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A Phase II Study of Orally Administered BEZ235 Monotherapy in Patients With Metastatic or Unresectable Malignant PEComa

Phase 2
18 Years
Open (Enrolling)
Malignant PEComa (Perivascular Epithelioid Cell Tumors)

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Trial Information

A Phase II Study of Orally Administered BEZ235 Monotherapy in Patients With Metastatic or Unresectable Malignant PEComa

Inclusion Criteria

Inclusion criteria

1. Histologically confirmed diagnosis of malignant PEComa (included epithelioid AML) of
primary disease or of metastatic lesion from archival tissue if it has been obtained
within 12 months prior to enrollment in this study. This histological diagnosis
includes immunochemistry as follows:

- Immunohistochemically positive expression of a melanocytic marker (HMB45, MelanA
or microphtalmia transcription factor) AND of a smooth muscle marker (smooth
muscle actin, pan-muscle actin, h-caldesmon or calponin) is mandatory on primary
or metastatic tumor biopsy.

- Note: According to Folpe (2002), criteria for malignancy in non-AML PEComas are:

- tumor size of more than 5 cm,

- infiltrative growth pattern,

- high nuclear grade,

- mitotic activity of more than 1/50 high power field (HPF),

- necrosis,

- vascular invasion

- Thus, to be included in the trial, the patient should have tumor presenting with
2 or more criteria for malignancy associated with aggressive clinical behavior.

2. If the primary diagnosis was performed more than 12 months before enrollment, the
histology of a primary/metastatic lesion should be reconfirmed with a fresh biopsy.

3. Availability of a representative tumor specimen, either archival or fresh tumor
tissue for PI3K pathway analysis.

4. Unresectable/advanced and/or metastatic and documented progressive measurable disease
as defined by RECIST 1.1 criteria. Prior to study entry, the progression of the
disease should be confirmed by at least 2 sequential CT scans available for
documentation (will be collected and hold).

5. Presence of measurable disease according to RECIST 1.1. Note: Lesions in previously
irradiated areas can only be considered measurable if they have clearly progressed
since the radiotherapy.

6. Treated with 1 or 2 prior lines of treatment Exclusion criteria

1. Disease exclusions:

1. Lymphangioleiomyomatosis (LAM) exclusively

2. Active uncontrolled or symptomatic CNS metastases Note: A patient with controlled and
asymptomatic CNS metastases may participate in this trial. As such, the patient must
have completed any prior treatment for CNS metastases > 28 days (including
radiotherapy and/or surgery) prior to start of treatment in this study and should not
be receiving chronic corticosteroid therapy for the CNS metastases.

3. Concurrent malignancy or malignancy in the last 3 years prior to start the study
treatment (with the exception of adequately treated cervical carcinoma in situ or
nonmelanoma skin cancer).

Type of Study:


Study Design:

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

Outcome Measure:

Proportion of Patients with best Objective Response Rate (ORR)

Outcome Description:

Objective Response Rate is defined as the proportion of patients with a best overall response of Complete Response (CR) or Partial Response (PR) according to RECIST. 1.1.

Outcome Time Frame:

From treatment start to end of follow-up, assessed up to 30 months

Safety Issue:


Principal Investigator

Novartis Pharmaceuticals

Investigator Role:

Study Director

Investigator Affiliation:

Novartis Pharmaceuticals


Italy: Ministry of Health

Study ID:




Start Date:

September 2012

Completion Date:

January 2015

Related Keywords:

  • Malignant PEComa (Perivascular Epithelioid Cell Tumors)
  • Malignant PEComa
  • Angiomyolipoma
  • Tuberous Sclerosis Complex
  • Pi3kinase /m-tor inhibitors
  • Perivascular Epithelioid Cell Neoplasms