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

August 29, 2007 updated by: Prothya Biosolutions

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

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

12

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Netherlands
      • Amsterdam, Netherlands, 1105 AZ
        • Academic Medical Centre
      • Rotterdam, Netherlands, 3015 GD
        • Erasmus Medical Centre

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 10 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Prevention
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: A
MBL until the patient's absolute neutrophil count (ANC) is above 500/microL blood.
Drug: Mannan Binding Lectin (MBL)
Drug: Mannan Binding Lectin

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

Other Names:
  • MBL SSI

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
pharmacokinetics of MBL
Time Frame: until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
until the patient's absolute neutrophil count (ANC) is above 500/uL blood.

Secondary Outcome Measures

Outcome Measure
Time Frame
days of fever
Time Frame: until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
days of hospital admission
Time Frame: until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
use of antibiotics or antifungal medication
Time Frame: until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
number and type of infections
Time Frame: until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
MBL-dependent opsonizing capacity in vitro
Time Frame: until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
safety and incidence of side effects
Time Frame: until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
until the patient's absolute neutrophil count (ANC) is above 500/uL blood.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Prothya Biosolutions

Collaborators

Landsteiner Foundation for Blood Transfusion

Investigators

  • Principal Investigator: T W Kuijpers, MD, PdD, Dept. of Paediatric Immunology, AMC, Amsterdam, The Netherlands
  • Principal Investigator: H N Caron, Dept of Paediatric Oncology, AMC, Amsterdam, The Netherlands

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

April 1, 2004

Study Completion (Actual)

October 1, 2006

Study Registration Dates

First Submitted

August 29, 2005

First Submitted That Met QC Criteria

August 29, 2005

First Posted (Estimate)

August 30, 2005

Study Record Updates

Last Update Posted (Estimate)

August 30, 2007

Last Update Submitted That Met QC Criteria

August 29, 2007

Last Verified

August 1, 2007

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • KB2003.02

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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