Erlotinib in Treating Patients With Recurrent or Metastatic Skin Squamous Cell Carcinoma

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: NCT01198028
Recruitment Status : Active, not recruiting
First Posted : September 9, 2010
Last Update Posted : September 19, 2018
National Cancer Institute (NCI)
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

September 8, 2010
September 9, 2010
September 19, 2018
March 10, 2011
March 31, 2019   (Final data collection date for primary outcome measure)
Overall response rate defined as the percentage of patients who achieve an overall response of complete response or partial response in the total number of evaluable patients, assessed by Response Evaluation Criteria in Solid Tumors 1.1 [ Time Frame: After 8 weeks of study therapy ]
A Bayesian design based on predictive probability will be implemented.
Overall Response Rate (ORR) [ Time Frame: 8 weeks ]
Evaluated after 8 weeks, patient considered a non-responder if tumor does not regress to complete or partial response as specified in RECIST v1.1 at that time point. ORR, based on overall response of each evaluable patient, is defined as percentage of patients who achieve an overall response of complete response or partial response in total number of evaluable patients.
Complete list of historical versions of study NCT01198028 on Archive Site
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Erlotinib in Treating Patients With Recurrent or Metastatic Skin Squamous Cell Carcinoma
Phase II Study of Erlotinib, An Epidermal Growth Factor Receptor (EGFR) Tyrosine Kinase Inhibitor, in the Treatment of Recurrent or Metastatic Squamous Cell Carcinoma of the Skin
This phase II trial studies how well erlotinib works in treating participants with skin squamous cell carcinoma that has spread to other places in the body or has come back. Drugs used in chemotherapy, such as erlotinib, 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.


I. To determine the overall response rate with erlotinib in patients with locoregionally recurrent or metastatic squamous cell carcinoma of the skin (CSCC) that is not amenable to curative treatment.


I. To determine duration of response and duration of stable disease. II. To determine progression-free and overall survival. III. To determine safety and tolerability of erlotinib.


I. To correlate baseline expression of estimated glomerular filtration rate (EGFR), expression of markers of EGFR activation (such as phosphorylated [p] EGFR and pAKT) and related cell-signaling pathways, and EGFR mutation status with response to erlotinib therapy.

II. To determine the effects of erlotinib on relevant biomarkers of the EGFR pathway in tumor tissue and in normal skin, and to correlate with response to therapy.

III. To determine if there is a correlation between the development of erlotinib-induced skin rash and response to therapy.


Participants receive erlotinib orally (PO) once daily (QD) in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, participants are followed up every 3 months for up to 2 years.

Phase 2
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
  • Metastatic Skin Squamous Cell Carcinoma
  • Recurrent Skin Squamous Cell Carcinoma
Drug: Erlotinib
Given PO
Experimental: Treatment (erlotinib)
Participants receive erlotinib PO QD in the absence of disease progression or unacceptable toxicity.
Intervention: Drug: Erlotinib
Gold KA, Kies MS, William WN Jr, Johnson FM, Lee JJ, Glisson BS. Erlotinib in the treatment of recurrent or metastatic cutaneous squamous cell carcinoma: A single-arm phase 2 clinical trial. Cancer. 2018 May 15;124(10):2169-2173. doi: 10.1002/cncr.31346. Epub 2018 Mar 26.

