E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
progressive or metastatic bone or soft tissue sarcomas |
|
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10006007 |
E.1.2 | Term | Bone sarcoma |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate preliminary efficacy of RAD001 in bone and soft tissue sarcoma. Efficacy is defined as complete response, partial response or stable disease at 16 weeks. |
|
E.2.2 | Secondary objectives of the trial |
To evaluate the tolerability and safety profile of RAD001 in this patient population.  To evaluate the objective tumor response rate based on RECIST-criteria (complete response [CR] and partial response [PR]) at 16 weeks.  To evaluate duration of response.  To evaluate progression-free survival (PFS) at 16 weeks.  To evaluate overall survival (OS). To evaluate PFS at month 12 for patients with data available from follow-up observation (optional).  To evaluate OS at 12 months for patients with data available from follow-up observation (optional). |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal 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;  Ewings sarcoma;  rhabdomyosarcoma. 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. PLS see protocol |
|
E.4 | Principal 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;  chondrosarcoma;  malignant mesothelioma;  neuroblastoma.  Prior therapy with RAD001 (everolimus) or other rapamycins (sirolimus, temsirolimus).  neurotoxicity ≥ grade 2 CTC.  Radiation of the lung.  Patients taking drugs known to inhibit or induce isoenzyme CYP3A (Table 6-4) are excluded unless the drugs are medically necessary and no substitutes are available. If there are no acceptable substitutes, special precautions should be taken in these patients (section 6.3.5).  Patients with any concurrent major medical condition liable to compromise the patient`s participation in the study (e.g. known HIV infection, uncontrolled diabetes, serious cardiac dysrhythmia or condition, New York Heart Association classification of III or IV, congestive cardiac failure, myocardial infarction within 6 months, unstable angina, chronic or acute renal or liver disease, uncontrolled serious infections including abscess or fistulae, etc.).  Patients with a history of another malignancy prior to study entry, except curatively treated non-melanotic skin cancer or carcinoma in-situ cervical cancer unless in complete remission or no evidence of disease and off all therapy for that disease for a minimum of 5 years. |
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E.5 End points |
E.5.1 | Primary end point(s) |
To evaluate preliminary efficacy of RAD001 in bone and soft tissue sarcoma. Efficacy is defined as complete response, partial response or stable disease at 16 weeks. |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 2 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
| |
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 9 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 9 |