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Safety And Efficacy Study Of Palbociclib Plus Cetuximab Versus Cetuximab To Treat Head And Neck Cancer

Phase 2
Completed
Conditions
Squamous Cell Carcinoma of the Head and Neck (SCCHN)
Interventions
Registration Number
NCT02499120
Lead Sponsor
Pfizer
Brief Summary

The purpose of this study is to determine whether the combination of palbociclib with cetuximab is superior to cetuximab in prolonging overall survival in HPV-negative, cetuximab-naive patients with recurrent/metastatic squamous cell carcinoma of the head and neck.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
125
Inclusion Criteria
  • Histologically or cytologically confirmed squamous cell carcinoma of the oral cavity, oropharynx, hypopharynx or larynx, not amenable for salvage surgery or radiotherapy.
  • Measurable disease as defined per RECIST v. 1.1. Tumor lesions previously irradiated or subjected to other locoregional therapy will only be deemed measureable if disease progression at the treated site after completion of therapy is clearly documented.
  • HPV- negative SCCHN tumor as determined per institutional standard (eg, p16 IHC; multiplex nucleic acid sequence based amplification [NASBA] or other polymerase chain reaction [PCR]-based assays).
  • Documented progressive disease according to RECIST v1.1 (Appendix 2) following receipt of at least 2 cycles of one platinum-containing chemotherapy regimen administered for R/M disease (min. 50 mg/m2 for cisplatin, minimum area under the curve [AUC] > 4 for carboplatin).
  • Availability of a tumor tissue specimen (ie, archived formalin fixed paraffin embedded tissue [block preferred, or 15 unstained slides]), which will be used for centralized, retrospective biomarker analysis. If archived tumor tissue is not available, then a de novo biopsy will be required for patient participation.

Key

Exclusion Criteria
  • Prior nasopharyngeal cancer, salivary gland or sinus tumors.
  • More than one chemotherapeutic regimen given for R/M disease. Prior treatment with immunotherapy is allowed.
  • Known active uncontrolled or symptomatic central nervous system (CNS) metastases, carcinomatous meningitis, or leptomeningeal disease as indicated by clinical symptoms, cerebral edema, 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 (eg, radiotherapy, stereotactic surgery) and are clinically stable off anticonvulsants and steroids for at least 4 weeks before randomization.
  • Progressive disease within 3 months after completion of curatively intended treatment for locoregionally advanced SCCHN.
  • Difficulty swallowing capsules.
  • Prior use of cetuximab in the R/M disease treatment setting (except cetuximab during curative radiotherapy)

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Placebo plus CetuximabPlaceboPlacebo orally QD with food on Day 1 to Day 21 followed by 7 days off treatment in a 28-day cycle; in combination with Cetuximab, 400 mg/m2 initial dose as a 120-minute IV infusion followed by 250 mg/m2 weekly infused over 60 minutes.
Placebo plus CetuximabCetuximabPlacebo orally QD with food on Day 1 to Day 21 followed by 7 days off treatment in a 28-day cycle; in combination with Cetuximab, 400 mg/m2 initial dose as a 120-minute IV infusion followed by 250 mg/m2 weekly infused over 60 minutes.
Palbociclib plus CetuximabpalbociclibPalbociclib, 125 mg, orally once daily (QD) with food on Day 1 to Day 21 followed by 7 days off treatment in a 28-day cycle; in combination with Cetuximab, 400 mg/m2 initial dose as a 120-minute IV infusion followed by 250 mg/m2 weekly infused over 60 minutes.
Palbociclib plus CetuximabCetuximabPalbociclib, 125 mg, orally once daily (QD) with food on Day 1 to Day 21 followed by 7 days off treatment in a 28-day cycle; in combination with Cetuximab, 400 mg/m2 initial dose as a 120-minute IV infusion followed by 250 mg/m2 weekly infused over 60 minutes.
Primary Outcome Measures
NameTimeMethod
Overall Survival (OS)Baseline up to primary completion date (PCD) (about 34 months)

OS was defined as the time from the date of randomization to the date of death due to any cause. OS (in months) was calculated as (date of death - randomization date +1)/30.4. For participants lacking survival data beyond the date of their last follow-up, the OS time was censored on the last date they were known to be alive. Participants lacking survival data beyond randomization had their OS times be censored at randomization. Estimates of OS and its 95% confidence interval were determined using Kaplan-Meier method.

