Single-Patient Expanded Access Protocol - RNA-LP in a Patient With Recurrent Osteosarcoma

Single-Patient Expanded Access Treatment Protocol for Use of RNA Loaded Lipid Particles (RNA-LPs) in a Patient With Recurrent Osteosarcoma

Treatment of a patient with recurrent osteosarcoma with personalized pp65/tumor mRNA RNA-LP under an individual patient expanded access protocol.

The prognosis for patients with recurrent osteosarcoma and measurable disease is dismal and there are no FDA-approved therapies for this indication. A patient was enrolled on a clinical trial to develop a personalized pp65/tumor mRNA RNA-LP; however, the patient progressed following pp65 RNA-LP and had to be taken off treatment. Because the patient had not yet received personalized pp65/tumor mRNA RNA-LP, the IND sponsor treated the patient under an expanded access, individual patient protocol.

Inclusion Criteria:

• Participated in clinical trial
• Received pp65 RNA-LPs alone and had pp65/tumor mRNA RNA-LP manufactured
• Progressive disease prior to treatment with pp65/tumor mRNA RNA-LP

Exclusion Criteria:

• Diagnosis of recurrent OSA is not histopathologically confirmed (i.e. pseudoprogression)