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

Lymphoma Clinical Trial

Official title: 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Status Withdrawn

Clinical Trial 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.

Clinical Trial 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. ;

Study Design

Allocation: Non-Randomized, Masking: Open Label, Primary Purpose: Supportive Care

Related Conditions & MeSH terms

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

• NCT number: NCT00886496
• Study type: Interventional
• Source: Enzon Pharmaceuticals, Inc.
• Status: Withdrawn
• Phase: Phase 1
• Start date: November 2006
• Completion date: April 2011