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

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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

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

Detailed Summary:

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.


Sponsor: Sanquin

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

Original Primary Outcome: pharmacokinetics of MBL

Current Secondary Outcome:

  • days of fever [ Time Frame: 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. ]
  • use of antibiotics or antifungal medication [ Time Frame: 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. ]
  • MBL-dependent opsonizing capacity in vitro [ Time Frame: 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. ]


Original Secondary Outcome:

  • 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


Information By: Sanquin

Dates:
Date Received: August 29, 2005
Date Started: April 2004
Date Completion:
Last Updated: August 29, 2007
Last Verified: August 2007