A Multi-Center Study of the Safety, Tolerability, Pharmacokinetics and Dose Escalation of Intravenous Recombinant Human Mannose-Binding-Lectin (rhMBL) in MBL Deficient Pediatric Hematology/Oncology Patients With Fever and Neutropenia

OBJECTIVES:

Primary

• Determine the safety and tolerability of recombinant human mannose-binding lectin (MBL) in pediatric patients with MBL deficiency and fever and neutropenia who are undergoing cytotoxic chemotherapy for hematological/oncological disease.
• Determine the pharmacokinetics of this drug in these patients.

Secondary

• Determine the pharmacodynamic effect of this drug in these patients.
• Determine nonspecific activation of complement by in vivo determination of C3d complement activation in patients treated with this drug.
• Determine the ex-vivo activity of recombinant MBL in opsonization capacity of patients' sera to yeast and bacteria.
• Determine immunogenicity of this drug in these patients.
• Determine the incidence and duration of fever and breakthrough infections in patients treated with this drug.