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Fever, Sweats, and Hot FlashesPhase 1NCT00886496

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

A Phase 1 study testing recombinant human mannose-binding lectin in people with Fever, Sweats, and Hot Flashes and Infection, run by Enzon Pharmaceuticals, Inc. at 3 sites in United States.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 2 to 17 years
What is being tested?
recombinant human mannose-binding lectin
How many people
0 participants
Where
3 sitesUnited States
Study dates
Nov 2006to Apr 2011
Phase
Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 2 to 17 years
  • All sexes
  • No healthy volunteers
  • Child

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

Trial locations and contacts

3 sites in 1 country.

United States3 sites

  • Children's Hospital of Orange County

    Orange, California, 92868

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20010-2970

Other trials in United States

About this study

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

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.

Being tested
recombinant human mannose-binding lectin
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
Medical subject headings
Fever, Hot Flashes, Infections, Leukemia, Lymphoma, Myelodysplastic Syndromes, Neutropenia, Burkitt Lymphoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • recombinant human mannose-binding lectinBiological

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Toxicity

  2. Pharmacokinetics

  3. Efficacy

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted23 Apr 2009 (estimated)
  2. Study startNov 2006
  3. Study completionApr 2011

Why it stopped: No participants enrolled. IND withdrawn.

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Supportive care
Allocation
Non-randomised
Masking
None (open label)
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00886496
Sponsor's protocol ID
CDR0000523819
Other IDs
ENZON-EZN-2232-03, NCI-07-C-0027
Status last verified
Aug 2007
First submitted
21 Apr 2009
Last updated
20 Jun 2012
Results posted
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing recombinant human mannose-binding lectin in people with Fever, Sweats, and Hot Flashes and Infection, run by Enzon Pharmaceuticals, Inc. at 3 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: withdrawn, as of the sponsor's last update on 20 Jun 2012. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was withdrawn before enrolling participants, so there are no results.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Toxicity. The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00886496.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Jun 2012.

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