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Evaluation of Trastuzumab In Patients With HER2 Positive Persistent Or Recurrent Vulvar Paget's Disease

This study has been withdrawn prior to enrollment.
Genentech, Inc.
Information provided by (Responsible Party):
Yale University Identifier:
First received: August 31, 2011
Last updated: June 12, 2014
Last verified: June 2014
This is a phase II open-label study of the activity and safety of trastuzumab in patients with vulvar Paget's disease with overexpression of HER2 on biopsy.

Condition Intervention Phase
Paget's Disease of the Vulva
Drug: trastuzumab
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase II Evaluation of Trastuzumab In Patients With HER2 Positive Persistent Or Recurrent Vulvar Paget's Disease

Resource links provided by NLM:

Further study details as provided by Yale University:

Primary Outcome Measures:
  • measurement of the longest diameter for all target lesions [ Time Frame: 5 years ]
    Where possible measurement of the target lesion size in 2 perpendicular diameters will be required for follow up. A Change in the product of these 2 diameters affords some estimate of change in tumor size and hence therapeutic efficacy. Response criteria will be based on the RECIST modified response evaluation criteria (appendix I).The reporting of these changes will be on an individual case basis and will be in terms of the best response achieved by that case.

Secondary Outcome Measures:
  • To assess the safety profile of trastuzumab in persistent or recurrent vulvar Paget's disease [ Time Frame: 5 years ]
    To assess the safety profile for all patients who receive any amount of trastuzumab will be evaluable for toxicity.At each visit, a brief focused history will be obtained and any indication of treatment related toxicity will be evaluated by appropriate examination and/or laboratory/radiographic studies using the CTCAE v4.0 toxicity grades for both laboratory and non-laboratory data.The evaluation period should extend from date of first treatment until 30 days (or longer if so specified) from the last dose, or until resolution from all acute toxicities associated with the drug administration.

Enrollment: 0
Study Start Date: August 2011
Estimated Study Completion Date: September 2015
Estimated Primary Completion Date: September 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Trastuzumab
Open Label
Drug: trastuzumab
monoclonal antibody

Detailed Description:

The primary objective will be to estimate the anti-tumor activity of trastuzumab by evaluating the response to trastuzumab therapy in patients with recurrent or persistent vulvar Paget's disease demonstrating HER2/neu overexpression by immunohistochemistry and/ or FISH ((fluorescence in situ hybridization).

The secondary objectives:

  • To evaluate the quality of life, based on the severity of the patients' vulvar itching on a 4-point verbal rating scale from 0 to 3 (0=no itching, 1=mild itching/ bothersome, 2=moderate itching/ troublesome, 3=severe itching/ extremely troublesome) as well as on the severity of the patients' vulvar pain on a numeric pain scale from 0-10 (0=no pain, 1-3=mild pain, 4-6=moderate pain, 7-10=severe pain).
  • To assess the frequency and severity of observed adverse effects
  • To assess the frequency/ incidence of HER2 in Paget's disease of the vulva

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

Inclusion Criteria:

  • recurrent or persistent vulvar Paget's disease with overexpression of HER2 by immunohistochemistry and or fluorescence in situ hybridization (FISH)
  • Biopsy for histologic confirmation
  • Measurable disease
  • Karnofsky score of 50-100
  • recovered from effects of recent surgery, radiotherapy, or chemotherapy
  • free of active infection requiring antibiotics
  • adequate bone marrow function,renal function,hepatic function,cardiac function,WOCBP-neg.serum pregnancy,meet requirements in section 7.0

Exclusion Criteria:

  • no measurable disease
  • tumors not HER2 positive by immunohistochemistry FISH
  • prior therapy with any anti-HER2 monoclonal antibody preparation
  • Karnofsky score of 0-40
  • other invasive malignancies, with the exception of non-melanoma skin cancer
  • Patients requiring supplemental oxygen
  • unstable medical conditions in the opinion of the treating physician place them at unacceptably increased risk from trastuzumab therapy
  • Patients with active or unstable cardiac disease, or myocardial infarction within 6 months
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Please refer to this study by its identifier: NCT01427244

United States, Connecticut
Smilow Cancer Hospital at Yale New Haven
New Haven, Connecticut, United States, 06520
Sponsors and Collaborators
Yale University
Genentech, Inc.
Principal Investigator: Thomas J. Rutherford, MD, PhD Yale University
  More Information

Responsible Party: Yale University Identifier: NCT01427244     History of Changes
Other Study ID Numbers: 1004006704
Study First Received: August 31, 2011
Last Updated: June 12, 2014

Keywords provided by Yale University:
Histologic confirmation
measurable disease
HER2 positive
HER2 overexpression
persistent vulvar Paget's disease
recurrent vulvar Paget's disease

Additional relevant MeSH terms:
Antineoplastic Agents processed this record on April 27, 2017