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This study has been withdrawn prior to enrollment.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 IND withdrawn.)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:
Enzon Pharmaceuticals, Inc.
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
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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:
Genetics Home Reference related topics:
cyclic neutropenia
familial acute myeloid leukemia with mutated CEBPA
mannose-binding lectin deficiency
MedlinePlus related topics:
Acute Myeloid Leukemia
Cancer
Chronic Lymphocytic Leukemia
Fever
Hodgkin Disease
Leukemia
Lymphoma
Myelodysplastic Syndromes
Sweat
U.S. FDA Resources
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 |
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
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
Additional 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:
|
Fever Leukemia Lymphoma Myelodysplastic Syndromes Preleukemia Neutropenia Lymphoma, Non-Hodgkin Hot Flashes Body Temperature Changes Signs and Symptoms Neoplasms by Histologic Type |
Neoplasms Lymphoproliferative Disorders Lymphatic Diseases Immunoproliferative Disorders Immune System Diseases Bone Marrow Diseases Hematologic Diseases Precancerous Conditions Agranulocytosis Leukopenia Leukocyte Disorders |
ClinicalTrials.gov processed this record on May 22, 2013