Recombinant Human Mannose-Binding Lectin (MBL) in Treating Young Patients With MBL Deficiency and Fever and Neutropenia
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.
|Fever, Sweats, and Hot Flashes Infection Leukemia Lymphoma Myelodysplastic Syndromes Neutropenia Unspecified Childhood Solid Tumor, Protocol Specific||Biological: recombinant human mannose-binding lectin||Phase 1|
|Study Design:||Allocation: Non-Randomized
Masking: None (Open Label)
Primary Purpose: Supportive Care
|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|
|Study Start Date:||November 2006|
|Study Completion Date:||April 2011|
- 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.
- 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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00886496
|United States, California|
|Children's Hospital of Orange County|
|Orange, California, United States, 92868|
|United States, District of Columbia|
|Children's National Medical Center|
|Washington, District of Columbia, United States, 20010-2970|
|United States, Pennsylvania|
|Children's Hospital of Philadelphia|
|Philadelphia, Pennsylvania, United States, 19104|
|Principal Investigator:||Thomas J. Walsh, MD||National Cancer Institute (NCI)|