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

NCT ID: NCT00520325

Last Updated: 2012-09-05

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

WITHDRAWN

Clinical Phase

PHASE1

Study Classification

INTERVENTIONAL

Study Start Date

2007-06-30

Study Completion Date

2009-08-31

Brief Summary

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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

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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

Conditions

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Cancer Hematologic Diseases Fever Neutropenia

Keywords

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MBL deficient oncologic hematologic pediatric fever neutropenia

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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0.5 mg/kg

Group Type OTHER

Intravenous recombinant human mannose binding lectin

Intervention Type DRUG

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

Group Type OTHER

Intravenous recombinant human mannose binding lectin

Intervention Type DRUG

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

Interventions

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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

Intervention Type 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

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* 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 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.
Minimum Eligible Age

2 Years

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Enzon Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Enzon Pharmaceuticals, Inc

Principal Investigators

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Thomas Walsh, MD

Role: PRINCIPAL_INVESTIGATOR

NIH/National Cancer Institute

Countries

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United States

Other Identifiers

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NCI # 07-C-0027

Identifier Type: -

Identifier Source: secondary_id

EZN-2232-03

Identifier Type: -

Identifier Source: org_study_id