LeukemiaPhase 2NCT00582933
Phase II Trial of a Chemotherapy Alone Regimen of IV Busulfan (Busulfex), Melphalan and Fludarabine as Myeloablative Regimen Followed by an Allogeneic T-Cell Depleted Hematopoietic Stem Cell Transplant From an HLA-Identical, or HLA-Non Identical Related or Unrelated Donor
A Phase 2 study testing BUSULFAN, MELPHALAN, FLUDARABINE, G-CSF in people with Leukemia and Myelodysplastic Syndrome, run by Memorial Sloan Kettering Cancer Center at 1 site in United States.
Trial summary
The purpose of this research study is:(1) to determine if high doses of chemotherapy without total body irradiation can allow selected stem cells to take and grow,(2) to determine if selected stem cells from the blood or marrow can take and not cause a complication called graft-versus-host disease (GvHD) and (3) to… Read more about this study
- Who is it for?
- Ages Up to 54 years
- Conditions the study is for
- Leukemia, Myelodysplastic Syndrome, Non-Hodgkin's Lymphoma
- What is being tested?
- BUSULFAN, MELPHALAN, FLUDARABINE, G-CSF
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 96 participants
- Where
- 1 siteUnited States
- Study dates
- May 2001to Apr 2009
- Phase
- Phase 2
- 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 54 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosis: Acute myelogenous leukemia (AML) Acute undifferentiated leukemia (AUL); Acute lymphoblastic leukemia (ALL); Acute biphenotypic leukemia (AbiL); Chronic myelogenous leukemia (CML); Non Hodgkin's lymphoma (NHL); Myelodysplastic syndrome (MDS) Aplastic anemia (AA)
- Status: CR1 first remission; CR2 second remission; CP chronic phase
- HLA-matched Related donors: Research participants who have an HLA-matched related donor are eligible for entry on this protocol. This will include a healthy related donor who is genotypically or phenotypically matched at all A, B and DRB1 loci, as tested by DNA analysis.
- HLA-compatible Unrelated donors: Research participants who do not have a related HLA-matched donor but have an unrelated donor who is either matched at all A, B and DRB1 loci or who is mismatched at 1/6 loci (A, B, or DRB1) as tested by DNA analysis, will be eligible for entry on this protocol.
- HLA-mismatched Related donors: Research participants who do not have a related or unrelated HLA-compatible donor must have a healthy family member who is at least HLA-haplotype identical to the recipient.
- Research participants must have a healthy HLA compatible related or unrelated donor who is willing to receive G-CSF injections and undergo apheresis for PBSC collection, or undergo a marrow harvesting procedure.
Show all 10Show fewer
- Research participants should be < 55 years. There is no lower age limit. Research participants > 55 years will be accrued on a case by case basis after discussion and approval by the BMT Service.
- Research participants may be of either gender or any ethnic background.
- Research participants must have a Karnofsky (adult) or Lansky (pediatric) Performance Status > 70%
- Research participants must have adequate physical function
Who cannot join
- Active CNS or skin leukemic/lymphomatous involvement
- Female research participants who are pregnant or breast-feeding
- Active viral, bacterial or fungal infection
- Research participant seropositive for HIV-I/II; HTLV -I/II
- Research participants who have undergone a prior allogeneic or autologous stem cell transplant within the previous six months.
Trial locations and contacts
1 site in 1 country.
