NCT00627393

Brief Summary

Neutropenia, a condition characterized by an abnormally low number of infection-fighting white blood cells called neutrophils, commonly develops in people who have undergone chemotherapy or hematopoietic stem cell (HSC) transplantation. The severely reduced immunity of those with neutropenia can put them at risk of entry of life-threatening infections, making the implementation of treatments that increase white blood cell numbers important. Several studies have shown that the transfusion of donor granulocytes, a type of white blood cell that includes neutrophils, is effective in promoting the recovery of adequate numbers of granulocytes. However, granulocyte transfusions can cause side effects, and it is not known whether the success of the therapy outweighs the health risks of the side effects. This study will evaluate the safety and effectiveness of granulocyte transfusions in treating people with a bacterial or fungal infection during neutropenia.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
114

participants targeted

Target at P25-P50 for phase_3

Timeline
Completed

Started Apr 2008

Longer than P75 for phase_3

Geographic Reach
1 country

13 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

February 28, 2008

Completed
4 days until next milestone

First Posted

Study publicly available on registry

March 3, 2008

Completed
29 days until next milestone

Study Start

First participant enrolled

April 1, 2008

Completed
5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2013

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2013

Completed
1.6 years until next milestone

Results Posted

Study results publicly available

December 23, 2014

Completed
Last Updated

April 17, 2015

Status Verified

April 1, 2014

Enrollment Period

5 years

First QC Date

February 28, 2008

Results QC Date

December 2, 2014

Last Update Submit

April 16, 2015

Conditions

NeutropeniaInfection

Keywords

Granulocyte Transfusions

Outcome Measures

Primary Outcomes (1)

  • Percentage of Participants Who Are Alive at 42 Days After Treatment and Have Had Microbial Response

    Microbial response was defined as follows: * A negative blood culture test at 42 days after randomization for subjects with fungemia (candidemia or fusariosis) or bacteremia. * Improvement of signs and symptoms of infectious disease (complete or partial response) at 42 days after randomization.

    Measured at Day 42

Secondary Outcomes (12)

  • Alloimmunization, Defined as the Appearance of Anti-human Leukocyte Antigen (HLA) or Antineutrophil Antibodies

    Measured at Days 14 and 42

  • Serious Granulocyte Transfusion Reactions, Including Febrile, Allergic, and Pulmonary Reactions (Transfusion Arm Only)

    Measured within 6 hours after end of transfusion

  • Graft Versus Host Disease Among Recipients of Allogeneic Stem Cell Transplantation

    Measured at Day 42

  • Overall Incidence of Adverse Effects

    Measured through Day 42

  • Fever Resolution

    Measured through Day 42

  • +7 more secondary outcomes

Study Arms (3)

1

EXPERIMENTAL

Participants will receive granulocyte transfusions in addition to standard antimicrobial therapy

Drug: Standard antimicrobial therapyBiological: Granulocyte transfusions

2

ACTIVE COMPARATOR

Participants will receive standard antimicrobial therapy alone

Drug: Standard antimicrobial therapy

3

OTHER

Participants will donate granulocytes after receiving a combination of two drugs, G-CSF and dexamethasone

Drug: G-CSF/dexamethasoneDevice: Apheresis machine

Interventions

Standard antimicrobial therapyDRUG

Antimicrobial therapy is broadly defined as therapy within the standard of care for a particular infection and should be consistent within a given institution. Participants will undergo the recommended therapy for specific infections for 42 days.

12
Granulocyte transfusionsBIOLOGICAL

Participants will receive one granulocyte transfusion per day until one of the following occurs: recovery from neutropenia, life-threatening toxicity, resolution or improvement of infection, or Day 42 after treatment. Granulocyte content of each transfusion is targeted to be at least 4 x 10\^10 per collection (or proportionately less for participants less than 30 kg in weight).

1
G-CSF/dexamethasoneDRUG

Twelve hours before each donation, participants will be injected with G-CSF and will take one dose of dexamethasone by mouth.

Also known as: Neupogen
3
Apheresis machineDEVICE

Participants will undergo a procedure using an apheresis machine for granulocyte collection. The procedure will last 3 to 4 hours and will involve the drawing of blood from each arm, the separation of granulocytes from the red cells and plasma in the machine, and the return of the red cells and plasma to the participants.

