A Study of Safety, Tolerability, and Pk of rhMBL in Pediatric Hematology/Oncology Pts With Fever and Neutropenia - Full Text View

Sponsored by:	Enzon Pharmaceuticals, Inc.
Information provided by:	Enzon Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier:	NCT00520325

Purpose

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.

Condition	Intervention	Phase
Cancer Hematologic Diseases Fever Neutropenia	Drug: Intravenous recombinant human mannose binding lectin	Phase I

MedlinePlus related topics: Cancer Fever

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Dose Comparison, Parallel Assignment, Safety Study
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

Further study details as provided by Enzon Pharmaceuticals, Inc.:

Primary Outcome Measures:

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. [ Time Frame: 1 month ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

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. [ Time Frame: 1 month ] [ Designated as safety issue: No ]

Estimated Enrollment:	24
Study Start Date:	June 2007
Estimated Study Completion Date:	August 2009
Estimated Primary Completion Date:	June 2009 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
0.5 mg/kg	Drug: Intravenous 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
1.0 mg/kg	Drug: Intravenous 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

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

Eligibility

Ages Eligible for Study:	2 Years to 17 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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 exclusion criteria will not be eligible for enrollment.

Patients receiving the preparative regimen for a bone marrow or hematopoietic stem cell transplantation.
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 study.
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 sterile.
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 days.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00520325

Locations

United States, California
Children's Hospital Orange County
Orange, California, United States, 92686-3874

Sponsors and Collaborators

Enzon Pharmaceuticals, Inc.

Investigators

Principal Investigator:

Thomas Walsh, MD

NIH/National Cancer Institute

More Information

No publications provided

Responsible Party:	Enzon Pharmaceuticals, Inc ( Nazish Huq )
Study ID Numbers:	EZN-2232-03, NCI # 07-C-0027
Study First Received:	August 23, 2007
Last Updated:	February 18, 2009
ClinicalTrials.gov Identifier:	NCT00520325 History of Changes
Health Authority:	United States: Food and Drug Administration

Keywords provided by Enzon Pharmaceuticals, Inc.:

MBL deficient oncologic hematologic pediatric fever neutropenia

Study placed in the following topic categories:

Fever
Signs and Symptoms
Neutropenia
Hematologic Diseases

Agranulocytosis
Leukocyte Disorders
Leukopenia
Granulocytopenia

Additional relevant MeSH terms:

Fever
Signs and Symptoms
Neutropenia
Hematologic Diseases

Agranulocytosis
Leukocyte Disorders
Body Temperature Changes
Leukopenia

ClinicalTrials.gov processed this record on May 07, 2009