Cancer, Fever, Hematologic Diseases, Neutropenia
Conditions
Keywords
MBL deficient oncologic hematologic pediatric fever neutropenia
Brief summary
The goal of this clinical research study is to find the dose of EZN-2232 that can be given to MBL deficient pediatric cancer patients undergoing chemotherapy. The pharmacokinetics, pharmacodynamics, and safety of the study drug will also be studied.
Detailed description
MBL deficient patients will be randomized in a ratio of 1:1 to receive a single dose of either 0.5 mg/kg or 1.0 mg/kg of intravenous rhMBL. A total of 24 patients will be treated in each of the rhMBL arms, (12 in the 0.5 mg/kg and 12 in the 1.0 mg/kg. All patients are to receive anti-infectious prophylactic supportive therapy as per institutional standards
Interventions
DRUGIntravenous recombinant human mannose binding lectin
Intravenous(i.v.) administration for one dose. Patients will be randomized to one of three cohorts: 0 mg/kg; 0.5 mg/kg, or 1.0 mg.kg
Sponsors
Enzon Pharmaceuticals, Inc.
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Years to 17 Years
Healthy volunteers
No
Inclusion criteria
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 screening protocol. * 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 mL/min/1.73m2. * 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Determine the safety and tolerability of intravenous rhMBL administered at dosages of 0.5 and 1.0 mg/kg to MBL deficient pediatric hematology/oncology patients during episodes of fever and neutropenia. | 1 month |
Secondary
| Measure | Time frame |
|---|---|
| Determine the PK profile of intravenous rhMBL as a single dose escalation cohort design in MBL deficient pediatric oncology patients during episodes of fever and neutropenia. | 1 month |
Outcome results
None listed