Safety and efficacy of BEZ235 in patients with perivascular epithelioid cell tumors (PEComas)

A phase II study of orally administered BEZ235 monotherapy in patients with metastatic or unresectable malignant PEComa

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2011-001884-39-HU

Enrollment

Registered

2012-09-27

Start date

2012-11-05

Completion date

Unknown

Last updated

2013-06-24

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Adult patient with metastatic or unresectable malignant perivascular epithelioid cell tumors (PEComa) MedDRA version: 14.1 Level: PT Classification code 10039491 Term: Sarcoma System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)

Interventions

Product Name: BEZ235 Product Code: BEZ235 Pharmaceutical Form: Powder for oral suspension INN or Proposed INN: not yet available CAS Number: 1028385-32-1 Current Sponsor code: BEZ235 Concentration uni

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Histologically confirmed diagnosis of malignant PEComa (included epithelioid angiomyolipoma (AML)) in adult patients Unresectable/advanced and/or metatstatic and documented progressive measurable disease Treated with 1 or 2 prior lines of treatment Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range ;Inclusion criteria: Histologically confirmed diagnosis of malignant PEComa (included epithelioid angiomyolipoma (AML)) in adult patients Unresectable/advanced and/or metatstatic and documented progressive measurable disease Treated with 1 or 2 prior lines of treatment Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: Disease exclusions : lymphangioleiomyomatosis (LAM) exclusively, ative uncontrolled or symptomatic CNS metastases, concurrent malignancy or malignancy in last 3 years Concurrent severe and/or uncontrolled medical conditions (for details see protocol) Other protocol-defined inclusion/exclusion criteria may apply ;Exclusion criteria: Disease exclusions : lymphangioleiomyomatosis (LAM) exclusively, ative uncontrolled or symptomatic CNS metastases, concurrent malignancy or malignancy in last 3 years Concurrent severe and/or uncontrolled medical conditions (for details see protocol) Other protocol-defined inclusion/exclusion criteria may apply

Design outcomes

Primary

Measure	Time frame
Main Objective: To determine the efficacy of BEZ235 in PEComa patients;Secondary Objective: To determine the PFS rate at 32 weeks in the included population To assess the duration of response among responders To evaluate time to response To evaluate the time to progression To assess the overall survival To evaluate safety and tolerability of BEZ235;Primary end point(s): Objective Response Rate (best response on study) according to RECIST 1.1 criteria;Timepoint(s) of evaluation of this end point: 32 weeks after the last enrolled patient ;Main Objective: To determine the efficacy of BEZ235 in PEComa patients;Secondary Objective: To determine the PFS rate at 32 weeks in the included population To assess the duration of response among responders To evaluate time to response To evaluate the time to progression To assess the overall survival To evaluate safety and tolerability of BEZ235;Primary end point(s): Objective Response Rate (best response on study) according to RECIST 1.1 criteria;Timepoint(s) of evaluation of this end point: 32 weeks after the last enrolled patient	—

Measure

Time frame

Main Objective: To determine the efficacy of BEZ235 in PEComa patients;Secondary Objective: To determine the PFS rate at 32 weeks in the included population To assess the duration of response among responders To evaluate time to response To evaluate the time to progression To assess the overall survival To evaluate safety and tolerability of BEZ235;Primary end point(s): Objective Response Rate (best response on study) according to RECIST 1.1 criteria;Timepoint(s) of evaluation of this end point: 32 weeks after the last enrolled patient ;Main Objective: To determine the efficacy of BEZ235 in PEComa patients;Secondary Objective: To determine the PFS rate at 32 weeks in the included population To assess the duration of response among responders To evaluate time to response To evaluate the time to progression To assess the overall survival To evaluate safety and tolerability of BEZ235;Primary end point(s): Objective Response Rate (best response on study) according to RECIST 1.1 criteria;Timepoint(s) of evaluation of this end point: 32 weeks after the last enrolled patient

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Secondary

Measure	Time frame
Secondary end point(s): PFS rate at 32 weeks of follow-up Duration of response using with RECIST 1.1 criteria Time to response Time to progression Overall survival Frequency and severity of adverse events and laboratory values abnormalities Other safety data as considered appropriate;Timepoint(s) of evaluation of this end point: 32 weeks after the last enrolled patient ;Secondary end point(s): PFS rate at 32 weeks of follow-up Duration of response using with RECIST 1.1 criteria Time to response Time to progression Overall survival Frequency and severity of adverse events and laboratory values abnormalities Other safety data as considered appropriate;Timepoint(s) of evaluation of this end point: 32 weeks after the last enrolled patient	—

Measure

Time frame

Secondary end point(s): PFS rate at 32 weeks of follow-up Duration of response using with RECIST 1.1 criteria Time to response Time to progression Overall survival Frequency and severity of adverse events and laboratory values abnormalities Other safety data as considered appropriate;Timepoint(s) of evaluation of this end point: 32 weeks after the last enrolled patient ;Secondary end point(s): PFS rate at 32 weeks of follow-up Duration of response using with RECIST 1.1 criteria Time to response Time to progression Overall survival Frequency and severity of adverse events and laboratory values abnormalities Other safety data as considered appropriate;Timepoint(s) of evaluation of this end point: 32 weeks after the last enrolled patient

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Countries

Hungary, Netherlands, Spain

Contacts

Public ContactClinical Trial Information Desk;Clinical Trial Information Desk ;

Novartis Pharma Services AG;Novartis Pharma Services AG

clinicaltrial.enquiries@novartis.com;clinicaltrial.enquiries@novartis.com+41613241111;+41613241111

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026