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Study on Mannan Binding Lectin (MBL) Substitution in MBL-Deficient Children With Chemotherapy-Induced Neutropenia

Primary Purpose

MBL-Deficient, Neutropenia

Status

Completed

Phase

Phase 2

Locations

Netherlands

Study Type

Interventional

Intervention

Mannan Binding Lectin (MBL)

Mannan Binding Lectin

About this trial

This is an interventional prevention trial for MBL-Deficient focused on measuring MBL-deficient, chemotherapy, neutropenia, chemotherapy-induced neutropenia

Eligibility Criteria

undefined - 12 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Children ages 0 - 12 years, during chemotherapy, and expected to become neutropenic MBL deficiency by genotype or phenotype (< 100 ng/ml) Informed consent and assent of patient and/or legal representative Exclusion Criteria: Inability or unwillingness to comply with the protocol or likely inability to complete the study period Known allergic reactions to MBL and other human plasma products Participation in other investigational drug studies within the last month Clinically relevant abnormalities in: serum immunoglobulins IgG, IgA, IgM; blood counts; complement factors measured by AP50, CH50; urine protein and cell counts; serum creatinine and liver enzymes, as routinely determined for regular patient care.

Sites / Locations

Academic Medical Centre
Erasmus Medical Centre

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Arm Description

MBL until the patient's absolute neutrophil count (ANC) is above 500/microL blood.

Outcomes

Primary Outcome Measures

pharmacokinetics of MBL

Secondary Outcome Measures

days of fever

days of hospital admission

use of antibiotics or antifungal medication

number and type of infections

MBL-dependent opsonizing capacity in vitro

safety and incidence of side effects

Full Information

NCT ID

NCT00138736

First Posted

August 29, 2005

Last Updated

August 29, 2007

Sponsor

Prothya Biosolutions

Collaborators

Landsteiner Foundation for Blood Transfusion

1. Study Identification

Unique Protocol Identification Number

NCT00138736

Brief Title

Study on Mannan Binding Lectin (MBL) Substitution in MBL-Deficient Children With Chemotherapy-Induced Neutropenia

Official Title

Phase II Study on Mannan Binding Lectin (MBL) Substitution in MBL-Deficient Children With Chemotherapy-Induced Neutropenia

Study Type

Interventional

2. Study Status

Record Verification Date

August 2007

Overall Recruitment Status

Completed

Study Start Date

April 2004 (undefined)

Primary Completion Date

undefined (undefined)

Study Completion Date

October 2006 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor

Prothya Biosolutions

Collaborators

Landsteiner Foundation for Blood Transfusion

4. Oversight

Data Monitoring Committee

5. Study Description

Brief Summary

The pharmacokinetics, and clinical and biological effects of MBL replacement therapy in MBL-deficient children during chemotherapy-induced neutropenia were studied.

Detailed Description

Mannan Binding Lectin (MBL) is a member of the lectin pathway of the complement system and plays an important role in the innate immune system. MBL replacement in MBL-deficient children with chemotherapy-induced neutropenia represents a new approach to lower the risk of febrile episodes, of hospital admission, of prolonged use of intravenous antibiotics and of severe infections. The aim of the Phase II study is to find evidence for the correct prediction of plasma levels of MBL necessary for clinical effects and biological efficacy, to confirm the dosage regimen needed to reach the required MBL plasma levels, and reconfirm the safety and lack of side-effects.

6. Conditions and Keywords

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

MBL-Deficient, Neutropenia

Keywords

MBL-deficient, chemotherapy, neutropenia, chemotherapy-induced neutropenia

7. Study Design

Primary Purpose

Prevention

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

12 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Arm Type

Experimental

Arm Description

MBL until the patient's absolute neutrophil count (ANC) is above 500/microL blood.

Intervention Type

Drug

Intervention Name(s)

Mannan Binding Lectin (MBL)

Intervention Type

Drug

Intervention Name(s)

Mannan Binding Lectin

Other Intervention Name(s)

MBL SSI

Intervention Description

MBL dose at a twice weekly dose interval (3 or 4 days): 0.2 mg/kg, for a 3-day interval; 0.3 mg/kg, for a 4-day interval

Primary Outcome Measure Information:

Title

pharmacokinetics of MBL

Time Frame