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Combination Chemotherapy With or Without Erlotinib Hydrochloride in Treating Patients With Metastatic or Recurrent Squamous Cell Carcinoma of the Head and Neck

A Randomized, Placebo-Controlled, Phase 2 Study of Docetaxel and Cisplatin/Carboplatin With or Without Erlotinib in Patients With Metastatic or Recurrent Squamous Cell Carcinoma of the Head and Neck

Status
Completed
Phases
Phase 2
Study type
Interventional
Source
ClinicalTrials.gov
Registry ID
NCT01064479
Enrollment
123
Registered
2010-02-08
Start date
2010-02-05
Completion date
2020-11-03
Last updated
2024-07-16

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Metastatic Squamous Cell Carcinoma of the Hypopharynx, Metastatic Squamous Cell Carcinoma of the Larynx, Metastatic Squamous Cell Carcinoma of the Oral Cavity, Metastatic Squamous Cell Carcinoma of the Oropharynx, Recurrent Hypopharyngeal Squamous Cell Carcinoma, Recurrent Laryngeal Squamous Cell Carcinoma, Recurrent Oral Cavity Squamous Cell Carcinoma, Recurrent Oropharyngeal Squamous Cell Carcinoma, Stage IVA Hypopharyngeal Squamous Cell Carcinoma AJCC v7, Stage IVA Laryngeal Squamous Cell Carcinoma AJCC v7, Stage IVA Oral Cavity Squamous Cell Carcinoma AJCC v6 and v7, Stage IVA Oropharyngeal Squamous Cell Carcinoma AJCC v7, Stage IVB Hypopharyngeal Squamous Cell Carcinoma AJCC v7, Stage IVB Laryngeal Squamous Cell Carcinoma AJCC v7, Stage IVB Oral Cavity Squamous Cell Carcinoma AJCC v6 and v7, Stage IVB Oropharyngeal Squamous Cell Carcinoma AJCC v7, Stage IVC Hypopharyngeal Squamous Cell Carcinoma AJCC v7, Stage IVC Laryngeal Squamous Cell Carcinoma AJCC v7, Stage IVC Oral Cavity Squamous Cell Carcinoma AJCC v6 and v7, Stage IVC Oropharyngeal Squamous Cell Carcinoma AJCC v7, Stage IV Hypopharyngeal Squamous Cell Carcinoma AJCC v7, Stage IV Laryngeal Squamous Cell Carcinoma AJCC v7, Stage IV Oral Cavity Squamous Cell Carcinoma AJCC v6 and v7, Stage IV Oropharyngeal Squamous Cell Carcinoma AJCC v7

Brief summary

This randomized phase II trial studies how well combination chemotherapy with or without erlotinib hydrochloride works in treating patients with squamous cell carcinoma of the head and neck that has spread to other parts of the body or has come back. Drugs used in chemotherapy, such as docetaxel, cisplatin, and carboplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Erlotinib hydrochloride may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving combination chemotherapy with or without erlotinib hydrochloride may be an effective treatment for squamous cell carcinoma of the head and neck.

Detailed description

PRIMARY OBJECTIVES: I. Assess the efficacy of adding erlotinib hydrochloride (erlotinib) to chemotherapy to improve progression free survival in patients with metastatic or recurrent squamous cell carcinoma of the head and neck. SECONDARY OBJECTIVES: I. Evaluate overall survival, response rate, disease control rate, and duration of response by treatment with or without erlotinib. II. Evaluate quality of life (patient reported outcomes) by treatment with or without erlotinib. III. Evaluate the safety profile of erlotinib in combination with chemotherapy. IV. Correlate the occurrence of erlotinib-induced rash with outcomes. V. To evaluate the steady-state pharmacokinetics of erlotinib. VI. To explore the prognostic and predictive value of epidermal growth factor receptor related biomarkers and other biomarkers, including blood and tissue proteomic and blood and tissue genomic markers, that may be associated with clinical outcomes. OUTLINE: Patients are randomized to 1 of 2 arms. ARM A: Patients receive docetaxel intravenously (IV) over 1 hour and cisplatin IV over 2 hours or carboplatin IV over 2 hours on day 1, and erlotinib hydrochloride orally (PO) daily on days 1-21. Treatment repeats every 21 days for up to 6 courses in the absence of disease progression and unacceptable toxicity. Patients achieving complete response, partial response, or stable disease may continue erlotinib hydrochloride treatment. ARM B: Patients receive docetaxel and cisplatin or carboplatin as in Arm I and placebo PO daily on days 1-21. Treatment repeats every 21 days for up to 6 courses in the absence of disease progression and unacceptable toxicity. Patients achieving complete response, partial response, or stable disease may continue placebo treatment. After completion of study treatment, patients are followed up at 30 days.

