RAD001 in Advanced Sarcoma

Study Overview
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Brief Summary
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This trial intends to test the efficacy and safety of RAD001 in patients with advanced sarcoma who failed to conventional chemotherapy.

Detailed Description
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This multicenter, phase II trial evaluated the efficacy and safety of everolimus in patients with metastatic or recurrent bone and soft tissue sarcoma after failure of anthracycline and/or ifosfamide.

Official Title
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A Phase II Multicenter Trial of RAD001 in Patients With Metastatic or Recurrent Sarcomas

Conditions
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Soft Tissue Sarcomas, Bone Sarcomas

Intervention / Treatment
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* Drug: RAD001


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients with histologically confirmed metastatic, unresectable bone or soft tissue sarcomas who had past treatment with anthracycline and/or ifosfamide to which the disease was primarily refractory or progressed after initial response. Any of above drugs is allowed to be used as adjuvant treatment. Unidimensionally measurable disease 3 or less than prior chemotherapies Age 17 years old or older ECOG performance status 2 or less, Life expectancy 6 month or less Adequate bone marrow, liver, kidney, and cardiac function Written informed consent Exclusion Criteria: Pregnant or lactating patients Patients with resectable metastasis Patients with history of CNS metastasis Gastrointestinal stromal tumors, chondrosarcoma, neuroblastoma Hypersensitivity to the active substance, to other rapamycin derivatives, or to any of the excipients. Any preexisting medical condition of sufficient severity to prevent full compliance with the study

Ages Eligible for Study
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Minimum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: RAD001<br>RAD001 was given as 10 mg orally once daily, and one cycle was defined as 28 days. | Drug: RAD001<br> <br> * Other names: Everolimus;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Progression-free survival rate at 16 weeks | Determined as the proportion of progression-free (complete response, partial response and stable disease) at 16 weeks of treatment | 16 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Progression-free survival | Defined as the time from the initiation of everolimus to disease progression or death, whichever occurred first. | Up to 24 months | 
| Overall survival | Defined from the initiation of everolimus to death of any cause. | Up to 24 months | 
| Response rate | Defined as the proportion of complete response and partial response per RECIST criteria. | Up to 24 months | 
| Toxicity | Any adverse events occurred during the treatment with study drug. | Up to 24 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Soft tissue sarcomas, Bone sarcomas, RAD001, Refractory to conventional chemotherapy