Secondary Outcome Measures
NameTimeMethod
Percentage of Participants With Objective Response (OR)Baseline up to PCD (about 34 months)

OR was defined as the overall complete response (CR) or partial response (PR) according to the Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1. Objective response rate was defined as the proportion of participants with best overall response (BOR) of CR or PR relative to all randomized.

Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-C30 (EORTC QLQ-C30)Baseline up to PCD (about 34 months)

The EORTC QLQ-C30 is a 30 item questionnaire composed of 5 multi-item functional subscales (physical, role, cognitive, emotional, and social functioning), 3 multi-item symptom scales (fatigue, nausea/vomiting, and pain), a global health/quality of life (QOL) subscale, and 6 single items assessing other cancer related symptoms (dyspnea, sleep disturbance, appetite, diarrhea, constipation, and the financial impact of cancer). The questionnaire employed twenty-eight 4 point Likert scales with responses from "not at all" to "very much" and two 7 point Likert scales for global health and overall QOL. For functional and global QOL scales, higher scores represented a better level of functioning and all scores were converted to a 0 to 100 scale. For symptom oriented scales, a higher score represented more severe symptoms, and all scores were converted to a 0-100 scale. Negative changes from baseline indicate deterioration in functioning / global QoL scales and improvement in symptom scales.

Ctrough and Cendinf, WPM-Ctrough and WPM-Cendinf at Steady State for Serum CetuximabPre-dose and end-of infusion of Day 15 in Cycle 1 and Cycle 2

Ctrough is steady-state pre-dose concentration. Cendinf is steady-state end-of-infusion concentration. Ctrough and Cendinf were observed directly from data. WPM-Ctrough and WPM-Cendinf are within-participant mean steady-state pre-dose concentration and end-of-infusion concentration. Acceptance criteria for a steady-state Cendinf was defined as a PK sample that was 1) collected after at least 3 consecutive weeks of cetuximab IV infusions without interruption or prior dose reduction and 2) was collected at the end of cetuximab infusion time +/- 10% of the actual duration of the cetuximab infusion.

Number of Participants With Treatment-Emergent Adverse Events(TEAEs)From the first dose through and including 28 calendar days after the last administration of the study treatment (up to 6.9 years)

AE was defined as any untoward medical occurrence in a clinical investigation participant administered a product or medical device, regardless of the causal relationship to study treatment. TEAEs were defined as AEs which occurred for the first time during the effective duration of treatment or AEs that increased in severity during treatment. Serious AEs (SAEs) were defined as any untoward medical occurrence at any dose that resulted in death; was life-threatening (immediate risk of death); required inpatient hospitalization or caused prolongation of existing hospitalization; resulted in persistent or significant disability/incapacity (substantial disruption of the ability to conduction normal life functions). AEs included SAEs and non-serious AEs. Causality to study treatment was determined by the investigator. Severity was graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.03.

Summary of PFS and OS for P16 Negative (%Positive Tumor Cells < 70%)Screening

A central test was defined as the tumor tissue-based p16 IHC test performed at a central laboratory (Ventana). The analysis of concordance between HPV status as assessed by local or central laboratory included the number and percentage of participants with p16 detected or not detected at the central laboratory, given that all local testing must have been negative for HPV in order for the patient to be eligible for the study. Initial analysis of the p16 status was based on the conventional cutoff of 70% p16-positive tumor cells to call out cases that might be considered HPV-positive. P16 expression was scored as positive if strong and diffuse nuclear and cytoplasmic staining was present in at least 70% of the tumor cells.

