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

Primary Purpose

Cancer, Hematologic Diseases, Fever

Status

Withdrawn

Phase

Phase 1

Locations

Study Type

Interventional

Intervention

Intravenous recombinant human mannose binding lectin

About this trial

This is an interventional treatment trial for Cancer focused on measuring MBL deficient oncologic hematologic pediatric fever neutropenia

Eligibility Criteria

2 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm Type

Other

Arm Label

0.5 mg/kg

1.0 mg/kg

Arm Description

Outcomes

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.

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.

Full Information

NCT ID

NCT00520325

First Posted

August 23, 2007

Last Updated

September 4, 2012

Sponsor

Enzon Pharmaceuticals, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT00520325

Brief Title

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

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 Type

Interventional

2. Study Status

Record Verification Date

February 2009

Overall Recruitment Status

Withdrawn

Why Stopped

Sponsor decision to terminate the study

Study Start Date

June 2007 (undefined)

Primary Completion Date

June 2009 (Actual)

Study Completion Date

August 2009 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor

Enzon Pharmaceuticals, Inc.

4. Oversight

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Cancer, Hematologic Diseases, Fever, Neutropenia

Keywords

MBL deficient oncologic hematologic pediatric fever neutropenia

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

0 (Actual)

8. Arms, Groups, and Interventions

Arm Title

0.5 mg/kg

Arm Type

Other

Arm Title

1.0 mg/kg

Arm Type

Other

Intervention Type

Drug

Intervention Name(s)

Intravenous recombinant human mannose binding lectin

Intervention Description

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

Intervention Name(s)

Intravenous recombinant human mannose binding lectin

Intervention Description

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

Primary Outcome Measure Information:

Title

Time Frame

1 month

Secondary Outcome Measure Information:

Title

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

10. Eligibility

Sex

All

Minimum Age & Unit of Time

2 Years

Maximum Age & Unit of Time

17 Years

Accepts Healthy Volunteers

Eligibility 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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Thomas Walsh, MD

Organizational Affiliation

NIH/National Cancer Institute

Official's Role

Principal Investigator

12. IPD Sharing Statement

Learn more about this trial

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

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