Hematologic MalignanciesG-CSF PMRDNCT00228813
G-CSF PMRD: Granulocyte Colony Stimulating Factor (G-CSF) Stimulated Bone Marrow and In Vivo T-Cell Depletion in Patients With Hematologic Malignancies or Bone Marrow Failure Syndrome
A study testing Granulocyte Colony Stimulating Factor in people with Hematologic Malignancies, run by Emory University at 1 site in United States.
Trial summary
The purposes of this study are: To examine the engraftment rate in patients receiving in vivo T-cell-depleted G-CSF stimulated bone marrow from partially mismatched related donors. Read more about this study
- Who is it for?
- Ages Up to 22 years
- Conditions the study is for
- Hematologic Malignancies
- What is being tested?
- Granulocyte Colony Stimulating Factor
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2004to Jan 2015
- 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 22 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients with hematologic malignancies or bone marrow failure syndrome who are candidates for allogeneic bone marrow transplantation are eligible for this study. Hematologic malignancies indicated for transplantation:
- Acute lymphoblastic leukemia (ALL) in first remission (with high risk feature), 2nd or greater remission.
- Acute myeloid leukemia (AML) in first remission (with high risk feature), 2nd or greater remission.
- Chronic myeloid leukemia (CML) in 2nd chronic phase or accelerated phase.
- Juvenile myelomonocytic leukemia (JMML).
- Myelodysplastic syndrome.
Show all 14Show fewer
- Biphenotypic leukemia in first (with high risk feature), 2nd or greater remission.
- Induction failure leukemia.
- Refractory relapsed leukemia.
- Bone marrow failure syndrome.
- Severe aplastic anemia failed immunotherapy.
- Patients who do not have a 6 out of 6 matched related or unrelated donor or 4/6 and 5/6 matched cord blood will be eligible for this study.
- Partially mismatched related donor availability as defined by molecular typing with 3 to 5 HLA matches.
- Patients who are under 22 years of age.
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 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 (certain fungal species, HIV, etc.).
- Patients who have been treated for infections must have appropriate responses as documented by 2 (two) consecutive negative cultures and/or stable radiographic examinations.
- Patients who have active central nervous system (CNS) leukemic disease.
- 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 a previous hematopoietic 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: Feasibility Study of Using G-CSF Stimulated Bone Marrow and In Vivo T-Cell Depletion in Patients With Hematologic Malignancies or Bone Marrow Failure Syndrome With Partially Mismatched Related Donors
The purposes of this study are:
- To examine the engraftment rate in patients receiving in vivo T-cell-depleted G-CSF stimulated bone marrow from partially mismatched related donors.
- To evaluate the incidence and severity of acute and chronic graft-versus-host disease in patients receiving in vivo T-cell-depleted G-CSF stimulated bone marrow from partially mismatched related donors.
Read the detailed description
This study is a single-arm, non-randomized feasibility study. 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 the following conditioning regimen based on their primary disease:
- Total body irradiation (1400 rads in 8 fractionated doses) and high dose chemotherapy, including cytosine arabinoside, etoposide, and cyclophosphamide. Patients with bone marrow failure syndrome will not receive etoposide in the conditioning regimen.
- Post transplant immunosuppression prophylaxis against acute GVHD will include sequential administration of cyclosporine, methotrexate, basiliximab and mycophenolate.
- The donor will receive 3 daily G-CSF injections prior to marrow harvest starting on day -3. The injections may be initiated by the donor's primary physician prior to donor's arrival, or by the BMT service at Children's Healthcare of Atlanta.
- Patients will receive daily GM-CSF injections (250 mcg/m2) starting from day +7 post transplant until absolute neutrophil count (ANC) is greater than 2,000/µL for three days.
- Conditions
- Hematologic Malignancies
- Being tested
- Granulocyte Colony Stimulating Factor
- Medical subject headings
- 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
FILGRASTIM: G-CSF (NEUPOGEN®) is administered as a short IV infusion over 30 minutes or subcutaneously. It is given beginning on day -3 for 3 days to the donor prior to the bone marrow harvest.
Drug Information: FILGRASTIM: G-CSF (Neupogen®) Formulation: G-CSF is available as a preservative-free solution for injection in 1.0 ml and 1.6 ml vials containing 300 mcg/ml.
Administration: G-CSF 5 mcg/kg/d will be given subcutaneously or as a short I.V. infusion over 30 minutes.
Recombinant GM-CSF at the dose of 250 mcg/m2 will be given intravenously from day +7 to help white counts recovery. The drug will be diluted in NS at a concentration of at least 10 mcg/ml.
Drug Information: Sargramostim (Leukine) Formulation: 250 mcg, 500 mcg lyophlized powder for injection
| Study group | Role | What they receive |
|---|---|---|
| Granulocyte Colony Stimulating Factor (G-CSF) stimulation | Other | Participants will receive bone marrow from donors who undergo Granulocyte Colony Stimulating Factor (G-CSF) stimulation prior to bone marrow collection. Receives: Drug: Granulocyte Colony Stimulating Factor |
What is being measured
The primary outcome is the main question the study is designed to answer.
Engraftment Rate
The number of participants who received in vivo T-cell-depleted G-CSF stimulated bone marrow from partially mismatched related donor who reached engraftment by Day 45.
Measured: Day 45
Incidence of Acute Graft-versus-host Disease
The number of participants diagnosed with new acute graft-versus-host disease (GVHD).