Percentage of Participants With Clinical Benefit Response (CBR)Baseline up to PCD (about 34 months)

CBR was defined as the overall CR, PR, or stable disease\>=24 weeks according to the RECIST version 1.1. Clinical benefit response rate was defined as the proportion of participants with CR, PR, or stable disease\>= 24 weeks relative to all randomized participants and randomized participants with measurable disease at baseline.

Duration of Response (DR)Baseline up to PCD (about 34 months)

DR was defined as the time from the first documentation of objective tumor response (CR or PR) to the first documentation of disease progression or to death due to any cause, whichever occurred first. If tumor progression data included more than 1 date, the first date was used. DR was calculated as \[the date response ended (ie, date of PD or death) - first CR or PR date + 1\]/30.4.

Number of Participants With Laboratory AbnormalitiesFrom the Screening (Day -28) through and including 28 calendar days after the last administration of the study treatment (up to 7 years)

The hematology, chemistry and coagulation tests were included in the laboratory examination. Hematology evaluation included hemoglobin, platelets, white blood cell, absolute neutrophils, absolute lymphocytes. Chemistry evaluation included alanine aminotransferase (ALT), aspartate aminotransferase (AST), alkaline phosphatase, sodium, potassium, magnesium, chloride, total calcium, total bilirubin, blood urea nitrogen (BUN) or urea, creatinine, uric acid, glucose (non-fasted), albumin, phosphorus or phosphate and hemoglobin A1c (HbA1c). Coagulation evaluation included activated partial thromboplastin time/partial thromboplastin time, international normalized ratio (INR) or prothrombin time.

Trough Plasma Concentration (Ctrough) and Within-participant Mean Steady-state Pre-dose Concentration (WPM-Ctrough) at Steady State for PalbociblibPre-dose of Day 15 in Cycle 1 and Cycle 2

Ctrough is steady-state pre-dose concentration, which was observed directly from data. WPM-Ctrough is within-participant mean steady-state pre-dose concentration. For palbociclib, a steady-state trough was to be defined as a pre-dose plasma concentration following at least 7 consecutive days of 125 mg daily dose without dosing interruption and the time window for the PK collection was to be between 24 hr +/- 2 hr and 24 min post-dose the day prior to PK collection and no more than 1 hr post-dose on the day of PK collection.

Progression Free Survival (PFS)Baseline up to PCD (about 34 months)

PFS was defined as the time from the date of randomization to the date of the first documentation of objective progression of disease (PD) or death due to any cause, whichever was earlier. Estimates of the PFS curves from the Kaplan Meier method were presented.

Change From Baseline in European Organization for Research and Treatment of Cancer Head and Neck Module35 (EORTC QLQ-H&N35)Baseline up to PCD (about 34 months)

The EORTC QLQ-H\&N35 is designed to be used together with the core QLQ-C30. The recall period for the items in the module was "the past week". Items hn1 to hn30 were scored on 4 point Likert type categorical scales ("not at all", "a little", "quite a bit", "very much"). Items hn31 to hn35 had a "no/yes" response format. The scores were transformed into 0 to 100 scales, with a high score implying a high level of symptoms.Negative changes from baseline indicate deterioration in functioning / global QoL scales and improvement in symptom scales.

Summary of PFS and OS Based on Investigator Assessment by Rb Expression >= 1%Screening

Rb expression in the palbociclib and cetuximab treatment group, the relationship of the biomarker (individually) with PFS and OS were explored using graphical methods such as box plots, at baseline. The tumors of participants were Rb-positive, which was defined by Rb IHC with\>=1% positive tumor cells.

