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

• Sponsor: Emory University (Other)
• Overall Status: Completed
• CT.gov ID: NCT00231309
• Collaborator: (none)
• Enrollment: 10
• Location: 1
• Arm: 1
• Duration (Months): 100
• Patients Per Site Per Month: 0.1

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
Hematologic Diseases; Hematologic Malignancies	Drug: Granulocyte Colony Stimulating Factor	N/A

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.

Study Design

• Study Type: Interventional
• Actual Enrollment: 10 participants
• Allocation: Non-Randomized
• Intervention Model: Single Group Assignment
• Masking: None (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
• Study Start Date: Jul 1, 2003
• Actual Primary Completion Date: Nov 1, 2011
• Actual Study Completion Date: Nov 1, 2011

Arms and Interventions

Arm	Intervention/Treatment
Other: single arm	Drug: Granulocyte Colony Stimulating Factor; Granulocyte Colony Stimulating Factor

Outcome Measures

Primary Outcome Measures

1. Numbers of Participants With Disease-free Survival. [260 days]

   Evaluate the numbers of participants with disease-free survival

Secondary Outcome Measures

1. Hospital Length of Stay [inpatient hospital stay]

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

Eligibility Criteria

Criteria

• Ages Eligible for Study: N/A to 55 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: Yes

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

Locations

	Site	City	State	Country	Postal Code
1	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

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Kuang-Yueh Chiang, Associate Professor, Emory University
• ClinicalTrials.gov Identifier: NCT00231309
• Other Study ID Numbers: 0561-2003
• First Posted: Oct 4, 2005
• Last Update Posted: Aug 22, 2014
• Last Verified: Aug 1, 2014

• Keywords provided by Kuang-Yueh Chiang, Associate Professor, Emory University: G-CSF; Bone Marrow; Hematologic malignancies; Non-malignancies; Matched sibling donor; Allogeneic BMT
• Additional relevant MeSH terms: Neoplasms; Hematologic Neoplasms; Hematologic Diseases; Lenograstim

Study Results

Participant Flow

Recruitment Details	10 patients were enrolled between July 2003 and March 2006. Patients were enrolled during consent visits for upcoming bone marrow transplants.
Pre-assignment Detail	Patients were not excluded before assignment to groups.

Arm/Group Title	Granulocyte Colony Stimulating Factor
Arm/Group Description	Granulocyte Colony Stimulating Factor : Granulocyte Colony Stimulating Factor
Period Title: Overall Study
STARTED	10
COMPLETED	10
NOT COMPLETED	0

Baseline Characteristics

Arm/Group Title	Single Arm
Arm/Group Description	Granulocyte Colony Stimulating Factor : Granulocyte Colony Stimulating Factor
Overall Participants	10
Age (Count of Participants)
<=18 years	10 100%
Between 18 and 65 years	0 0%
>=65 years	0 0%
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	11.47 (4.78)
Sex: Female, Male (Count of Participants)
Female	0 0%
Male	10 100%
Region of Enrollment (participants) [Number]
United States	10 100%

Outcome Measures

1. Primary Outcome

Title	Numbers of Participants With Disease-free Survival.
Description	Evaluate the numbers of participants with disease-free survival
Time Frame	260 days

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Granulocyte-stimulating Factor
Arm/Group Description	Granulocyte Colony Stimulating Factor : Granulocyte Colony Stimulating Factor
Measure Participants	10
Number [participants]	10 100%

2. Secondary Outcome

Title	Hospital Length of Stay
Description	hospital length of stay of each patient enrolled, an average of 5 weeks.
Time Frame	inpatient hospital stay

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Granulocyte Colony Stimulating Factor
Arm/Group Description	Granulocyte Colony Stimulating Factor : Granulocyte Colony Stimulating Factor
Measure Participants	10
Median (Standard Deviation) [average days]	35 (70)

Adverse Events

Time Frame	260 days.
Arm/Group Title	Single Arm
Arm/Group Description	Granulocyte Colony Stimulating Factor : Granulocyte Colony Stimulating Factor
All Cause Mortality
	Single Arm
	Affected / at Risk (%)	# Events
Total	/ (NaN)
Serious Adverse Events
	Single Arm
	Affected / at Risk (%)	# Events
Total	0/10 (0%)
Other (Not Including Serious) Adverse Events
	Single Arm
	Affected / at Risk (%)	# Events
Total	0/10 (0%)

Limitations/Caveats

Our study is also limited by its small patient population and short follow-up time.

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.