Trial Information

Phase Ib/IIa Study of SLIT Cisplatin by Inhalation in the Treatment of Patients Wtih Relapsed/Progressive Osteosarcoma Metastatic to the Lung

Osteosarcoma, preferentially metastasizes to the lung. The presence of lung metastases has a
major impact on the prognosis of patients with osteosarcoma. Upon surgical removal of the
tumor in the lung, new pulmonary metastases often recur within months suggesting
micro-metastatic disease resistant to systemic chemotherapy.

The Sustained release lipid inhalation targeting (SLIT) technology offers the potential
ability to attain a prolonged therapeutic effect of cisplatin in the lung by sustained
release. The ability to give SLIT Cisplatin by inhalation directly to the lung permits high
drug levels at the site of disease with low systemic exposure.

Patients will receive SLIT Cisplatin by inhalation for a 14-day treatment cycle in this
phase Ib/IIa, two-center, open-label, study designed to characterize the maximum tolerated
dose. Clinical efficacy endpoints will be included and compared to historical controls, in
addition to pharmacokinetics characterization. Efficacy will be evaluated after at least 2
cycles of therapy. Safety data, including laboratory parameters and adverse events will be
collected to determine the qualitative and quantitative toxicity, and reversibility of
toxicity, of SLIT Cisplatin. Pulmonary function tests will be performed at baseline, prior
to each course and at off-study.