Recombinant Human Mannose-Binding Lectin (MBL) in Treating Young Patients With MBL Deficiency and 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.

OUTLINE: This is a non-randomized, multicenter, open-label, prospective, cohort study.
Patients are assigned to 1 of 2 treatment groups.

- Group I: Patients receive low-dose recombinant human mannose-binding lectin (MBL) IV
over 1 hour within 72 hours of onset of fever and neutropenia.

- Group II: Patients receive high-dose recombinant human MBL IV over 1 hour within 72
hours of onset of fever and neutropenia.

Patients undergo blood collection periodically during study for pharmacokinetic,
pharmacodynamic, MBL immunogenicity, and opsonization/phagocytosis studies.

After completion of study treatment, patients are followed for 30 days.

PROJECTED ACCRUAL: A total of 48 patients will be accrued for this study.