NCT00886496

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

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

• Status: Withdrawn
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: Enzon Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 2 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

RATIONALE: Recombinant human mannose-binding lectin (MBL) may be effective in preventing infection in young patients with fever and neutropenia receiving chemotherapy for blood disease or cancer. PURPOSE: This phase I trial is studying the side effects and best dose of recombinant human mannose-binding lectin in treating young patients with MBL deficiency and fever and neutropenia.

Detailed description

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.

Conditions

• Fever, Sweats, and Hot Flashes
• Infection
• Leukemia
• Lymphoma
• Myelodysplastic Syndromes
• Neutropenia
• Unspecified Childhood Solid Tumor, Protocol Specific

Interventions

Type	Name	Description
BIOLOGICAL	recombinant human mannose-binding lectin

Timeline

• Start date: 2006-11-01
• Completion: 2011-04-01
• First posted: 2009-04-23
• Last updated: 2012-06-20

Locations

3 sites across 1 country: United States