Interventions

DRUGCarboplatin

Given IV

DRUGCisplatin

Given IV

DRUGDocetaxel

Given IV

DRUGErlotinib Hydrochloride

Given PO

OTHERLaboratory Biomarker Analysis

Optional correlative studies

OTHERPharmacological Study

Optional correlative studies

OTHERPlacebo

Given PO

OTHERQuality-of-Life Assessment

Ancillary studies

Sponsors

National Cancer Institute (NCI)
CollaboratorNIH
M.D. Anderson Cancer Center
Lead SponsorOTHER

Study design

Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)

Eligibility

Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No

Inclusion criteria

* Histologically confirmed metastatic or recurrent squamous cell carcinoma of the head and neck (SCCHN) of the oral cavity, oropharynx, hypopharynx or larynx; metastatic or recurrent lesions of the nasopharynx and sinus are excluded * Radiologically measurable disease, defined as at least one lesion that can be accurately measured in at least one dimension (longest diameter to be recorded) as \>= 20 mm with conventional techniques or as \>= 10 mm with spiral computed tomography (CT) scan; measurable lymph nodes are required to be \>= 15 mm in size (short axis diameter) * Eastern Cooperative Oncology Group (ECOG) performance status (PS) =\< 2 * Absolute neutrophil count (ANC) \>= 1.5 x 10\^9/L * Platelet count \>= 100 x 10\^9/L * Total bilirubin =\< upper limit of normal (ULN) (excluding Gilbert's disease) * Alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase \[SGPT\]) =\< 1.5 x ULN * Alkaline phosphatase =\< 2.5 x ULN * Serum creatinine =\< 1.5 x ULN * Patients with reproductive potential (e.g., females menopausal for less than 1 year and not surgically sterilized) must practice effective contraceptive measures for the duration of study drug therapy and for at least 30 days after completion of study drug therapy; female patients of childbearing potential must provide a negative pregnancy test (serum or urine) =\< 14 days prior to treatment initiation * Written informed consent to participate in the study according to the investigational review board (IRB) or independent ethics committee (IEC)

Exclusion criteria

* Histology other than squamous cell carcinoma * Primary sites other than oral cavity, oropharynx, hypopharynx, and larynx * Prior palliative chemotherapy for metastatic or recurrent disease * Prior biological therapy for metastatic or recurrent disease within 3 weeks prior to randomization * Patients with known, untreated brain metastases; patients with treated (irradiated or resected) brain metastases are eligible if treatment was completed more than 28 days prior to study entry and if clinical neurologic function is stable * Pre-existing peripheral neuropathy \>= grade 2 * History of poorly controlled gastrointestinal disorders that could affect the absorption of the study drug (e.g., Crohn's disease, ulcerative colitis); patients requiring feeding tubes are permitted * Other active malignancies requiring chemotherapy treatment within 2 years prior to randomization, except for adequately treated basal cell or squamous cell skin cancer or in situ cervical or breast cancer or superficial, resected melanoma * Serious underlying medical condition which would impair the ability of the patient to receive protocol treatment, in the opinion of the treating physician * History of allergic reactions to compounds of similar chemical composition to the study drugs (docetaxel, cisplatin, carboplatin, erlotinib or their excipients), or other drugs formulated with polysorbate 80 * Any concurrent anti-cancer therapy, excluding hormonal therapy for prostate or breast cancer * Dementia or significantly altered mental status that would prohibit the understanding and giving of informed consent * Women who are pregnant or breast-feeding and women or men not practicing effective birth control

