 'Very much'; 2 questions used 7-point scale \[1 'very poor' to 7 'Excellent'\]). Scores averaged, transformed to 0-100 scale; for functional scores, a higher score represents a better level of functioning. For symptom scores, a higher score represents a more severe level of symptoms. For the global health status scores, a higher score represents a better quality of life.

Part 2: Percentage of Participants With Adverse Events (AEs)Approximately 28 months (assessed at screening, baseline until 28 days after the last dose of study treatment)

An AE can be any unfavorable and unintended sign (including an abnormality laboratory finding), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product.

Trial Locations

Locations (68)

Academ Ziekenhuis Groningen; Medical Oncology

🇳🇱

Groningen, Netherlands

Academisch Ziekenhuis Leiden; Clinical Oncology

🇳🇱

Leiden, Netherlands

UMC St Radboud

🇳🇱

Nijmegen, Netherlands

Herlev Hospital; Onkologisk afdeling

🇩🇰

Herlev, Denmark

Medizinische Universität Wien; Univ.Klinik für Frauenheilkunde - Klinik für Gynäkologie

🇦🇹

Wien, Austria

UZ Leuven Gasthuisberg

🇧🇪

Leuven, Belgium

Tiroler Landeskrankenanstalten Ges.M.B.H.; Abt. Für Gynäkologie

🇦🇹

Innsbruck, Austria

Centre Francois Baclesse; Oncologie

🇫🇷

Caen, France

Ico Rene Gauducheau; Oncologie

🇫🇷

Saint Herblain, France

Universitätsklinikum Essen; Zentrum Für Frauenheilkunde

🇩🇪

Essen, Germany

Universitätsklinikum Greifswald; Klinik für Frauenheilkunde und Brustzentrum

🇩🇪

Greifswald, Germany

Gynaekologisch-Onkologische Schwerpunktpraxis Prof. Dr. med. Lueck, Dr. Schrader und Dr. Noeding

🇩🇪

Hannover, Germany

Ente Ospedaliero Ospedali Galliera; S.C. Oncologia Medica

🇮🇹

Genova, Liguria, Italy

Centre Georges Francois Leclerc; Oncologie 3

🇫🇷

Dijon, France

UNI-Klinikum Campus Kiel Klinik f.Gynäkologie u.Geburtshilfe

🇩🇪

Kiel, Germany

Klinikum rechts der Isar der TU München; Frauenklinik & Poliklinik

🇩🇪

München, Germany

Hospital Son Llatzer; Servicio de Oncologia

🇪🇸

Palma de Mallorca, Islas Baleares, Spain

Centre Alexis Vautrin; Oncologie Medicale

🇫🇷

Vandoeuvre Les Nancy, France

Institut Gustave Roussy; Oncologie Medicale

🇫🇷

Villejuif, France

A.O.Spedali Civili; Ostetricia e Ginecologia

🇮🇹

Brescia, Lombardia, Italy

Hopital Tenon; Oncologie Radiotherapie

🇫🇷

Paris, France

Institut Bergonie; Oncologie

🇫🇷

Bordeaux, France

St. Elisabeth Krankenhaus Köln GmbH; Gynäkologie und Geburtshilfe

🇩🇪

Koeln, Germany

Klinikum Konstanz, Frauenklinik

🇩🇪

Konstanz, Germany

The Norvegian Radium Hospital Montebello; Dept of Oncology

🇳🇴

Oslo, Norway

Corporacio Sanitaria Parc Tauli; Servicio de Oncologia

🇪🇸

Sabadell, Barcelona, Spain

Ch Lyon Sud; Chir Onc Gyne Sct Jules Courmont

🇫🇷

Pierre Benite, France

Clinique Armoricaine Radiologie; Hopital de Jour

🇫🇷

Plerin, France

Kliniken Essen-Mitte Evang. Huyssens-Stiftung, Klinik für Gynäkologie und gynäkologische Onkologie

🇩🇪

Essen, Germany

Universitätsklinikum Freiburg; Frauenklinik

🇩🇪

Freiburg, Germany

Sana Klinikum Offenbach GmbH; Klinik für Gynäkologie & Geburtshilfe

🇩🇪

Offenbach, Germany

Hämatologisch/Onkologische Praxis Dr. Herbrick - Zipp/Prof. Dr. Decker, Studienzentrum

