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

Prothya Biosolutions

Status and phase

Completed

Phase 2

Conditions

Neutropenia

MBL-Deficient

Treatments

Drug: Mannan Binding Lectin

Drug: Mannan Binding Lectin (MBL)

Study type

Interventional

Funder types

Other

Industry

Identifiers

NCT00138736

KB2003.02

Details and patient eligibility

About

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

Full 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.

Enrollment

12 patients

Sex

All

Ages

Under 12 years old

Volunteers

No 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.