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Active, not recruiting
Same as current
March 31, 2019
March 31, 2019   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • Have histologically or cytologically confirmed cutaneous squamous cell carcinoma (CSCC) that is not amenable to curative therapy. If the biopsy was collected outside of MD Anderson Cancer Center (MDACC), the MDACC Pathology Department must assess and confirm the squamous cell carcinoma (SCC) diagnosis.
  • Have measurable disease.
  • Have Eastern Cooperative Oncology Group (ECOG) performance status 0-2.
  • Must have ability to understand and the willingness to sign a written Informed Consent Document (ICD). In the event that non-English speaking participants are eligible for this study, a short form (if applicable) or an ICD in their language will be utilized and completed in accordance with the MDACC "Policy For Consenting Non-English Speaking Participants.
  • Leukocytes >= 3,000/mm^3.
  • Absolute neutrophil count >= 1,500/mm^3.
  • Platelets >= 75,000/mm^3.
  • Hemoglobin >= 8g/dL.
  • Total bilirubin =< 2 x institutional upper limit of normal (ULN).
  • Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase [SGOT])/alanine aminotransferase (serum glutamic pyruvic transaminase [SGPT]) =< 2.5 x ULN if alkaline phosphatase is normal, or alkaline phosphatase =< 4 x ULN if transaminases are normal.
  • Creatinine =< 2.0 x ULN or creatinine clearance >= 60 mL/min/1.73 m^2.
  • Prior radiotherapy is allowed if: (a) there is measurable disease outside the radiation field OR (b) radiotherapy was completed more than 4 weeks ago and there is clearly recurrent and growing disease within the radiation field.
  • Must be able to take intact tablets by mouth, or be able to take tablets dissolved in water by mouth or by a percutaneous gastrostomy tube.
  • Patients - both males and females - with reproductive potential (includes women who are menopausal for less than 1 year and not surgically sterilized) must practice effective contraceptive measures such as barrier methods, condom or diaphragm with spermicide, or abstinence throughout the study. Birth control should continue for 4 weeks after discontinuation of erlotinib therapy. Women of childbearing potential must provide a negative pregnancy test (serum beta human chorionic gonadotropin [HCG]) within 72 hours prior to first receiving protocol therapy.
  • Organ transplant patients are eligible as long as they do not have active signs of rejection and have adequate bone marrow function.

Exclusion Criteria:

  • Women who are pregnant, breastfeeding, and women and men not practicing effective birth control. Erlotinib is a signal transduction inhibitor agent with the potential for teratogenic or abortifacient effects. There is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with erlotinib. Breastfeeding should be discontinued if the mother is treated with erlotinib.
  • Prior estimated glomerular filtration rate (EGFR) inhibitor therapy is not allowed (including, but not limited to, erlotinib, gefitinib, cetuximab, panitumumab, vandetanib).
  • Patients who are receiving any other anticancer or investigational agents at time of study enrollment. Patients may have received one other systemic therapy or investigational agent in the past, but a washout time period of at least 4 weeks and recovery of any treatment-related toxicities to < Common Terminology Criteria for Adverse Events version 4 (CTCAEv4) grade 2 is required.
  • History of allergic reactions attributed to compounds of similar chemical or biologic composition to erlotinib.
  • Patients with a history of an invasive malignancy (other than the one treated in this study) or lymphoproliferative disorder within the past 3 years. Patients with a history of adequately treated non-melanoma skin cancer, ductal carcinoma in situ of the breast, or carcinoma in situ of the cervix are allowed.
  • Patients with incomplete healing from previous surgery.
  • Patients with pulmonary fibrosis (other than in a radiated field) or active interstitial lung disease.
  • Patients with active gastrointestinal disease or a disorder that alters gastrointestinal motility or absorption, including lack of integrity of the gastrointestinal tract (for example, a significant surgical resection of the stomach or small bowel, inflammatory bowel disease or uncontrolled chronic diarrhea.
  • Patients with skin rash CTCAEv4 grade 2.
  • In the opinion of the investigator, patients with any condition that is unstable or could jeopardize the safety of the patient or could limit compliance with the study's requirements. These include, but are not limited to, ongoing or active infection requiring parenteral antibiotics at time of study registration, psychiatric illness that would limit compliance with study requirements or symptomatic congestive heart failure (New York Heart Association [NYHA] class II or greater), unstable angina pectoris or cardiac arrhythmia requiring maintenance medication.
  • Patient is unwilling or unable to discontinue prohibited concomitant therapies, (i.e St. John's wort, grapefruit juice, histamine type 2 receptor [H2] blockers/proton pump inhibitors, strong CYP3A4 inhibitors and inducers).
Sexes Eligible for Study: All
18 Years and older   (Adult, Older Adult)
Contact information is only displayed when the study is recruiting subjects
United States
NCI-2018-01834 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
2009-0888 ( Other Identifier: M D Anderson Cancer Center )
P30CA016672 ( U.S. NIH Grant/Contract )
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Not Provided
M.D. Anderson Cancer Center
M.D. Anderson Cancer Center
National Cancer Institute (NCI)
Principal Investigator: Bonnie Glisson M.D. Anderson Cancer Center
M.D. Anderson Cancer Center
September 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP