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Granulocyte Colony Stimulating Factor (G-CSF) for Bone Marrow Transplant (BMT)

Primary Purpose

Hematologic Diseases, Hematologic Malignancies

Status
Completed
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
Granulocyte Colony Stimulating Factor
Sponsored by
Emory University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hematologic Diseases focused on measuring G-CSF, Bone Marrow, Hematologic malignancies, Non-malignancies, Matched sibling donor, Allogeneic BMT

Eligibility Criteria

undefined - 55 Years (Child, Adult)All SexesAccepts Healthy Volunteers

Inclusion Criteria: Patients with hematologic malignancies and non-malignancies who are candidates for matched sibling donor allogeneic bone marrow transplantation are eligible for this study. Patients who are under 55 years of age. Patients who have a life expectancy of at least 12 weeks and a performance status of at least 2 Zubrod or 70% Karnofsky status prior to transplantation. Patients who are acceptable candidates for marrow transplantation based on their pre-BMT evaluation. Patients who have available histocompatible siblings who have been medically approved as marrow donors. Patients who sign informed consent for the protocol approved by the Institutional Review Board of Emory University/Children's Healthcare of Atlanta. Donors must be 5 years of age or older, and have completed routine donor evaluations and signed (by parent or legal guardian) informed consent for the protocol approved by the Institutional Review Board of Emory University/Children's Healthcare of Atlanta. Exclusion Criteria: Patients will not be excluded based on sex, racial, or ethnic background. Patients will be excluded if they demonstrate significant functional deficits in major organs, which would obviously interfere with a successful outcome following bone marrow transplant utilizing the following guidelines: Evidence of active, deep seated, life-threatening infections for which there is no known effective therapy (e.g. certain fungal species, HIV, etc.). Patients with hemoglobinopathy (e.g. sickle cell disease and thalassemia) will not be eligible for this protocol. However, filgrastim mobilization, large volume apheresis, processing, and cryopreservation appears to be safe in donors with sickle cell trait. Patients have had greater than two leukemic episodes, active central nervous system (CNS) and/or leukemic disease and blast crisis in chronic myelogenous leukemia (CML) patients. Patients will be excluded if they are women of childbearing potential who are currently pregnant (beta-hCG+) or who are not practicing adequate contraception. Patients who have had previous stem cell transplant will be excluded. Donors will be excluded if they are sensitive to E. coli-derived protein.

Sites / Locations

  • Children's Healthcare of Atlanta/Emory University

Arms of the Study

Arm 1

Arm Type

Other

Arm Label

single arm

Arm Description

Outcomes

Primary Outcome Measures

Numbers of Participants With Disease-free Survival.
Evaluate the numbers of participants with disease-free survival

Secondary Outcome Measures

Hospital Length of Stay
hospital length of stay of each patient enrolled, an average of 5 weeks.

Full Information

First Posted
October 3, 2005
Last Updated
August 19, 2014
Sponsor
Emory University
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1. Study Identification

Unique Protocol Identification Number
NCT00231309
Brief Title
Granulocyte Colony Stimulating Factor (G-CSF) for Bone Marrow Transplant (BMT)
Official Title
Pilot Study of Using Granulocyte Colony Stimulating Factor-Primed Bone Marrow in Histocompatible Sibling Allogeneic Bone Marrow Transplantation for Patients With Hematologic Malignancies and Non-Malignancies
Study Type
Interventional

2. Study Status

Record Verification Date
August 2014
Overall Recruitment Status
Completed
Study Start Date
July 2003 (undefined)
Primary Completion Date
November 2011 (Actual)
Study Completion Date
November 2011 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Emory University

