Neoadjuvant Erlotinib (Tarceva) in Transitional 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: NCT00749892
Recruitment Status : Active, not recruiting
First Posted : September 9, 2008
Last Update Posted : January 10, 2018
Genentech, Inc.
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

September 5, 2008
September 9, 2008
January 10, 2018
June 2008
June 2019   (Final data collection date for primary outcome measure)
Response Rate (pT0 rate) of participants with urothelial cancer treated with erlotinib prior to cystectomy [ Time Frame: Baseline response to response after 3 weeks of therapy ]
Response Rate (ie: pT0 rate), defined as the absence of residual cancer in the resected specimen. All specimens reviewed by a genitourinary pathologist.
To learn if Tarceva (erlotinib hydrochloride; OSI-774) can help to shrink the tumor or slow its growth, before surgery, in patients with transitional cell carcinoma. [ Time Frame: 4 Years ]
Complete list of historical versions of study NCT00749892 on Archive Site
Disease-Free Survival of Patients with Urothelial Cancer Treated with Erlotinib Prior to Cystectomy [ Time Frame: Every 6 months for first year after surgery, then once a year for up to 4 years ]
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Neoadjuvant Erlotinib (Tarceva) in Transitional Cell Carcinoma
A Phase II Exploratory Study of Pre-Operative Treatment With Erlotinib (Tarceva) in Muscle Invasive or Recurrent Transitional Cell Carcinoma Requiring Cystectomy
The goal of this clinical research study is to learn if Tarceva™ (erlotinib hydrochloride; OSI-774) can help to shrink the tumor or slow its growth, before surgery, in patients with transitional cell carcinoma.

The Study Drug:

Erlotinib hydrochloride is designed to block the activity of a protein found on the surface of many tumor cells that may control tumor growth and survival. This may stop tumors from growing.

Study Drug Administration:

If you are found to be eligible to take part in this study, you will begin taking erlotinib hydrochloride (a pill) by mouth once a day. You should take it at about the same time each day, at least 1 hour before or 2 hours after a meal. You should swallow the pill with about a cup (8 ounces) of water.

It is important that you take the drug every day, as prescribed. If necessary during the study, the study doctor may decide to lower the dose.

Study Visits:

Once a week during this study, you will have the following tests performed before you take the study drug:

  • Your vital signs will be measured.
  • You will be asked about any side effects you may have experienced.
  • Blood (about 1 teaspoon) will be drawn for routine tests.

Length of Study Participation:

You may continue receiving the study drug for 3-5 weeks (depending on when your surgery is scheduled). After that, you will be offered surgery to remove the tumor. You will take your last study drug dose within 24 hours before having surgery. You will sign a separate consent form for the surgery, and the procedure and its risks will be described in more detail at that time.

If the disease gets worse or intolerable side effects occur, you will be taken off study early.

If you stop taking the study drug early because of intolerable side effects, you will need to return to the clinic every 2 weeks until surgery. At these visits, your side effects will be monitored, for example with routine blood tests and/or scans, depending on what the doctor decides is necessary.

Long-Term Follow-Up:

After surgery, all study participants will return for routine follow-up visits with the surgeon.

Every 6 months for the first year after surgery and once a year for the next 4 years after that, the study staff will call you to see how you are doing.

This is an investigational study. Erlotinib hydrochloride is FDA approved and commercially available for the treatment of non-small cell lung cancer and pancreatic cancer. Its use in this study is investigational and not FDA approved. For this purpose, at this time it is being used in research only.

Up to 40 patients will take part in this study. All will be enrolled at M. D. Anderson.

