Recombinant Human Mannose-Binding Lectin (MBL) in Treating Young Patients With MBL Deficiency and Fever and Neutropenia
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|ClinicalTrials.gov Identifier: NCT00886496|
Recruitment Status : Withdrawn (No participants enrolled. IND withdrawn.)
First Posted : April 23, 2009
Last Update Posted : June 20, 2012
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.
|Condition or disease||Intervention/treatment||Phase|
|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|
- 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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||0 participants|
|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|
|Actual Study Completion Date :||April 2011|
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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)|