Design outcomes

Primary

MeasureTime frameDescription
Progression Free Survival (PFS)5 yearsKaplan-Meier methods will be used to summarize PFS. In the primary analysis, differences in PFS in Arm A versus Arm B will be tested using a stratified log-rank test with a two-sided alpha of 0.10. Hazard ratios for PFS will be presented using point estimates and 95% confidence intervals.

Secondary

MeasureTime frameDescription
Overall Survival (OS)5 yearsKaplan-Meier methods will be used to summarize OS. Hazard ratios for OS will be presented using point estimates and 95% confidence intervals.
Number of Participants With Tumor Response (Complete Response [CR] + Partial Response [PR])5 yearsPer Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions
Disease Control (CR + PR + Stable Disease [SD])5 yearsComplete Response (CR) + Partial Response (PR) + Stable disease
Rash Rates5 yearsParticipants with a Rash of at least grade 2 within cycle 1.

Countries

United States

Participant flow

Recruitment details

Patients with histologically confirmed metastatic or recurrent SCCHN of the oral cavity, oropharynx, hypopharynx or larynx who have an ECOG performance status of 0-2, measurable disease and no prior chemotherapy for their metastatic or recurrent disease were enrolled

Pre-assignment details

123 participants registered-4 participants were inevaluable (2 refused not due to toxicity, 2 other).

Participants by arm

ArmCount
Arm A (Combination Chemotherapy and Erlotinib Hydrochloride)
Patients receive docetaxel IV over 1 hour and cisplatin IV over 2 hours or carboplatin IV over 2 hours on day 1 and erlotinib hydrochloride PO daily on days 1-21. Treatment repeats every 21 days for up to 6 courses in the absence of disease progression and unacceptable toxicity. Patients achieving complete response, partial response, or stable disease may continue erlotinib hydrochloride treatment.
60
Arm B (Combination Chemotherapy and Placebo)
Patients receive docetaxel and cisplatin or carboplatin as in Arm I and placebo PO daily on days 1-21. Treatment repeats every 21 days for up to 6 courses in the absence of disease progression and unacceptable toxicity. Patients achieving complete response, partial response, or stable disease may continue placebo treatment.
59
Total119

Withdrawals & dropouts

PeriodReasonFG000FG001
Overall StudyDeath1011
Overall StudyDisease progression11
Overall StudyOther12
Overall StudyToxicity11
Overall StudyWithdrawal by Subject22

Baseline characteristics

CharacteristicArm A (Combination Chemotherapy and Erlotinib Hydrochloride)Arm B (Combination Chemotherapy and Placebo)Total
Age, Categorical
<=18 years
0 Participants0 Participants0 Participants
Age, Categorical
>=65 years
8 Participants8 Participants16 Participants
Age, Categorical
Between 18 and 65 years
52 Participants51 Participants103 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants0 Participants0 Participants
Race (NIH/OMB)
Asian
2 Participants1 Participants3 Participants
Race (NIH/OMB)
Black or African American
2 Participants4 Participants6 Participants
Race (NIH/OMB)
More than one race
0 Participants0 Participants0 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants0 Participants0 Participants
Race (NIH/OMB)
Unknown or Not Reported
5 Participants5 Participants10 Participants
Race (NIH/OMB)
White
51 Participants49 Participants100 Participants
Region of Enrollment
United States
60 participants59 participants119 participants
Sex: Female, Male
Female
13 Participants14 Participants27 Participants
Sex: Female, Male
Male
47 Participants45 Participants92 Participants

Adverse events

Event typeEG000
affected / at risk
EG001
affected / at risk
deaths
Total, all-cause mortality
37 / 6053 / 59
other
Total, other adverse events
56 / 6055 / 59
serious
Total, serious adverse events
21 / 6046 / 59

Outcome results

Primary

Progression Free Survival (PFS)

Kaplan-Meier methods will be used to summarize PFS. In the primary analysis, differences in PFS in Arm A versus Arm B will be tested using a stratified log-rank test with a two-sided alpha of 0.10. Hazard ratios for PFS will be presented using point estimates and 95% confidence intervals.