Phase 2
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Transitional Cell Carcinoma
Drug: Erlotinib Hydrochloride
150 mg (pill) by mouth daily for 3 to 5 weeks prior to surgery
Other Names:
  • Tarceva
  • OSI-774
Experimental: Erlotinib Hydrochloride
Erlotinib Hydrochloride 150 mg (pill) by mouth daily for 3 to 5 weeks prior to surgery.
Intervention: Drug: Erlotinib Hydrochloride
Not Provided

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

Inclusion Criteria:

  1. Patients must have histologic proof of urothelial cancer. This includes bladder cancer, in addition to other tumors of the urothelial lining including renal pelvis, ureteral, and urethral cancer. This group may include any patient requiring cystectomy, including patients with recurrent or extensive superficial disease (cTa-T1N0M0), CIS (carcinoma in situ), or muscle invasive disease (cT2-3aN0M0), whose tumor could not be completely removed at transurethral resection.
  2. Patients with the following high-risk features: Micropapillary features (more than focal on pathology); Small cell carcinoma; 3-D mass on exam under anesthesia (EUA); Lymphovascular invasion; Hydronephrosis (unless in the opinion of the treating physician, this is not due to tumor);High grade (grade 3) tumors of the ureter, renal pelvis, or urethra, or tumors in these areas with radiographic abnormality large enough to recognize as an abnormal mass by CT or MRI imaging;
  3. (# 2 cont'd) Direct invasion of the prostatic stroma or the vaginal wall (ie: cT4a disease) should be offered neoadjuvant cytoreductive chemotherapy (ie: cisplatin-based). Patients refusing or who are not considered candidates for cytoreductive chemotherapy may be considered eligible. Dr. Siefker-Radtke will be the final arbiter in determining eligibility for the trial.
  4. Please note that the presence of variant histologic subtypes is acceptable, except in the case for small cell variant which is traditionally treated with cytoreductive chemotherapy. Patients with small cell who refuse recommended cytoreductive chemotherapy may still be considered eligible.
  5. Patients must have an evaluation in the department of urology, and be deemed an acceptable surgical candidate.
  6. Patients must NOT have clinical evidence of metastatic disease by either CT or MRI of the abdomen and pelvis, and chest x-ray. In the absence of a bone scan, patients should be free of bone pain and have an alkaline phosphatase < 1.5 x ULN of the upper limit of normal, or a normal bone fraction of alkaline phosphatase. If these features are present, patients should have a bone scan and this should be interpreted as showing no evidence of metastatic disease in order to be eligible.
  7. Individuals must be > 18 years of age. In general, urothelial cancer occurs in the 6th to 7th decade of life, so it is unlikely that pediatric patients will be included.
  8. Patients, 18 years and older, must either be not of child bearing potential or have a negative pregnancy test within 2 weeks of treatment. Patients are considered not of child bearing potential if they are surgically sterile (they have undergone a hysterectomy, bilateral tubal ligation, or bilateral oophorectomy) or they are postmenopausal.
  9. Bone marrow function: absolute neutrophil count (ANC) >/= 1,000/ul; platelets >/= 75,000/microliters.
  10. Renal function: creatinine </= 2.0 x institutional upper limit of normal (ULN), or a creatinine clearance of > 30 ml/min as calculated by Cockcroft-Gault or by 24-hour urine collection.
  11. Hepatic function: bilirubin </= 2.5 x ULN; AST </= 5.0 x ULN.
  12. Zubrod PS </= 2.
  13. Patients with second malignancies are eligible provided that the expected outcome from the second cancer is such that this will not interfere in the delivery of this therapy, or in doing cystectomy, and provided that the expectation of survival from any prior malignancy is reliably > 4 years.

Exclusion Criteria:

  1. Acute hepatitis or known HIV.
  2. Active or uncontrolled infection.
  3. Significant history of uncontrolled cardiac disease; i.e., uncontrolled hypertension, unstable angina, recent myocardial infarction (within prior 6 months), uncontrolled congestive heart failure, and cardiomyopathy with decreased ejection fraction </= 40%.
  4. Prior therapy specifically and directly targeting the EGFR pathway.
  5. Patients with interstitial lung disease.
  6. Any concurrent chemotherapy not indicated in the study protocol or any other investigational agent(s).
  7. Patients with metastatic or surgically unresectable disease are not eligible for this study. In addition, patients who do not agree to surgery are not eligible for this trial.
  8. Patients who have received prior systemic chemotherapy or radiation therapy for urothelial cancer are not eligible. Any prior intravesical chemotherapy is allowed.
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-2010-01025 ( Registry Identifier: NCI CTRP )
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
Genentech, Inc.
Principal Investigator: Arlene Siefker-Radtke, MD M.D. Anderson Cancer Center
M.D. Anderson Cancer Center
January 2018

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