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The major purpose of this study is to evaluate the curative potential of white cell growth hormone (G-CSF)-stimulated bone marrow cells in allogeneic bone marrow transplants. Patients with cancers or blood diseases, who have poor potential for a cure with standard treatment, will be able to participate in the study. Donors will receive the white cell growth hormone (G-CSF) as a shot (injection) in their arm once a day for three days before they donate their bone marrow cells. Total body irradiation and/or chemotherapy will be given first to prepare the patient's body for the infusion of new bone marrow cells from the donor. Two medicines (cyclosporine and methotrexate) will be used to prevent the new bone marrow cells (graft) from attacking the patient's body (host) (graft-versus-host disease; GVHD). Certain safety checkpoints were built into the study if unwanted/unexpected events were to occur. If the outcomes appear better than could be expected, this will provide a bridge to extend this current approach for other innovative therapies.
Detailed Description
This study is a single-arm, non-randomized trial. Patients meeting the criteria for this study will be entered sequentially until completion or closure of the study. Early stopping rules will be employed to ascertain whether an unacceptable rate of toxicity (non-engraftment, and/or acute GvHD) occurs. Patients will be prepared for transplant through the administration of one of the following conditioning regimen based on his/her primary disease: 5.1 Total body irradiation 1200 rads in 6 fractionated doses and high dose chemotherapy, including etoposide and cyclophosphamide. 5.2 High dose chemotherapy with busulfan and cyclophosphamide. 5.2.1 Patients who are not candidates for TBI will receive chemotherapy-based conditioning regimen. 5.3 Post transplant immunosuppression prophylaxis against acute GVHD will include cyclosporine and methotrexate. 5.4 Donor will receive 3 daily G-CSF injections (starting on day -3) prior to marrow harvest. The injections may be initiated by the donor's primary physician prior to donor's arrival here, or by BMT service at Children's Healthcare of Atlanta. 5.5 Patients will receive daily G-CSF injections (5 mcg/kg) starting from day+5 post transplant.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hematologic Diseases, Hematologic Malignancies
Keywords
G-CSF, Bone Marrow, Hematologic malignancies, Non-malignancies, Matched sibling donor, Allogeneic BMT

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
10 (Actual)

8. Arms, Groups, and Interventions

Arm Title
single arm
Arm Type
Other
Intervention Type
Drug
Intervention Name(s)
Granulocyte Colony Stimulating Factor
Intervention Description
Granulocyte Colony Stimulating Factor
Primary Outcome Measure Information:
Title
Numbers of Participants With Disease-free Survival.
Description
Evaluate the numbers of participants with disease-free survival
Time Frame
260 days
Secondary Outcome Measure Information:
Title
Hospital Length of Stay
Description
hospital length of stay of each patient enrolled, an average of 5 weeks.
Time Frame
inpatient hospital stay

10. Eligibility

Sex
All
Maximum Age & Unit of Time
55 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Eligibility Criteria
Inclusion Criteria: Patients with hematologic malignancies and non-malignancies who are candidates for matched sibling donor allogeneic bone marrow transplantation are eligible for this study. Patients who are under 55 years of age. Patients who have a life expectancy of at least 12 weeks and a performance status of at least 2 Zubrod or 70% Karnofsky status prior to transplantation. Patients who are acceptable candidates for marrow transplantation based on their pre-BMT evaluation. Patients who have available histocompatible siblings who have been medically approved as marrow donors. Patients who sign informed consent for the protocol approved by the Institutional Review Board of Emory University/Children's Healthcare of Atlanta. Donors must be 5 years of age or older, and have completed routine donor evaluations and signed (by parent or legal guardian) informed consent for the protocol approved by the Institutional Review Board of Emory University/Children's Healthcare of Atlanta. Exclusion Criteria: Patients will not be excluded based on sex, racial, or ethnic background. Patients will be excluded if they demonstrate significant functional deficits in major organs, which would obviously interfere with a successful outcome following bone marrow transplant utilizing the following guidelines: Evidence of active, deep seated, life-threatening infections for which there is no known effective therapy (e.g. certain fungal species, HIV, etc.). Patients with hemoglobinopathy (e.g. sickle cell disease and thalassemia) will not be eligible for this protocol. However, filgrastim mobilization, large volume apheresis, processing, and cryopreservation appears to be safe in donors with sickle cell trait. Patients have had greater than two leukemic episodes, active central nervous system (CNS) and/or leukemic disease and blast crisis in chronic myelogenous leukemia (CML) patients. Patients will be excluded if they are women of childbearing potential who are currently pregnant (beta-hCG+) or who are not practicing adequate contraception. Patients who have had previous stem cell transplant will be excluded. Donors will be excluded if they are sensitive to E. coli-derived protein.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Kuang-Yueh Chiang, M.D.
Organizational Affiliation
Emory University/CHOA
Official's Role
Principal Investigator
Facility Information:
Facility Name
Children's Healthcare of Atlanta/Emory University
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States

12. IPD Sharing Statement

Learn more about this trial

Granulocyte Colony Stimulating Factor (G-CSF) for Bone Marrow Transplant (BMT)

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