Protocol Number: 07-C-0027

Title:

A Multi-Center Study of the Safety, Tolerability, Pharmacokinetics, and Dose Escalation of Intravenous Recombinant Human Mannose-Binding-Lectin (rhMBL) in MBL Deficient Pediatric Hematology/Oncology Patients with Fever and Neutropenia

Number:

07-C-0027

Summary:

Background:

-Infections are a major cause of sickness and death in children with cancer.

-Studies show that a protein called mannose-binding lectin (MBL) is important in immunity and that changes in blood levels of MBL are associated with serious infectious complications when the immune system is stressed.

-A new genetically engineered MBL protein called rhMBL may be useful in treating some patients with low MBL levels.

Objectives:

-To determine the pharmacokinetics, safety and tolerability of rhMBL in children with cancer and examine how the body handles the drug.

-To see if rhMBL helps to control infections in children with cancer.

Eligibility:

-Children ages 2 to 17 who are receiving cancer chemotherapy, who have low MBL levels and low neutrophil (type of white blood cell) count and fever (temperature over 100.4 F).

Design:

-Patients receive a one-time dose of recombinant rhMBL through a central line or IV within three days after developing a fever and low neutrophil count.

-The first group of children entering the study receives a dose of 0.5 mg/kg; the second group receives a dose of 1 mg/kg. Both dosages are infused over 60 minutes.

-Patients have small amounts of blood drawn over the duration of the 4-week study to measure the levels of MBL in the blood, look for possible changes in the immune system and check for any side effects of rhMBL.

-Patients also receive standard antibiotics used to treat and prevent infections during fever and neutropenia occurring after chemotherapy.

Sponsoring Institute:

National Cancer Institute (NCI)

Recruitment Detail

Type: Clinical hold/Recruitment or enrollment suspended

Gender: Male & Female

Referral Letter Required: Yes

Population Exclusion(s): None

Eligibility Criteria: This study is not currently recruiting new subjects. If you have questions about participating in a study, please contact the Patient Recruitment and Public Liaison Office, CC.

Special Instructions:

Currently Not Provided

Keyword(s):

Infection

Immunogenicity

C4 Deposition

Host Defense

Innate Immunity

Recruitment Keyword(s):

Neutropenia

Fever

Infection

Cancer

Pediatrics

Condition(s):

Fever

Neutropenia

Investigational Drug(s):

Recombinant Human Mannose Binding Lectin

Investigational Device(s):

None

Interventions:

Drug: Recombinant Human Mannose Binding Lectin

Supporting Site:

Enzon Pharmaceuticals

Contact(s):

This study is not currently recruiting new subjects. If you have questions about participating in a study, please contact the Patient Recruitment and Public Liaison Office, CC.

Citation(s):

Babula O, Danielsson I, Sjoberg I, Ledger WJ, Witkin SS. Altered distribution of mannose-binding lectin alleles at exon I codon 54 in women with vulvar vestibulitis syndrome . Am J Obstet Gynecol. 2004 Sep;191(3):762-6

Fidler KJ, Wilson P, Davies JC, Turner MW, Peters MJ, Klein NJ. Increased incidence and severity of the systemic inflammatory response syndrome in patients deficient in mannose-binding lectin. Intensive Care Med. 2004 Jul;30(7):1438-45. Epub 2004 May 4.

Garred P, Nielsen MA, Kurtzhals JA, Malhotra R, Madsen HO, Goka BQ, Akanmori BD, Sim RB, Hviid L. Mannose-binding lectin is a disease modifier in clinical malaria and may function as opsonin for Plasmodium falciparum-infected erythrocytes. Infect Immun. 2003 Sep;71(9):5245-53

• Questions about participating in a study, please contact the Patient Recruitment and Public Liaison Office, CC.
• Technical questions regarding the Clinical Center web site, please contact the Department Clinical Research Informatics, CC.

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