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

Neutropenia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00138736
• Other study ID #: KB2003.02
• Status: Completed
• Phase: Phase 2
• First received: August 29, 2005
• Last updated: August 29, 2007
• Start date: April 2004
• Est. completion date: October 2006

Study information

• Verified date: August 2007
• Source: Sanquin
• Contact: n/a
• Is FDA regulated: No
• Health authority: Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 12
• Est. completion date: October 2006
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A to 12 Years

Eligibility

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 Design

Allocation: Non-Randomized, Endpoint Classification: Pharmacokinetics Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Prevention

Related Conditions & MeSH terms

• MBL-Deficient
• Neutropenia

Intervention

Drug:
• Mannan Binding Lectin (MBL)

• 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

Locations

Country	Name	City	State
Netherlands	Academic Medical Centre	Amsterdam
Netherlands	Erasmus Medical Centre	Rotterdam

Sponsors (2)

Lead Sponsor	Collaborator
Sanquin	Landsteiner Foundation Blood Research (LSBR), Amsterdam

Country where clinical trial is conducted

Netherlands, 

Outcome

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