Hematologic DiseasesNCT00231309
Granulocyte Colony Stimulating Factor (G-CSF) for Bone Marrow Transplant (BMT)
A study testing Granulocyte Colony Stimulating Factor in people with Hematologic Diseases and Hematologic Malignancies, run by Emory University at 1 site in United States.
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. Read more about this study
- Who is it for?
- Ages Up to 55 yearsHealthy volunteers can take part
- Conditions the study is for
- Hematologic Diseases, Hematologic Malignancies
- What is being tested?
- Granulocyte Colony Stimulating Factor
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2003to Nov 2011
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 55 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Healthcare of Atlanta/Emory University
Atlanta, Georgia, 30322
About this study
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
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.
Read the 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.
- Conditions
- Hematologic Diseases, Hematologic Malignancies
- Being tested
- Granulocyte Colony Stimulating Factor
- Keywords
- G-CSF, Bone Marrow, Hematologic malignancies, Non-malignancies, Matched sibling donor, Allogeneic BMT
- Medical subject headings
- Hematologic Diseases, Hematologic Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Granulocyte Colony Stimulating FactorDrug
Granulocyte Colony Stimulating Factor
| Study group | Role | What they receive |
|---|---|---|
| single arm | Other | Not described Receives: Drug: Granulocyte Colony Stimulating Factor |
What is being measured
The primary outcome is the main question the study is designed to answer.
Numbers of Participants With Disease-free Survival.
Evaluate the numbers of participants with disease-free survival
Measured: 260 days
Secondary outcomes (1)
Hospital Length of Stay
hospital length of stay of each patient enrolled, an average of 5 weeks.
Measured: inpatient hospital stay
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Oct 2005 (estimated)
- Study startJul 2003
- Primary completionNov 2011
- Study completionNov 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Hematologic DiseasesStudies of the same condition, open and under way.
- More trials run by Emory UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Kuang-Yueh Chiang, Emory University
- Investigators
- Kuang-Yueh Chiang, M.D., Principal investigator, Emory University/CHOA
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00231309
- Sponsor's protocol ID
- 0561-2003
- Status last verified
- Aug 2014
- First submitted
- 3 Oct 2005
- Last updated
- 22 Aug 2014
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 22 Aug 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT00231309
- Acronym / sponsor ID
- 0561-2003
- Conditions
- Hematologic Diseases, Hematologic Malignancies
- Keywords
- G-CSF, Bone Marrow, Hematologic malignancies, Non-malignancies, Matched sibling donor, Allogeneic BMT
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Study start
- Jul 2003
- Primary completion
- Nov 2011 (actual)
- Study completion
- Nov 2011 (actual)
- First posted
- 4 Oct 2005 (estimated)
- Results
- Posted 19 Aug 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| single arm | Other | Drug: Granulocyte Colony Stimulating Factor |
Who may qualify
- Ages
- Up to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Granulocyte Colony Stimulating FactorGranulocyte Colony Stimulating Factor | Drug | single arm |
Primary outcome
Numbers of Participants With Disease-free Survival.
Time frame: 260 days
Evaluate the numbers of participants with disease-free survival
1 secondary outcome
Hospital Length of Stay
Time frame: inpatient hospital stay
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Aug 2014.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Kuang-Yueh Chiang, M.D. principal investigator
Emory University/CHOA
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Granulocyte Colony Stimulating Factor in people with Hematologic Diseases and Hematologic Malignancies, run by Emory University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Aug 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 19 Aug 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Numbers of Participants With Disease-free Survival. (time frame: 260 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Aug 2014.