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

Phase 1

Withdrawn

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

• Registration Number: NCT00886496
• Lead Sponsor: Enzon Pharmaceuticals, Inc.

Brief 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.

Study Timeline

• Study Start: 2006-11
• Status Verified: 2007-08
• Study First Submitted: 2009-04-21
• Study First Submitted QC: 2009-04-21
• Study First Posted: 2009-04-23
• Study Completion: 2011-04
• Last Update Submitted: 2012-06-19
• Last Update Posted: 2012-06-20

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Efficacy
Pharmacokinetics
Toxicity

Trial Locations

Locations (3)

Children's Hospital of Orange County; 🇺🇸 Orange, California, United States

Children's National Medical Center; 🇺🇸 Washington, District of Columbia, United States

Children's Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States