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
Patients must meet all of the following criteria to be eligible for
enrollment into the study:
- Children ages 2 to 17 receiving chemotherapy for hematological/oncological diseases.
- Patients must have documented MBL levels <300 ng/mL in serum as evaluated in the
- Fever defined as an oral temperature equivalent of >100.4°F (38°C).
- Neutropenia defined as an absolute neutrophil count ≤1,000/mm3 with the anticipation
that the counts will fall below 500/mm3.
- Receiving broad spectrum antibiotics for fever and neutropenia.
- Patients with age-adjusted normal serum creatinine OR a creatinine clearance >60
- Informed consent of the patient, parent or legally authorized representative obtained
prior to entry.
- Reliable and willing to make themselves available for the duration of the study and
to abide by the study restrictions.
Exclusion Criteria: Patients meeting any of the following exclusion criteria will not be
eligible for enrollment.
- Patients receiving the preparative regimen for a bone marrow or hematopoietic stem
- Concurrent serious illness in the opinion of the principal investigator that could
potentially interfere with protocol compliance.
- Patient is pregnant, breast feeding or planning a pregnancy during the course of the
- Sexually active male and female patients not using an acceptable barrier method of
contraception or practicing abstinence throughout the study and for at least 30 days
after receiving their last treatment unless the patient is surgically or medically
- Patients with moderate or severe liver disease, as defined by:
AST or ALT >5 times upper limit of normal (ULN) OR Total bilirubin >2.5 times ULN.
- Patients with poor venous access that would preclude intravenous drug delivery or
multiple blood draws.
- Current participation in another clinical study with an investigational agent and/or
use of an investigational drug (not including investigational use of an FDA approved
drug) in the 30 days before scheduled administration of rhMBL.
- Known allergic reactions to MBL or other human plasma products.
- Patients on hemodialysis unable to tolerate the volume of IV fluid on non-dialysis