Clinical trial NCT00138736: 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
Overall status: COMPLETED
Phase: PHASE2
Conditions: MBL-Deficient
Neutropenia
Keywords: MBL-deficient
chemotherapy
neutropenia
chemotherapy-induced neutropenia
Lead sponsor: Prothya Biosolutions
Sponsor class: INDUSTRY
Collaborators: name: Landsteiner Foundation for Blood Transfusion
First posted: 2005-08-30
Enrollment: 12
Sex: ALL
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: PREVENTION

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.

Eligibility criteria: 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.

Primary outcomes:
measure: pharmacokinetics of MBL
timeFrame: until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
Secondary outcomes:
measure: days of fever
timeFrame: until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
measure: days of hospital admission
timeFrame: until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
measure: use of antibiotics or antifungal medication
timeFrame: until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
measure: number and type of infections
timeFrame: until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
measure: MBL-dependent opsonizing capacity in vitro
timeFrame: until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
measure: safety and incidence of side effects
timeFrame: until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
Other outcomes:
Locations:
city: Amsterdam
country: Netherlands
facility: Academic Medical Centre
city: Rotterdam
country: Netherlands
facility: Erasmus Medical Centre
Study officials:
affiliation: Dept. of Paediatric Immunology, AMC, Amsterdam, The Netherlands
name: T W Kuijpers, MD, PdD
role: PRINCIPAL_INVESTIGATOR
affiliation: Dept of Paediatric Oncology, AMC, Amsterdam, The Netherlands
name: H N Caron
role: PRINCIPAL_INVESTIGATOR
References: