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

Hematologic Diseases Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00231309
• Other study ID #: 0561-2003
• Status: Completed
• Phase: N/A
• First received: October 3, 2005
• Last updated: August 19, 2014
• Start date: July 2003
• Est. completion date: November 2011

Study information

• Verified date: August 2014
• Source: Emory University
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 10
• Est. completion date: November 2011
• Est. primary completion date: November 2011
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: Both
• Age group: N/A to 55 Years

Eligibility

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.

Study Design

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

Related Conditions & MeSH terms

• Hematologic Diseases
• Hematologic Malignancies
• Neoplasms

Intervention

Drug:
• Granulocyte Colony Stimulating Factor
Granulocyte Colony Stimulating Factor

Locations

Country	Name	City	State
United States	Children's Healthcare of Atlanta/Emory University	Atlanta	Georgia

Sponsors (1)

Lead Sponsor	Collaborator
Emory University

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Numbers of Participants With Disease-free Survival.	Evaluate the numbers of participants with disease-free survival	260 days	No
Secondary	Hospital Length of Stay	hospital length of stay of each patient enrolled, an average of 5 weeks.	inpatient hospital stay	No