Finding studies
Finding studies
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Lead
Enzon Pharmaceuticals, Inc.
With
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.
Age
2–17
Sex
ALL
Healthy volunteers
Not accepted
