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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| single arm | Other |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Granulocyte Colony Stimulating Factor | Drug | Granulocyte Colony Stimulating Factor |
|
| Measure | Description | Time Frame |
|---|---|---|
| Numbers of Participants With Disease-free Survival. | Evaluate the numbers of participants with disease-free survival | 260 days |
| Measure | Description | Time Frame |
|---|---|---|
| Hospital Length of Stay | hospital length of stay of each patient enrolled, an average of 5 weeks. | inpatient hospital stay |
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Inclusion Criteria:
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:
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.
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| Name | Affiliation | Role |
|---|---|---|
| Kuang-Yueh Chiang, M.D. | Emory University/CHOA | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Healthcare of Atlanta/Emory University | Atlanta | Georgia | 30322 | United States |
Patients were not excluded before assignment to groups.
10 patients were enrolled between July 2003 and March 2006. Patients were enrolled during consent visits for upcoming bone marrow transplants.
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| ID | Title | Description |
|---|---|---|
| FG000 | Granulocyte Colony Stimulating Factor | Granulocyte Colony Stimulating Factor : Granulocyte Colony Stimulating Factor |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Single Arm | Granulocyte Colony Stimulating Factor : Granulocyte Colony Stimulating Factor |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Numbers of Participants With Disease-free Survival. | Evaluate the numbers of participants with disease-free survival | Posted | Number | participants | 260 days |
|
|
260 days.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Single Arm | Granulocyte Colony Stimulating Factor : Granulocyte Colony Stimulating Factor |
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Our study is also limited by its small patient population and short follow-up time.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Kuang-Yueh Chiang, MD | Emory University | 404-785-1441 | kuang-yueh.chiang@choa.org |
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| ID | Term |
|---|---|
| D006402 | Hematologic Diseases |
| D019337 | Hematologic Neoplasms |
| ID | Term |
|---|---|
| D006425 | Hemic and Lymphatic Diseases |
| D009371 | Neoplasms by Site |
| D009369 | Neoplasms |
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| ID | Term |
|---|---|
| D016179 | Granulocyte Colony-Stimulating Factor |
| ID | Term |
|---|---|
| D003115 | Colony-Stimulating Factors |
| D006023 | Glycoproteins |
| D006001 | Glycoconjugates |
| D002241 | Carbohydrates |
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| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
|
| Secondary | Hospital Length of Stay | hospital length of stay of each patient enrolled, an average of 5 weeks. | Posted | Median | Standard Deviation | average days | inpatient hospital stay |
|
|
|
| 0 |
| 10 |
| 0 |
| 10 |
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| D016298 |
| Hematopoietic Cell Growth Factors |
| D016207 | Cytokines |
| D036341 | Intercellular Signaling Peptides and Proteins |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
| D011506 | Proteins |
| D001685 | Biological Factors |