Trial Locations

Locations (71)

Shizuoka Cancer Center

🇯🇵

Sunto-gun, Shizuoka, Japan

Henry Joyce Cancer Clinic

🇺🇸

Nashville, Tennessee, United States

Aichi cancer center Central hospital

🇯🇵

Nagoya, Aichi, Japan

Oaxaca Site Management Organization S C

🇲🇽

Oaxaca, Mexico

Uniwersyteckie Centrum Kliniczne Klinika Onkologii i Radioterapii

🇵🇱

Gdansk, Poland

State Budgetary Healthcare Institution of Arkhangelsk Region

🇷🇺

Arkhangelsk, Arkhangelsk Region, Russian Federation

Szpital Specjalistyczny w Brzozowie Podkarpacki Osrodek Onkologiczny im. ks. B. Markiewicza

🇵🇱

Brzozow, Poland

State Autonomous Healthcare Institution Republican Clinical Oncology Dispensary of the Ministry

🇷🇺

Kazan, Tatarstan Republic, Russian Federation

CI Dnipropetrovsk City Multifunctional Clinical Hospital #4 of Dnipropetrovsk Regional Council

🇺🇦

Dnipropetrovsk, Ukraine

Debreceni Egyetem klinikai Koezpont Onkologiai Intezet

🇭🇺

Debrecen, Hungary

S.C. ONCOCENTER Oncologie Clinica S.R.L.

🇷🇴

Timisoara, Timis, Romania

Regional Clinical Hospital, Department of microsurgery of otolaryngology organs

🇺🇦

Ivano-Frankivsk, Ukraine

Mayo Clinic

🇺🇸

Rochester, Minnesota, United States

Hokkaido University Hospital/Otolaryngology

🇯🇵

Sapporo, Hokkaido, Japan

Institute for Oncology and Radiology of Serbia

🇷🇸

Belgrade, Serbia

POKO Poprad, s.r.o.

🇸🇰

Poprad, Slovakia

China Medical University Hospital

🇨🇳

Taichung, Taiwan

Communal Institution "Krivorizhskiy Oncology Dispensary" of Dnipropetrovsk Regional Council,

🇺🇦

Kriviy Rig, Ukraine

Podilskiy Regional Center of Oncology, Chemotherapy Department

🇺🇦

Vinnytsia, Ukraine

Cirugia y Ginecobstetricia de Oaxaca S.A de C.V Hospital Reforma

🇲🇽

Oaxaca, Oaxaca DE Juarez, Mexico

SC Oncolab SRL

🇷🇴

Craiova, Dolj, Romania

Taichung Veterans General Hospital

🇨🇳

Taichung City, Taiwan

Communal Institution of Kherson Regional Council Kherson Regional Oncological Dispensary

🇺🇦

Antonivka, Kherson Region, Ukraine

Tri-Service General Hospital

🇨🇳

Taipei, Taiwan

UC San Diego Medical Center - La Jolla (Thornton Hospital)

