Phase 2
Completed N=71
Treatment of Patients With RAD001 With Progressive Sarcoma
Progressive Sarcoma
Source: ClinicalTrials.gov NCT00767819 ↗
Enrolled (actual)
71
Serious AEs
52.1%
Results posted
Apr 2019
Primary outcomePrimary: Best Overall Response Rates by Week 16 (ITT) — 0; 0; 0; 0 percentage of participants — p=0.1
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Best Overall Response Rates by Week 16 (ITT) |
0; 0; 0; 0; 0; 0 | 0.1 |
| SECONDARY Objective Tumor Response Rates (Complete Response and Partial Response) at Week 16 (ITT) |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Percentage of Participants With Duration of Response (CR, PR, SD) at 16 Weeks. |
60; 62.5; 100 | — |
| SECONDARY Percentage of Participants With Progression-free Survival (PFS) at 16 Weeks |
35.1; 30.5; 100.0 | — |
| SECONDARY Time to Progression (TTP) (ITT) |
57; 57; 499 | — |
| SECONDARY Percentage of Participants With Overall Survival (OS) at Week 16 (ITT) |
68.8; 56.9; 100.0 | — |
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
Data sourced from ClinicalTrials.gov (NCT00767819). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.