Erlotinib in Women With Squamous Cell Carcinoma of the Vulvar

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00476476
Recruitment Status : Completed
First Posted : May 22, 2007
Results First Posted : March 19, 2015
Last Update Posted : May 7, 2015
Genentech, Inc.
Beth Israel Deaconess Medical Center
Brigham and Women's Hospital
Women and Infants Hospital of Rhode Island
Information provided by (Responsible Party):
Neil S. Horowitz, MD, Dana-Farber Cancer Institute

Brief Summary:
In this research study we are looking to see how vulvar cancer responds to erlotinib therapy. Two distinct patient populations are targeted: women with locally advanced measurable squamous cell carcinoma of the vulva, primary or recurrent, who are candidates for definitive treatment with surgery or chemoradiation (Cohort 1) and women with radiographically measurable distant metastatic cancer either at time of presentation or with recurrence (Cohort 2). Another goal of this study is to learn more about the proteins and genes present in vulvar cancer and how they may affect response to erlotinib. Erlotinib treats cancer by preventing cancer cells from growing and multiplying. It does this by blocking certain proteins that are on the surface of some types of cancer cells. Laboratory tests show that vulvar cancer cells have high levels of these proteins.

Condition or disease Intervention/treatment Phase
Squamous Cell Carcinoma Drug: Erlotinib Phase 2

Detailed Description:



• To determine the clinical efficacy of erlotinib in reducing the size of vulvar squamous cell cancer and /or metastatic lesions.


  • To determine the safety and tolerability of oral erlotinib.
  • To evaluate apoptosis and assess the Ki67, phospho-EGFR, EGFR mutation and EGFR amplification status of the vulvar cancer prior to and after therapy and correlate observed changes with response to therapy.
  • To evaluate the impact of medical treatment on the subsequent surgery for vulvar cancer when surgery is chosen as the definitive therapy.


This study used a two-stage design to evaluate efficacy of erlotinib based on response determined prior to definitive surgery or chemoradiation (cohort 1) or after every 2 cycles of erlotinib (cohort 2). The null and alternative response rates defined as achieving partial response (PR) or better were 3.5% and 15%. If 1 or more patients enrolled in stage one (n=17 patients) achieved PR or better than accrual would proceed to stage two (n=24 patients). There was 0.55 probability of stopping the trial at stage one if the true OR rate was 3.5%. If 3 or fewer responses were observed by the end of stage two, erlotinib would be deemed ineffective. The significance level of the study design was 0.0495 with a power of 85% to rule out a poor response rate of less than 3.5%.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 41 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase II Trial of Tarceva (Erlotinib) in Women With Squamous Cell Carcinoma of the Vulvar
Study Start Date : December 2006
Actual Primary Completion Date : October 2012
Actual Study Completion Date : October 2012

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: Erlotinib
Patients rcvd oral erlotinib 150 mg/day. Cohort 1 pts would have at least 28 days and no more than 42 days of therapy in advance of definitive therapy (surgery or chemoradiation). Cohort 2 pts continued on therapy (28 days per cycle) until disease progression, unacceptable toxicity or withdrawal of consent. Two potential dose reductions were prescribed to 100 and 50 mg/day.
Drug: Erlotinib
Orally every day for about 4-6 weeks
Other Name: Tarceva

Primary Outcome Measures :
  1. Response Rate [ Time Frame: Assessed prior to definitive surgery or chemoradiation therapy (cohort 1 pts) or after 2 cycles of therapy (cohort 2 pts). ]
    Response is defined as achieving complete or partial response.Complete response (CR) for both cohorts was defined as resolution of all identified tumor masses on the vulva or disappearance of all target and non-target lesions with no evidence of new lesions documented by two disease assessments at least 4 weeks apart. For cohort 1 pts, a partial response (PR) was defined as a 30% reduction in the product of all diameters of the vulva tumor/tumors compared to baseline measurements. For cohort 2 pts, PR defined according to Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST 1.1) was at least a 30% decrease in the sum of the longest diameter (LD) of all target measurable lesions (baseline sum LD reference).

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Female
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Histologically confirmed measurable squamous cell carcinoma of the vulvar with an assessable lesion on the vulva or measurable metastatic disease. Tumors may be primary or recurrent. Patients must have plans for surgery or definitive treatment with chemotherapy +/-radiation unless they have measurable metastatic disease.
  • 18 years of age or older
  • No concurrent chemotherapy or radiotherapy
  • NO previous chemotherapy or radiotherapy within the preceding 1 month
  • ECOG performance status of 0-1

Exclusion Criteria:

  • Known hypersensitivity reaction to erlotinib
  • Other coexisting malignancies diagnosed within the last 5 years, with the exception of basal cell carcinoma
  • Treatment with a non-FDA approved or investigational drug within 30 days
  • Persistent toxicities (grade 2 or above) from previous treatment, expect alopecia or lymphedema
  • Serum creatinine level greater than CTC grade 2
  • Pregnancy or breast feeding
  • Severe or uncontrolled systemic disease
  • Significant clinical disorder or laboratory finding that makes it potentially unsafe for the subject to participate

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00476476

United States, Massachusetts
Beth Israel Deaconess Medical Center
Boston, Massachusetts, United States, 02115
Massachusetts General Hospital
Boston, Massachusetts, United States, 02214
Dana-Farber Cancer Institute
Boston, Massachusetts, United States, 02215
United States, Rhode Island
Women and Infants Hospital of Rhode Island
Providence, Rhode Island, United States, 02905
Sponsors and Collaborators
Dana-Farber Cancer Institute
Genentech, Inc.
Beth Israel Deaconess Medical Center
Brigham and Women's Hospital
Women and Infants Hospital of Rhode Island
Principal Investigator: Neil S. Horowitz, MD Dana-Farber Cancer Institute/Brigham and Women's Hospital

Publications of Results:
Responsible Party: Neil S. Horowitz, MD, Principal Investigator, Dana-Farber Cancer Institute Identifier: NCT00476476     History of Changes
Other Study ID Numbers: 06-174
First Posted: May 22, 2007    Key Record Dates
Results First Posted: March 19, 2015
Last Update Posted: May 7, 2015
Last Verified: March 2015

Keywords provided by Neil S. Horowitz, MD, Dana-Farber Cancer Institute:
squamous cell carcinoma of the vulvar

Additional relevant MeSH terms:
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Neoplasms, Squamous Cell
Carcinoma, Squamous Cell
Erlotinib Hydrochloride
Antineoplastic Agents
Protein Kinase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action