3

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Severe neutropenia (Absolute Neutrophil Count \< 500/mm\^3) due to marrow failure caused by underlying disease or therapy
  • Must have one of the following: fungemia; bacteremia; proven or presumptive invasive tissue bacterial infection; or proven, probable, or presumptive invasive fungal infection

You may not qualify if:

  • Unlikely to survive 5 days
  • Evidence that patient will not be neutropenic at least 5 days
  • Previously enrolled in this study

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (13)

University of Iowa Hospitals and Clinics

Iowa City, Iowa, 52242, United States

Location

Johns Hopkins Hospital

Baltimore, Maryland, 21267, United States

Location

Children's Hospital Boston

Boston, Massachusetts, 02115, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Weill Medical College, Cornell University

New York, New York, 10021, United States

Location

Montefiore Medical Center

The Bronx, New York, 10461, United States

Location

University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, 73104, United States

Location

Chlidren's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

University of Pittsburgh Presbyterian and Shadyside

Pittsburgh, Pennsylvania, 15213, United States

Location

University of Washington Medical Center

Seattle, Washington, 98195, United States

Location

University of Wisconsin at Madison

Madison, Wisconsin, 53792, United States

Location

Froedtert Memorial Lutheran Hospital

Milwaukee, Wisconsin, 53201, United States

Location

Related Publications (1)

  • Price TH, Boeckh M, Harrison RW, McCullough J, Ness PM, Strauss RG, Nichols WG, Hamza TH, Cushing MM, King KE, Young JA, Williams E, McFarland J, Holter Chakrabarty J, Sloan SR, Friedman D, Parekh S, Sachais BS, Kiss JE, Assmann SF. Efficacy of transfusion with granulocytes from G-CSF/dexamethasone-treated donors in neutropenic patients with infection. Blood. 2015 Oct 29;126(18):2153-61. doi: 10.1182/blood-2015-05-645986. Epub 2015 Sep 2.

    PMID: 26333778DERIVED

MeSH Terms

Conditions

NeutropeniaInfections

Interventions

Filgrastim

Condition Hierarchy (Ancestors)

AgranulocytosisLeukopeniaCytopeniaHematologic DiseasesHemic and Lymphatic DiseasesLeukocyte Disorders

Intervention Hierarchy (Ancestors)

Granulocyte Colony-Stimulating FactorColony-Stimulating FactorsGlycoproteinsGlycoconjugatesCarbohydratesHematopoietic Cell Growth FactorsCytokinesIntercellular Signaling Peptides and ProteinsPeptidesAmino Acids, Peptides, and ProteinsProteinsBiological Factors

Results Point of Contact

Title
Susan Assmann, PhD
Organization
New England Research Institutes, Inc.
sassmann@neriscience.com
617-972-3048

Study Officials

  • Susan F. Assmann, PhD

    Carelon Research

    PRINCIPAL INVESTIGATOR

  • Jan McFarland, MD

    Froedtert Hospital

    PRINCIPAL INVESTIGATOR

  • Eliot Williams, MD

    University of Wisconsin, Madison

    PRINCIPAL INVESTIGATOR

  • Ellis Neufeld, MD

    Children's Hospital Boston/Brigham and Women's Hospital

    PRINCIPAL INVESTIGATOR

  • James Bussel, MD

    Weill Medical College, Cornell University

    PRINCIPAL INVESTIGATOR

  • Cassandra Josephson, MD

    Emory University

    PRINCIPAL INVESTIGATOR

  • Paul Ness, MD

    Johns Hopkins University

    PRINCIPAL INVESTIGATOR

  • Sherrill Slichter, MD

    University of Washington

    PRINCIPAL INVESTIGATOR

  • Thomas Price, MD

    Bloodworks

    STUDY CHAIR

  • Ronald Strauss, MD

    University of Iowa

    PRINCIPAL INVESTIGATOR

  • Jeffrey McCullough, MD

    University of Minnesota

    PRINCIPAL INVESTIGATOR

  • James George, MD

    University of Oklahoma

    PRINCIPAL INVESTIGATOR

  • Bruce Sachais, MD, PHD

    University of Pennsylvania

    PRINCIPAL INVESTIGATOR

  • David Friedman, MD

    Children's Hospital of Philadelphia

    PRINCIPAL INVESTIGATOR

  • Darrell Triulzi, MD

    University of Pittsburgh Presbyterian and Shadyside/Children's Hospital Pittsburgh

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
SINGLE
Who Masked
OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 28, 2008

First Posted

March 3, 2008

Study Start

April 1, 2008

Primary Completion

April 1, 2013

Study Completion

May 1, 2013

Last Updated

April 17, 2015

Results First Posted

December 23, 2014

Record last verified: 2014-04

Locations

US(13)