A Study of SGT-53 in Children With Refractory or Recurrent Solid Tumors

Inclusion Criteria:

• All patients and/or their parents or legally authorized representatives must sign a written informed consent.
• Patients must be > than 12 months and ≤ 21 years of age at the time of study enrollment.
• Body surface Area (For Dose Level -1): Patients must be ≥ 0.38 m² at the time of study enrollment.
• Patients with relapsed or refractory solid tumors (excluding primary central nervous system tumors) are eligible. Patients must have had histologic verification of malignancy at original diagnosis or relapse.
• Patients must have either measurable or evaluable disease.
• Patient's current disease state must be one for which there is no known curative therapy or therapy proven to prolong survival with an acceptable quality of life.
• Karnofsky ≥ 50% for patients > 16 years of age and Lansky ≥ 50 for patients ≤ 16 years of age.

• Patients must have fully recovered from the acute toxic effects of all prior anti-cancer chemotherapy:

  • At least 21 days after the last dose of myelosuppressive chemotherapy (42 days if prior nitrosourea).
  • At least 14 days after the last dose of a long-acting growth factor (e.g. Neulasta) or 7 days for short-acting growth factor.
  • At least 7 days after the last dose of a biologic agent.
  • At least 42 days after the completion of any type of immunotherapy, e.g. tumor vaccines.
  • At least 3 half-lives of the antibody after the last dose of a monoclonal antibody.
  • At least 14 days after local palliative XRT (small port); At least 150 days must have elapsed if prior TBI, craniospinal XRT or if ≥ 50% radiation of pelvis; At least 42 days must have elapsed if other substantial bone marrow radiation.
  • No evidence of active graft vs. host disease and at least 84 days must have elapsed after transplant or stem cell infusion.
  • Patient must not have had prior exposure to gene vector delivery products within 3 months.
  • Patients may not have had prior SGT-53. Patient who have received prior topotecan, cyclophosphamide, or both are eligible.

• Adequate Bone Marrow Function:

  • Peripheral absolute neutrophil count (ANC) ≥ 1000/mm³.
  • Platelet count ≥ 100,000/mm³.

• Adequate Renal Function:

  • Creatinine clearance or radioisotope GFR ≥ 70ml/min/1.73 m² OR age/gender appropriate serum creatinine.

• Adequate Liver Function:

  • Bilirubin (sum of conjugated + unconjugated) ≤ 1.5 x upper limit of normal (ULN) for age.
  • SGPT (ALT) ≤ 110 U/L.
  • Serum albumin ≥ 2 g/dL.

Exclusion Criteria:

• Are pregnant or breast-feeding women.

• Concomitant medications:

  • Patients receiving corticosteroids who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible.
  • Patients who are currently receiving another investigational drug are not eligible.
  • Patients who are currently receiving other anti-cancer agents are not eligible.
  • Patients who are receiving cyclosporine, tacrolimus or other agents to prevent graft-versus-host disease post bone marrow transplant are not eligible for this trial.
• Patients who have an uncontrolled infection are not eligible.
• Patients who have received a solid organ transplantation are not eligible.
• Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible.