Clinical trial NCT01427244: Evaluation of Trastuzumab In Patients With HER2 Positive Persistent Or Recurrent Vulvar Paget's Disease
Official title: A Phase II Evaluation of Trastuzumab In Patients With HER2 Positive Persistent Or Recurrent Vulvar Paget's Disease
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE2
Conditions: Paget's Disease of the Vulva
Keywords: HER2+
Histologic confirmation
measurable disease
HER2 positive
HER2 overexpression
persistent vulvar Paget's disease
recurrent vulvar Paget's disease
Lead sponsor: Yale University
Sponsor class: OTHER
Collaborators: name: Genentech, Inc.
First posted: 2011-09-01
Enrollment: 0
Sex: FEMALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.

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

Eligibility criteria: 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

Primary outcomes:
measure: measurement of the longest diameter for all target lesions
timeFrame: 5 years
Secondary outcomes:
measure: To assess the safety profile of trastuzumab in persistent or recurrent vulvar Paget's disease
timeFrame: 5 years
Other outcomes:
Locations:
city: New Haven
country: United States
facility: Smilow Cancer Hospital at Yale New Haven
state: Connecticut
Study officials:
affiliation: Yale University
name: Thomas J. Rutherford, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: