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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Kuang-Yueh Chiang, Emory University
ClinicalTrials.gov Identifier:
NCT00231309
First received: October 3, 2005
Last updated: August 19, 2014
Last verified: August 2014
  Purpose

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.


Condition Intervention
Hematologic Diseases
Hematologic Malignancies
Drug: Granulocyte Colony Stimulating Factor

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Emory University:

Primary Outcome Measures:
  • Numbers of Participants With Disease-free Survival. [ Time Frame: 260 days ] [ Designated as safety issue: No ]
    Evaluate the numbers of participants with disease-free survival


Secondary Outcome Measures:
  • Hospital Length of Stay [ Time Frame: inpatient hospital stay ] [ Designated as safety issue: No ]
    hospital length of stay of each patient enrolled, an average of 5 weeks.


Enrollment: 10
Study Start Date: July 2003
Study Completion Date: November 2011
Primary Completion Date: November 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
single arm Drug: Granulocyte Colony Stimulating Factor
Granulocyte Colony Stimulating Factor

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.

  Eligibility

Ages Eligible for Study:   up to 55 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   Yes
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00231309

Locations
United States, Georgia
Children's Healthcare of Atlanta/Emory University
Atlanta, Georgia, United States, 30322
Sponsors and Collaborators
Emory University
Investigators
Principal Investigator: Kuang-Yueh Chiang, M.D. Emory University/CHOA
  More Information

No publications provided

Responsible Party: Kuang-Yueh Chiang, Associate Professor, Emory University
ClinicalTrials.gov Identifier: NCT00231309     History of Changes
Other Study ID Numbers: 0561-2003
Study First Received: October 3, 2005
Results First Received: August 14, 2013
Last Updated: August 19, 2014
Health Authority: United States: Institutional Review Board

Keywords provided by Emory University:
G-CSF
Bone Marrow
Hematologic malignancies
Non-malignancies
Matched sibling donor
Allogeneic BMT

Additional relevant MeSH terms:
Hematologic Diseases
Neoplasms
Lenograstim
Adjuvants, Immunologic
Immunologic Factors
Pharmacologic Actions
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on October 20, 2014