Time frame: 5 years

ArmMeasureValue (MEDIAN)
Arm A (Combination Chemotherapy and Erlotinib Hydrochloride)Progression Free Survival (PFS)6.08 months
Arm B (Combination Chemotherapy and Placebo)Progression Free Survival (PFS)4.4 months
Secondary

Disease Control (CR + PR + Stable Disease [SD])

Complete Response (CR) + Partial Response (PR) + Stable disease

Time frame: 5 years

ArmMeasureValue (COUNT_OF_PARTICIPANTS)
Arm A (Combination Chemotherapy and Erlotinib Hydrochloride)Disease Control (CR + PR + Stable Disease [SD])47 Participants
Arm B (Combination Chemotherapy and Placebo)Disease Control (CR + PR + Stable Disease [SD])41 Participants
Secondary

Number of Participants With Tumor Response (Complete Response [CR] + Partial Response [PR])

Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions

Time frame: 5 years

ArmMeasureGroupValue (COUNT_OF_PARTICIPANTS)
Arm A (Combination Chemotherapy and Erlotinib Hydrochloride)Number of Participants With Tumor Response (Complete Response [CR] + Partial Response [PR])Partial Response27 Participants
Arm A (Combination Chemotherapy and Erlotinib Hydrochloride)Number of Participants With Tumor Response (Complete Response [CR] + Partial Response [PR])Stable Disease16 Participants
Arm A (Combination Chemotherapy and Erlotinib Hydrochloride)Number of Participants With Tumor Response (Complete Response [CR] + Partial Response [PR])Progressive Disease8 Participants
Arm A (Combination Chemotherapy and Erlotinib Hydrochloride)Number of Participants With Tumor Response (Complete Response [CR] + Partial Response [PR])Inevaluable5 Participants
Arm A (Combination Chemotherapy and Erlotinib Hydrochloride)Number of Participants With Tumor Response (Complete Response [CR] + Partial Response [PR])Complete Response4 Participants
Arm B (Combination Chemotherapy and Placebo)Number of Participants With Tumor Response (Complete Response [CR] + Partial Response [PR])Inevaluable5 Participants
Arm B (Combination Chemotherapy and Placebo)Number of Participants With Tumor Response (Complete Response [CR] + Partial Response [PR])Complete Response5 Participants
Arm B (Combination Chemotherapy and Placebo)Number of Participants With Tumor Response (Complete Response [CR] + Partial Response [PR])Partial Response19 Participants
Arm B (Combination Chemotherapy and Placebo)Number of Participants With Tumor Response (Complete Response [CR] + Partial Response [PR])Progressive Disease13 Participants
Arm B (Combination Chemotherapy and Placebo)Number of Participants With Tumor Response (Complete Response [CR] + Partial Response [PR])Stable Disease17 Participants
Secondary

Overall Survival (OS)

Kaplan-Meier methods will be used to summarize OS. Hazard ratios for OS will be presented using point estimates and 95% confidence intervals.

Time frame: 5 years

ArmMeasureValue (MEDIAN)
Arm A (Combination Chemotherapy and Erlotinib Hydrochloride)Overall Survival (OS)16.95 months
Arm B (Combination Chemotherapy and Placebo)Overall Survival (OS)13.67 months
Secondary

Rash Rates

Participants with a Rash of at least grade 2 within cycle 1.

Time frame: 5 years

ArmMeasureValue (COUNT_OF_PARTICIPANTS)
Arm A (Combination Chemotherapy and Erlotinib Hydrochloride)Rash Rates20 Participants
Arm B (Combination Chemotherapy and Placebo)Rash Rates4 Participants

Source: ClinicalTrials.gov · Data processed: Feb 26, 2026