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

Phase 2
18 Years
Open (Enrolling)
Paget's Disease of the Vulva

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Trial Information

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

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

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

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

measurement of the longest diameter for all target lesions

Outcome Description:

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.

Outcome Time Frame:

5 years

Safety Issue:


Principal Investigator

Thomas J. Rutherford, MD, PhD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Yale University


United States: Institutional Review Board

Study ID:




Start Date:

August 2011

Completion Date:

September 2015

Related Keywords:

  • Paget's Disease of the Vulva
  • HER2+
  • Histologic confirmation
  • measurable disease
  • HER2 positive
  • HER2 overexpression
  • persistent vulvar Paget's disease
  • recurrent vulvar Paget's disease



Smilow Cancer Hospital at Yale New Haven New Haven, Connecticut  06510