---
title: Recombinant Human Mannose-Binding Lectin (MBL) in Treating Young Patients With MBL Deficiency and Fever and Neutropenia
nct_id: NCT00886496
overall_status: WITHDRAWN
phase: PHASE1
sponsor: Enzon Pharmaceuticals, Inc.
study_type: INTERVENTIONAL
primary_condition: Fever, Sweats, and Hot Flashes
countries: United States
canonical_url: "https://parkinsonspathways.com/agent/trials/NCT00886496.md"
clinicaltrials_gov: "https://clinicaltrials.gov/study/NCT00886496"
ct_last_update_post_date: 2012-06-20
last_seen_at: "2026-05-12T06:59:35.685Z"
source: ClinicalTrials.gov (mirrored, no enrichment)
---
# Recombinant Human Mannose-Binding Lectin (MBL) in Treating Young Patients With MBL Deficiency and Fever and Neutropenia

**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

**NCT ID:** [NCT00886496](https://clinicaltrials.gov/study/NCT00886496)

## Key Facts

- **Status:** WITHDRAWN
- **Why Stopped:** No participants enrolled. IND withdrawn.
- **Phase:** PHASE1
- **Study Type:** INTERVENTIONAL
- **Target Enrollment:** 0
- **Lead Sponsor:** Enzon Pharmaceuticals, Inc.
- **Collaborators:** National Cancer Institute (NCI)
- **Conditions:** Fever, Sweats, and Hot Flashes, Infection, Leukemia, Lymphoma, Myelodysplastic Syndromes, Neutropenia, Unspecified Childhood Solid Tumor, Protocol Specific
- **Start Date:** 2006-11
- **Completion Date:** 2011-04
- **CT.gov Last Update:** 2012-06-20

## Brief 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.

## 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

- **Minimum age:** 2 Years
- **Maximum age:** 17 Years
- **Sex:** ALL
- **Healthy Volunteers:** No

```
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
```

## Interventions

- **recombinant human mannose-binding lectin** (BIOLOGICAL)

## Primary Outcomes

- **Toxicity**
- **Pharmacokinetics**
- **Efficacy**

## Locations (3)

- Children's Hospital of Orange County, Orange, California, United States
- Children's National Medical Center, Washington D.C., District of Columbia, United States
- Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States

## Recent Field Changes (last 30 days)

- `status.overallStatus` — added _(2026-05-12)_
- `status.whyStopped` — added _(2026-05-12)_
- `status.completionDate` — added _(2026-05-12)_
- `status.lastUpdatePostDate` — added _(2026-05-12)_
- `design.phases` — added _(2026-05-12)_
- `design.enrollmentCount` — added _(2026-05-12)_
- `eligibility.criteria` — added _(2026-05-12)_
- `eligibility.minAge` — added _(2026-05-12)_
- `eligibility.maxAge` — added _(2026-05-12)_
- `eligibility.sex` — added _(2026-05-12)_
- `outcomes.primary` — added _(2026-05-12)_
- `armsInterventions.interventions` — added _(2026-05-12)_
- `sponsor.lead` — added _(2026-05-12)_
- `sponsor.collaborators` — added _(2026-05-12)_
- `results.hasResults` — added _(2026-05-12)_
- `locations.children's hospital of orange county|orange|california|united states` — added _(2026-05-12)_
- `locations.children's national medical center|washington d.c.|district of columbia|united states` — added _(2026-05-12)_
- `locations.children's hospital of philadelphia|philadelphia|pennsylvania|united states` — added _(2026-05-12)_

---

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