CClinicalTrials.gg
Sign In
WithdrawnNCT00886496Updated Jun 20, 2012

Recombinant Human Mannose-Binding Lectin (MBL) in Treating Young Patients With MBL Deficiency and Fever and Neutropenia

A Phase 1 interventional study of recombinant human mannose-binding lectin in Fever, Sweats, and Hot Flashes, Infection and Leukemia, sponsored by Enzon Pharmaceuticals, Inc.. Withdrawn at 3 sites in United States. Open to participants aged 2 Years to 17 Years. Per ClinicalTrials.gov, last updated 2012-06-20.

Sponsored by Enzon Pharmaceuticals, Inc. · Phase 1, Interventional, and Supportive care

Why this study was withdrawn
No participants enrolled. IND withdrawn.
Phase
Phase 1
Study type
Interventional
Enrollment
0
Allocation
Non-randomized
Ages
2 Years to 17 Years
Sex
All
01

Study summary

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.

Read the 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.

02

Conditions studied

  • Fever, Sweats, and Hot Flashes
  • Infection
  • Leukemia
  • Lymphoma
  • Myelodysplastic Syndromes
  • Neutropenia
  • Unspecified Childhood Solid Tumor, Protocol Specific

Keywords

  • 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
  • childhood acute myeloid leukemia in remission
  • recurrent childhood acute myeloid leukemia
  • untreated childhood acute myeloid leukemia and other myeloid malignancies
  • childhood grade III lymphomatoid granulomatosis
  • childhood nasal type extranodal NK/T-cell lymphoma
  • recurrent childhood grade III lymphomatoid granulomatosis
  • childhood diffuse large cell lymphoma
  • childhood immunoblastic large cell lymphoma
  • recurrent childhood large cell lymphoma
  • stage I childhood large cell lymphoma
  • stage II childhood large cell lymphoma
  • stage III and IV childhood large cell lymphoma
  • stage III childhood large cell lymphoma
  • stage IV childhood large cell lymphoma
03

In context

Lymphoma

5,578 studies on the registry are indexed under Lymphoma; 825 are open to participants now.

Browse Lymphoma studies →

Lead sponsor

Enzon Pharmaceuticals, Inc. is the lead sponsor of 14 studies on the registry; none are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
2 Years to 17 Years
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility 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
05

Study design

Phase
Phase 1
Primary purpose
Supportive care
Allocation
Non-randomized
Masking
None (open label)
Enrollment
0 participants (actual)

Interventions

  • Biologicalrecombinant human mannose-binding lectin
06

What researchers measure

Primary outcomes

  1. Toxicity

  2. Pharmacokinetics

  3. Efficacy

07

Study locations

3 sites
  • Children's Hospital of Orange County
    Orange, California 92868, United States
  • Children's National Medical Center
    Washington, District of Columbia 20010-2970, United States
  • Children's Hospital of Philadelphia
    Philadelphia, Pennsylvania 19104, United States
08

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jun 20, 2012, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
09

Registry details

Key details

Study ID
NCT00886496
Lead sponsor
Enzon Pharmaceuticals, Inc.
Collaborators
National Cancer Institute (NCI)
First posted
Apr 23, 2009
Start date
Nov 2006
Completion
Apr 2011
Last update
Jun 20, 2012

Study contacts

Thomas J. Walsh, MD
principal investigator · National Cancer Institute (NCI)
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is withdrawn, as verified in Aug 2007. You cannot join it, but the record below documents what was studied.

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Other studies in this condition

Lymphoma→

Other studies from this sponsor

Enzon Pharmaceuticals, Inc.

Start the discussion