🇺🇸

La Jolla, California, United States

UC San Diego Medical Center- Hillcrest

🇺🇸

San Diego, California, United States

UC San Diego Moores Cancer Center

🇺🇸

La Jolla, California, United States

University Medical Center, lnc.:DBA University of Louisville Hospital

🇺🇸

Louisville, Kentucky, United States

Siteman Cancer Center - West County

🇺🇸

Creve Coeur, Missouri, United States

Barnes-Jewish Hospital

🇺🇸

Saint Louis, Missouri, United States

Washington University School of Medicine, Siteman Cancer Center

🇺🇸

Saint Louis, Missouri, United States

Siteman Cancer Center - South County

🇺🇸

Saint Louis, Missouri, United States

Siteman Cancer Center

🇺🇸

Saint Peters, Missouri, United States

University of Cincinnati Investigational Pharmacy

🇺🇸

Cincinnati, Ohio, United States

University of Cincinnati Medical Center

🇺🇸

Cincinnati, Ohio, United States

UC Health Physicians Office South

🇺🇸

West Chester, Ohio, United States

Nemocnice Na Bulovce, Centralni laboratore Pavilon c. 8

🇨🇿

Praha 8, Czechia

Fakultni nemocnice Olomouc

🇨🇿

Olomouc, Czechia

Fakultni nemocnice Olomouc, Lekarna

🇨🇿

Olomouc, Czechia

Nemocnice Na Bulovce, Lekarna, Oddeleni Centralni pripravy

🇨🇿

Praha 8, Czechia

Nemocnice Na Bulovce, Ustav radiacni onkologie

🇨🇿

Praha 8, Czechia

Pecsi Tudomanyegyetem, Klinikai Kozpont, Laboratoriumi

🇭🇺

Pecs, Hungary

Neuro CT Kft

🇭🇺

Pecs, Hungary

Pecsi Tudomanyegyetem, Klinikai Kozpont,

🇭🇺

Pecs, Hungary

Jasz-Nagykun-Szolnok Megyei Hetenyi Geza Korhaz-Rendelointezet,Onkologiai Kozpont

🇭🇺

Szolnok, Hungary

Istituto Nazionale Tumori Napoli

🇮🇹

Napoli, Italy

National Cancer Center Hospital East

🇯🇵

Kashiwa, Chiba, Japan

Seoul National University Hospital

🇰🇷

Seoul, Korea, Republic of

Samsung Medical Center

🇰🇷

Seoul, Korea, Republic of

Hospital Universitario Dr. Jose Eleuterio Gonzalez

🇲🇽

Monterrey, Nuevo LEON, Mexico

Instituto Nacional de Cancerologia

🇲🇽

Mexico, Distrito Federal, Mexico

Diaz San Juan Noe, Imagenologia Siglo XXI San Felipe

🇲🇽

Oaxaca, Oaxaca DE Juarez, Mexico

Daniel Javier Mendez Lopez Imagen y Diagnostico Medico IDM

🇲🇽

Oaxaca, Oaxaca DE Juarez, Mexico

Wojewodzki Szpital Specjalistyczny im. M. Kopernika w Lodzi Oddzial Chemioterapii

🇵🇱

Lodz, Poland

SPZOZ MSWiA z Warminsko-Mazurskim Centrum Onkologii w Olsztynie Oddzial Kliniczny Onkologii

🇵🇱

Olsztyn, Poland

SC Medisprof SRL

🇷🇴

Cluj-Napoca, Cluj, Romania

Centrul de Oncologie Sf. Nectarie SRL

🇷🇴

Craiova, Dolj, Romania

Spitalul Clinic Judetean de Urgenta Sibiu, Clinica Oncologie Medicala

🇷🇴

Sibiu, Romania

State Budgetary Healthcare Institution "Oncology Center #2" of the Ministry of

🇷🇺

Sochi, Krasnodar Region, Russian Federation

FSBI "National Medical Scientific Centre of Oncology n.a.N.N.Petrov" of the MOH of Russia

🇷🇺

Saint-Petersburg, Russian Federation

Military Medical Academy

🇷🇸

Belgrade, Serbia

Narodny onkologicky ustav

🇸🇰

Bratislava, Slovakia

Servicio de Oncologia

🇪🇸

Pamplona, Navarra, Spain

Hospital Universitario Vall d'Hebron

🇪🇸

Barcelona, Spain

Hospital Universitario 12 de Octubre

🇪🇸

Madrid, Spain

National Cheng Kung University Hospital

🇨🇳

Tainan, Taiwan

National Cheng Kung University Hospital Department of Pathology

🇨🇳

Tainan, Taiwan

SI Dnipropetrovsk Medical Academy of MoH of Ukraine, Chair of Oncology and Medical Radiology

🇺🇦

Dnipropetrovsk, Ukraine

Communal Institution "Chernivtsi Regional clinical oncology dispensary",

🇺🇦

Chernivtsy, Ukraine

Clinic of SI "Institute of Otolaryngology n.a. Prof. O.S. Kolomyichenka of NAMSU"

🇺🇦

Kyiv, Ukraine

Asan Medical Center

🇰🇷

Seoul, Korea, Republic of

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