Measured: Day 100
Incidence of Chronic Graft-versus-host Disease
The number of participants diagnosed with chronic graft-versus-host disease (GVHD).
Measured: Duration of Study (Up to two years)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Sep 2005 (estimated)
- Study startApr 2004
- Primary completionJan 2015
- Study completionJan 2015
Why it stopped: lack of enrollment
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 MalignanciesStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Kuang-Yueh Chiang, M.D., Principal investigator, Children's Healthcare of Atlanta/Emory University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00228813
- Sponsor's protocol ID
- IRB00021819
- Other IDs
- 0159-2004
- Acronym
- G-CSF PMRD
- Status last verified
- Aug 2017
- First submitted
- 27 Sep 2005
- Last updated
- 11 Sep 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 11 Sep 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Feasibility Study of Using G-CSF Stimulated Bone Marrow and In Vivo T-Cell Depletion in Patients With Hematologic Malignancies or Bone Marrow Failure Syndrome With Partially Mismatched Related Donors
- ClinicalTrials.gov ID
- NCT00228813
- Acronym / sponsor ID
- G-CSF PMRD / IRB00021819
- Conditions
- Hematologic Malignancies
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: lack of enrollment
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Study start
- Apr 2004
- Primary completion
- Jan 2015 (actual)
- Study completion
- Jan 2015 (actual)
- First posted
- 29 Sep 2005 (estimated)
- Results
- Posted 6 Jul 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purposes of this study are:
- To examine the engraftment rate in patients receiving in vivo T-cell-depleted G-CSF stimulated bone marrow from partially mismatched related donors.
- To evaluate the incidence and severity of acute and chronic graft-versus-host disease in patients receiving in vivo T-cell-depleted G-CSF stimulated bone marrow from partially mismatched related donors.
Detailed description
This study is a single-arm, non-randomized feasibility study. 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 the following conditioning regimen based on their primary disease:
- Total body irradiation (1400 rads in 8 fractionated doses) and high dose chemotherapy, including cytosine arabinoside, etoposide, and cyclophosphamide. Patients with bone marrow failure syndrome will not receive etoposide in the conditioning regimen.
- Post transplant immunosuppression prophylaxis against acute GVHD will include sequential administration of cyclosporine, methotrexate, basiliximab and mycophenolate.
- The donor will receive 3 daily G-CSF injections prior to marrow harvest starting on day -3. The injections may be initiated by the donor's primary physician prior to donor's arrival, or by the BMT service at Children's Healthcare of Atlanta.
- Patients will receive daily GM-CSF injections (250 mcg/m2) starting from day +7 post transplant until absolute neutrophil count (ANC) is greater than 2,000/µL for three days.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Granulocyte Colony Stimulating Factor (G-CSF) stimulation | Other | Drug: Granulocyte Colony Stimulating FactorParticipants will receive bone marrow from donors who undergo Granulocyte Colony Stimulating Factor (G-CSF) stimulation prior to bone marrow collection. |
Who may qualify
- Ages
- Up to 22 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (14 listed)
- Patients with hematologic malignancies or bone marrow failure syndrome who are candidates for allogeneic bone marrow transplantation are eligible for this study. Hematologic malignancies indicated for transplantation:
- Patients who do not have a 6 out of 6 matched related or unrelated donor or 4/6 and 5/6 matched cord blood will be eligible for this study.
- Partially mismatched related donor availability as defined by molecular typing with 3 to 5 HLA matches.
- Patients who are under 22 years of age.
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 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 a previous hematopoietic 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 FactorFILGRASTIM: G-CSF (NEUPOGEN®) is administered as a short IV infusion over 30 minutes or subcutaneously. It is given beginning on day -3 for 3 days to the donor prior to the bone marrow harvest. Drug Information: FILGRASTIM: G-CSF (Neupogen®) Formulation: G-CSF is available as a preservative-free solution for injection in 1.0 ml and 1.6 ml vials containing 300 mcg/ml. Administration: G-CSF 5 mcg/kg/d will be given subcutaneously or as a short I.V. infusion over 30 minutes. Recombinant GM-CSF at the dose of 250 mcg/m2 will be given intravenously from day +7 to help white counts recovery. The drug will be diluted in NS at a concentration of at least 10 mcg/ml. Drug Information: Sargramostim (Leukine) Formulation: 250 mcg, 500 mcg lyophlized powder for injection | Drug | Granulocyte Colony Stimulating Factor (G-CSF) stimulation |
Primary outcome
Engraftment Rate
Time frame: Day 45
The number of participants who received in vivo T-cell-depleted G-CSF stimulated bone marrow from partially mismatched related donor who reached engraftment by Day 45.
Incidence of Acute Graft-versus-host Disease
Time frame: Day 100
The number of participants diagnosed with new acute graft-versus-host disease (GVHD).
Incidence of Chronic Graft-versus-host Disease
Time frame: Duration of Study (Up to two years)
The number of participants diagnosed with chronic graft-versus-host disease (GVHD).
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Sep 2017.
- 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
Children's Healthcare of Atlanta/Emory University
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 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 14 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: stopped early, as of the sponsor's last update on 11 Sep 2017. 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 6 Jul 2016). 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?
Engraftment Rate (time frame: Day 45). The study lists 3 primary outcomes in all. 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 11 Sep 2017.