Trial record 1 of 4 for:    MANNOSE-BINDING PROTEIN DEFICIENCY
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Recombinant Human Mannose-Binding Lectin (MBL) in Treating Young Patients With MBL Deficiency and Fever and Neutropenia

This study has been withdrawn prior to enrollment.
(No participants enrolled. IND withdrawn.)
Sponsor:
Collaborator:
Information provided by:
Enzon Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier:
NCT00886496
First received: April 21, 2009
Last updated: June 19, 2012
Last verified: August 2007
  Purpose

RATIONALE: Recombinant human mannose-binding lectin (MBL) may be effective in preventing infection in young patients with fever and neutropenia receiving chemotherapy for blood disease or cancer.

PURPOSE: This phase I trial is studying the side effects and best dose of recombinant human mannose-binding lectin in treating young patients with MBL deficiency and fever and neutropenia.


Condition Intervention Phase
Fever, Sweats, and Hot Flashes
Infection
Leukemia
Lymphoma
Myelodysplastic Syndromes
Neutropenia
Unspecified Childhood Solid Tumor, Protocol Specific
Biological: recombinant human mannose-binding lectin
Phase 1

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Masking: Open Label
Primary Purpose: Supportive Care
Official 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

Resource links provided by NLM:


Further study details as provided by Enzon Pharmaceuticals, Inc.:

Primary Outcome Measures:
  • Toxicity
  • Pharmacokinetics
  • Efficacy

Enrollment: 0
Study Start Date: November 2006
Study Completion Date: April 2011
Detailed Description:

OBJECTIVES:

Primary

  • Determine the safety and tolerability of recombinant human mannose-binding lectin (MBL) in pediatric patients with MBL deficiency and fever and neutropenia who are undergoing cytotoxic chemotherapy for hematological/oncological disease.
  • Determine the pharmacokinetics of this drug in these patients.

Secondary

  • Determine the pharmacodynamic effect of this drug in these patients.
  • Determine nonspecific activation of complement by in vivo determination of C3d complement activation in patients treated with this drug.
  • Determine the ex-vivo activity of recombinant MBL in opsonization capacity of patients' sera to yeast and bacteria.
  • Determine immunogenicity of this drug in these patients.
  • Determine the incidence and duration of fever and breakthrough infections in patients treated with this drug.

OUTLINE: This is a non-randomized, multicenter, open-label, prospective, cohort study. Patients are assigned to 1 of 2 treatment groups.

  • Group I: Patients receive low-dose recombinant human mannose-binding lectin (MBL) IV over 1 hour within 72 hours of onset of fever and neutropenia.
  • Group II: Patients receive high-dose recombinant human MBL IV over 1 hour within 72 hours of onset of fever and neutropenia.

Patients undergo blood collection periodically during study for pharmacokinetic, pharmacodynamic, MBL immunogenicity, and opsonization/phagocytosis studies.

After completion of study treatment, patients are followed for 30 days.

PROJECTED ACCRUAL: A total of 48 patients will be accrued for this study.

  Eligibility

Ages Eligible for Study:   2 Years to 17 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

DISEASE CHARACTERISTICS:

  • Undergoing cytologic chemotherapy for hematological/oncological disease
  • Must meet all of the following criteria:

    • Documented mannose-binding lectin (MBL) levels < 300 ng/mm³ within the past week
    • Fever (oral temperature > 100.4° F)
    • Neutropenia, defined as absolute neutrophil count ≤ 1,000/mm³ with the anticipation that the counts will fall below 500/mm^3
    • Receiving broad spectrum antibiotic therapy for fever and neutropenia

PATIENT CHARACTERISTICS:

  • No serious illness, in the opinion of the principal investigator, that would preclude study compliance
  • No known allergic reactions to mannose-binding lectin or other human plasma products
  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective barrier method contraception during and for ≥ 30 days after completion of study treatment
  • AST and ALT ≤ 5 times upper limit of normal (ULN)
  • Bilirubin ≤ 2.5 times ULN
  • Creatinine clearance > 60 mL/min OR creatinine based on age as follows:

    • No more than 0.8 mg/dL (for patients 5 years of age and under)
    • No more than 1.0 mg/dL (for patients 6-9 years of age)
    • No more than 1.2 mg/dL (for patients 10-12 years of age)
    • No more than 1.4 mg/dL (for patients over 13 years of age [female])
    • No more than 1.5 mg/dL (for patients 13-15 years of age [male])
    • No more than 1.7 mg/dL (for patients of 16 years of age [male])
  • No poor venous access that would preclude IV drug delivery or multiple blood draws
  • Patients on hemodialysis must be able to tolerate IV fluid on non-dialysis days

PRIOR CONCURRENT THERAPY:

  • See Disease Characteristics
  • More than 30 days since prior investigational agents

    • Investigational use of an FDA-approved drug allowed
  • No concurrent preparative regimen for a bone marrow or hematopoietic stem cell transplantation
  • No concurrent participation in another clinical trial with an investigational agent
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00886496

Locations
United States, California
Children's Hospital of Orange County
Orange, California, United States, 92868
United States, District of Columbia
Children's National Medical Center
Washington, District of Columbia, United States, 20010-2970
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Sponsors and Collaborators
Enzon Pharmaceuticals, Inc.
Investigators
Principal Investigator: Thomas J. Walsh, MD National Cancer Institute (NCI)
  More Information

No publications provided

ClinicalTrials.gov Identifier: NCT00886496     History of Changes
Other Study ID Numbers: CDR0000523819, ENZON-EZN-2232-03, NCI-07-C-0027
Study First Received: April 21, 2009
Last Updated: June 19, 2012
Health Authority: United States: Food and Drug Administration

Keywords provided by Enzon Pharmaceuticals, Inc.:
unspecified childhood solid tumor, protocol specific
fever, sweats, and hot flashes
neutropenia
infection
recurrent childhood small noncleaved cell lymphoma
stage I and II childhood small noncleaved cell lymphoma
stage I childhood small noncleaved cell lymphoma
stage II childhood small noncleaved cell lymphoma
stage III and IV childhood small noncleaved cell lymphoma
stage III childhood small noncleaved cell lymphoma
stage IV childhood small noncleaved cell lymphoma
recurrent childhood lymphoblastic lymphoma
stage I and II childhood lymphoblastic lymphoma
stage I childhood lymphoblastic lymphoma
stage II childhood lymphoblastic lymphoma
stage III and IV childhood lymphoblastic lymphoma
stage III childhood lymphoblastic lymphoma
stage IV childhood lymphoblastic lymphoma
childhood Burkitt lymphoma
recurrent/refractory childhood Hodgkin lymphoma
stage I childhood Hodgkin lymphoma
stage II childhood Hodgkin lymphoma
stage III childhood Hodgkin lymphoma
stage IV childhood Hodgkin lymphoma
childhood acute lymphoblastic leukemia in remission
recurrent childhood acute lymphoblastic leukemia
untreated childhood acute lymphoblastic leukemia
de novo myelodysplastic syndromes
previously treated myelodysplastic syndromes
secondary myelodysplastic syndromes

Additional relevant MeSH terms:
Infection
Myelodysplastic Syndromes
Preleukemia
Lymphoma
Leukemia
Syndrome
Fever
Neutropenia
Hot Flashes
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Neoplasms
Neoplasms by Histologic Type
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Disease
Pathologic Processes
Body Temperature Changes
Signs and Symptoms
Agranulocytosis
Leukopenia
Leukocyte Disorders

ClinicalTrials.gov processed this record on September 30, 2014