Clinical trial NCT00767819: Treatment of Patients With RAD001 With Progressive Sarcoma
Official title: Multicenter, Triple-arm, Single-stage, Phase II Trial to Determine the Preliminary Efficacy and Safety of RAD001 in Patients With Histological Evidence of Progressive or Metastatic Bone or Soft Tissue Sarcomas
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Progressive Sarcoma
Keywords: progressive GIST
progressive sarcoma
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2008-10-07
Enrollment: 71
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this multicenter, three-arm, exact binomial single-stage, phase II trial is to determine the preliminary efficacy and safety of RAD001 in patients with histological evidence of progressive or metastatic bone or soft tissue sarcoma.


Eligibility criteria: Inclusion Criteria:

Histological evidence of progressive or metastatic bone or soft tissue sarcoma.

The following tumor types are included:

* malignant fibrous histiocytoma
* liposarcoma
* synovial sarcoma
* malignant paraganglioma
* fibrosarcoma
* leiomyosarcoma
* angiosarcoma including haemangiopericytoma
* malignant peripheral nerve sheath tumor
* STS, not otherwise specified
* miscellaneous sarcoma including mixed mesodermal tumors of the uterus
* osteosarcoma
* Ewing's sarcoma
* rhabdomyosarcoma
* gastrointestinal stromal tumor (only after failure or intolerance of imatinib or sunitinib in 1st and 2nd line)
* alveolar soft part sarcoma (ASPS)

  * Objective progression of disease may be documented by RECIST criteria. Any of the following would be sufficient according to RECIST:
* a 20% increase in the sum of unidimensionally measured target lesions
* a new lesion
* unequivocal increase in non-measurable disease.

  * Patients must have disease not amenable to surgery, radiation, or combined modality therapy with curative intent.
  * ECOG performance status 0 - 2.

Exclusion Criteria:

Anticancer therapy within 3 weeks of enrollment including chemotherapy, hormonal therapy, immunotherapy, or radiotherapy.

* The following tumor types will not be included:

  * gastrointestinal stromal tumor (except for patients after treatment with imatinib or sunitinib in 1st and 2nd line)
  * chondrosarcoma
  * malignant mesothelioma
  * neuroblastoma.
* Prior therapy with RAD001 (everolimus) or other rapamycins (sirolimus, temsirolimus).
* Neurotoxicity \> grade 2 CTC.
* Radiation of the lung.

Other protocol-defined inclusion/exclusion criteria may apply

Primary outcomes:
measure: Best Overall Response Rates by Week 16 (ITT)
timeFrame: Baseline up to 16 weeks
Secondary outcomes:
measure: Objective Tumor Response Rates (Complete Response and Partial Response) at Week 16 (ITT)
timeFrame: Baseline up to approximately 16 weeks
measure: Percentage of Participants With Duration of Response (CR, PR, SD) at 16 Weeks.
timeFrame: Baseline up to 16 weeks
measure: Percentage of Participants With Progression-free Survival (PFS) at 16 Weeks
timeFrame: 16 weeks
measure: Time to Progression (TTP) (ITT)
timeFrame: Baseline up to 16 weeks
measure: Percentage of Participants With Overall Survival (OS) at Week 16 (ITT)
timeFrame: Baseline up to 16 weeks
Other outcomes:
Locations:
city: Bad Saarow
country: Germany
facility: Novartis Investigative Site
city: Duesseldorf
country: Germany
facility: Novartis Investigative Site
city: Essen
country: Germany
facility: Novartis Investigative Site
city: Mannheim
country: Germany
facility: Novartis Investigative Site
city: Muenchen
country: Germany
facility: Novartis Investigative Site
city: Milano
country: Italy
facility: Novartis Investigative Site
state: MI
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: