EP-ILC-201, Phase 2 Study of Inhaled Lipid Cisplatin in Pulmonary Recurrent Osteosarcoma
Single arm, open-label, multicenter, Simon Two-Stage Minimax Design Phase 2 study of iLC, having the following parameters: p(0)[?]0.63, p(1)[?]0.80, ([RegisteredTM],[MiCRo-SYMBoL]) [?] 0.05, 0.20.
* Proportion of patients with a Favorable Response, analyzed using a Simon Two-Stage Minimax Design. a [Quote]Favorable Response[Quote] will be defined as an observed RFi greater than the CoSS median RFi adjusted prospectively for each patient based on that patient's prior RFi. Data from the european CoSS series of osteosarcoma patients ([Quote]CoSS[Quote])1, 2, 4 indicates a median Relapse Free interval (RFi) of 0.76 years for patients experiencing a second relapse and 0.54 years for patients experiencing a third relapse.
* Percentage of patients with observed RFi exceeding their previous RFi.
* Pulmonary relapse free interval (pRFi) measured as the time
1. History of Osteosarcoma metastatic to the lung(s). (First or second pulmonary recurrence(s) removed by surgery, and previous second-line systemic chemotherapy is allowed).
2. Patients must be macroscopically disease free following pulmonary metastectomy of a single or multiple lesions. Complete Remission surgically (free of macroscopic disease) is required. Pleural disruption and/or microscopic positive margins are allowed.
3. Age >=13 years.
4. Patients must have recovered sufficiently from all acute adverse effects of prior therapies, excluding alopecia.
5. Patients must have an ECOG performance status of 0-2. (Lansky score of 50-100 if < 16 years old).
6. Patients must have recovered sufficiently from surgery and have adequate airflow and pulmonary reserve. This decision should be at the investigator[Single Quote]s discretion taking into consideration pre-surgery pulmonary function. (As a guideline: adequate airflow defined by a measured Forced
Expiratory Volume (FEV1) not less than 50% of the predicted value and adequate pulmonary reserve as evidenced by a FEV1/FVC ratio of 65% or greater).
7. Patients must have adequate renal function as defined by a serum creatinine of <= 1.5 mg/dl.
8. Patients must have adequate liver function as defined by total bilirubin of <= 1.5 mg/dl and ALT or AST < 2.5 times the institution's upper normal limit.
9. Patients must have adequate bone marrow function as defined by an absolute neutrophil count (ANC) of >= 1,000/mm3 and platelet count of >= 100,000/mm3.
10. Signed informed consent including, where applicable, the consent of the patient[Single Quote]s legal guardian.