🇩🇪

Ravensburg, Germany

Universitätsfrauen- und Poliklinik am Klinikum Suedstadt

🇩🇪

Rostock, Germany

A.O.U Pisana; Dipartimento di Ginecologia Oncologica

🇮🇹

Pisa, Toscana, Italy

Antoni van Leeuwenhoek Ziekenhuis

🇳🇱

Amsterdam, Netherlands

Rigshospitalet, Onkologisk Klinik

🇩🇰

København Ø, Denmark

CRLCC Val dAurelle Paul Lam

🇫🇷

Montpellier cedex 5, France

Universitätsklinikum "Carl Gustav Carus"; Frauenheilkunde und Geburtshilfe

🇩🇪

Dresden, Germany

Evangelischen Krankenhauses Düsseldorf; Frauenklinik

🇩🇪

Düsseldorf, Germany

Universitätsklinikum Hamburg-Eppendorf (UKE); Klinik und Poliklinik für Gynäkologie

🇩🇪

Hamburg, Germany

Nationales Centrum für Tumorerkrankungen (NCT) ; Gyn. Onk. Frauenklinik; Uniklinikum Heidelberg

🇩🇪

Heidelberg, Germany

Universitätsklinik Tübingen; Frauenklinik

🇩🇪

Tübingen, Germany

HELIOS Dr. Horst Schmidt Kliniken Wiesbaden; Klinik für Gynäkologie und gynäkologische Onkologie

🇩🇪

Wiesbaden, Germany

Universitätsklinikum Ulm Am Michelsberg; Frauenklinik

🇩🇪

Ulm, Germany

Istituto Regina Elena; Oncologia Medica A

🇮🇹

Roma, Lazio, Italy

Istituto Tumori Napoli;Unità Operativa Oncologia Medica Uro-Ginecologica

🇮🇹

Napoli, Campania, Italy

Istituto Nazionale dei Tumori; Divisione Oncologia Chirurgica e Ginecologica

🇮🇹

Milano, Lombardia, Italy

Istituto Europeo Di Oncologia

🇮🇹

Milano, Lombardia, Italy

Hospital Clínic i Provincial; Servicio de Hematología y Oncología

🇪🇸

Barcelona, Spain

Hospital Univ Vall d'Hebron; Servicio de Oncologia

🇪🇸

Barcelona, Spain

Hospital Duran i Reynals; Oncologia

🇪🇸

Barcelona, Spain

Hospital de la Santa Creu i Sant Pau; Servicio de Oncologia

🇪🇸

Barcelona, Spain

Hospital Universitari Germans Trias i Pujol; Servicio de Oncologia

🇪🇸

Barcelona, Spain

Hospital Universitario Reina Sofia; Servicio de Oncologia

🇪🇸

Cordoba, Spain

Hospital Universitari Arnau de Vilanova de Lleida; Servicio de Oncologia

🇪🇸

Lerida, Spain

Hospital Universitari de Girona Dr. Josep Trueta; Servicio de Oncologia

🇪🇸

Girona, Spain

Hospital General Universitario Gregorio Marañon; Servicio de Oncologia

🇪🇸

Madrid, Spain

Hospital Regional Universitario Carlos Haya; Servicio de Oncologia

🇪🇸

Malaga, Spain

Hospital Universitario Virgen de Arrixaca; Servicio de Oncologia

🇪🇸

Murcia, Spain

Instituto Valenciano Oncologia; Oncologia Medica

🇪🇸

Valencia, Spain

Hospital Universitario la Fe; Servicio de Oncologia

🇪🇸

Valencia, Spain

Skånes University Hospital, Skånes Department of Onclology

🇸🇪

Lund, Sweden

Universitetssjukhuset; Onkologkliniken

🇸🇪

Linkoeping, Sweden

Hospital Universitario Miguel Servet; Servicio Oncologia

🇪🇸

Zaragoza, Spain

Centro Oncologico MD Anderson Internacional; Servicio de Oncologia

🇪🇸

Madrid, Spain

Hospital Universitario 12 de Octubre; Servicio de Oncologia

🇪🇸

Madrid, Spain

Hospital Universitario La Paz; Servicio de Oncologia

🇪🇸

Madrid, Spain

Centro Integral Oncologico Clara Campal (CIOCC); Dirección Médica

🇪🇸

Madrid, Spain

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