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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
71
2.5 and 5 mg tablets taken orally and starting dose was 10 mg daily for all patients
Novartis Investigative Site
Bad Saarow, Germany
Novartis Investigative Site
Düsseldorf, Germany
Novartis Investigative Site
Essen, Germany
Novartis Investigative Site
Mannheim, Germany
Best Overall Response Rates by Week 16 (ITT)
The best overall response is the best response recorded from treatment start until disease progression/recurrence (both measurement and confirmation criteria). Best lesion response was defined by (Resist Criteria V1 for target and non-target lesions): Complete Response (CR)=at least two determinations of CR 4 weeks apart before progression, Partial Response (PR)=at least two determinations of CR 4 weeks apart before progression (and not qualifying for a CR), Stable Disease (SD)=at least one SD assessment \>6 weeks after start of treatment and Progressive Disease (PD)=Progression or death due to underlying cancer ≤16 weeks after start of treatment. PD without radiologic evidence were classified as progression only, when clear evidence of clinical deterioration was available and patient discontinued due to "disease progression". Unknown (UNK) = all other cases.
Time frame: Baseline up to 16 weeks
Objective Tumor Response Rates (Complete Response and Partial Response) at Week 16 (ITT)
The best overall response is the best response recorded from the start of the treatment until disease progression/recurrence (taking as reference for PD the smallest measurements recorded since the treatment started). In general, the patient's best response assignment will depend on the achievement of both measurement and confirmation criteria. Best lesion response was defined by (Resist Criteria V1 for target and non-target lesions).
Time frame: Baseline up to approximately 16 weeks
Percentage of Participants With Duration of Response (CR, PR, SD) at 16 Weeks.
Duration of response (CR, PR or SD) applied only to those patients whose best overall response was CR, PR or SD based on local radiologic assessments and was defined as the time from start of treatment to progression or death from underlying disease. Patients not experiencing progression or death at 16 weeks were censored with the date of their last tumor assessment. Duration of response was explored using the Kaplan-Meier method.
Time frame: Baseline up to 16 weeks
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Novartis Investigative Site
München, Germany
Novartis Investigative Site
Milan, MI, Italy
Percentage of Participants With Progression-free Survival (PFS) at 16 Weeks
Progression-free Survival (PFS) was defined as the time from the date of start of treatment to the date of event defined as the first documented progression or death from any cause. If a patient had not had an event, PFS was censored at the date of the last adequate tumor assessment at week 16
Time frame: 16 weeks
Time to Progression (TTP) (ITT)
Time to progression (TTP) was defined as the time from the date of start of treatment to the date of event defined as the first documented progression or death from underlying disease. If a patient had not had an event, TTP was censored at the date of the last adequate tumor assessment, which was the date of Visit 6 (Week 16) for the core phase and the last available tumor assessment for the follow-up phase. TTP was explored by using the Kaplan-Meier method.
Time frame: Baseline up to 16 weeks
Percentage of Participants With Overall Survival (OS) at Week 16 (ITT)
Overall survival (OS) was defined as the time from the date of start of treatment to death from any cause. If a patient was not known to have died (was alive), OS was censored at the date of the last contact, which was the date of Visit 6 (Week 16) for the core phase and the last available visit for the follow-up phase. OS was explored by using the Kaplan-Meier method. One death occurred in Arm 3 after Week 16.
Time frame: Baseline up to 16 weeks