United States1 site
Memorial Sloan-Kettering Cancer Center
New York, New York, 10065
About this study
Official title: Phase II Trial of a Chemotherapy Alone Regimen of IV Busulfan (Busulfex), Melphalan and Fludarabine as Myeloablative Regimen Followed by an Allogeneic T-Cell Depleted Hematopoietic Stem Cell Transplant From an HLA-Identical, or HLA-Non Identical Related or Unrelated Donor For the Treatment of Lymphohematopoietic Disorders
The purpose of this research study is:(1) to determine if high doses of chemotherapy without total body irradiation can allow selected stem cells to take and grow,(2) to determine if selected stem cells from the blood or marrow can take and not cause a complication called graft-versus-host disease (GvHD) and (3) to evaluate the side effects of the combination of chemotherapy drugs used for these transplants. In the last 10 years we have developed chemotherapy combinations to be used for this T-cell depleted transplant protocol. By using three chemotherapy drugs (IV busulfan, melphalan and fludarabine), we hope to have a good chemotherapy combination to kill cancer cells, and to make the graft take, without the side effects of total body irradiation. The chemotherapy drugs to be tested in this protocol are busulfan, melphalan and fludarabine, all of which have been used successfully for stem cell transplantation, but not given together as in this specific regimen. This is what is being tested in this study.
Our initial trials in the 1980's with T-cell depleted transplants showed less GvHD, but the overall results of the transplants were not better. The reason for this was that the stem cells did not take and engraft in 15% of our adult patients. This failure of the stem cells to take can leave patients without bone marrow or blood cells necessary for life. Most stem cell transplants were done using bone marrow (BMA) obtained from the donors. However, if we give a medication called G-CSF by shots to the donor, we can collect peripheral blood stem cells (PBSC) and use them for transplant. The advantage of this approach is that we can collect 2-20 times more stem cells than that obtained from the marrow. It has been proven that a larger number of stem cells in the graft make it more difficult for the patient to reject the stem cells. Some donors may be too small to provide peripheral blood stem cells or they may not want to take G-CSF shots. In these cases the donors will have their marrow collected in the operating room under general anesthesia.
Stem cell transplants can lead to a condition known as acute graft-versus-host disease or GvHD. This disease is caused by an assault by certain cells in the marrow or blood (T-cells) of the donor (graft) against your body (the host). These T-cells see your body as foreign and attack it. The disease causes a skin rash, liver disease, and diarrhea. Methods were developed at this institution to prevent GvHD. These methods take out most of the T-cells (responsible for GvHD) from the marrow or blood stem cells before transplant. This is called "T-cell depletion" or "stem cell selection". In this hospital, we use two types of methods of T-cell depletion: one method is used with peripheral blood stem cells and one for bone marrow. Both these techniques have been successful in preventing both acute and chronic GvHD. You will receive a T-cell depleted stem cell transplant.
Read the detailed description
The trial proposed is a single arm, phase II treatment protocol designed to examine the engraftment, toxicity, graft-versus-host disease and ultimate disease-free survival following transplants derived from (1) HLA-matched siblings or related donors, (2) HLA-compatible unrelated donors or (3) HLA haplo-type mismatched related donors using a new chemotherapeutic cytoreductive regimen. Candidates for transplant will be stratified according to their donor types.
Candidates for this trial will include patients with high risk forms of ALL, AML or CML, non-Hodgkin's lymphoma, or myelodysplastic syndrome for whom an allogeneic marrow transplant is clearly indicated, and patients with aplastic anemia refractory to ATG or cyclosporine treatment and who are transfusion dependent. All research participants will be conditioned for transplantation with intravenous busulfan (busulfex®) (0.8- 1.0 mg/Kg/dose Q6H x 10 doses), melphalan (70 mg/m2/dose x 2 doses) and fludarabine (25mg/m2/day x 5 doses). Doses of busulfan will be adjusted according to plasma levels. All research participants will also receive ATG (Thymoglobulin®) prior to transplant to promote engraftment. No drug prophylaxis againstGvHD will be administered post transplant. All research participants will also receive G-CSF post-transplant to foster engraftment.
The preferred source of stem cells will be peripheral blood stem cells (PBSC) induced and mobilized by treatment of the donor with G-CSF for 5-6 days. PBSC obtained through 2-3 leukaphereses will be Isolex® 300i separated CD34+ stem cell column selected and E-rosette depleted (E-). The CD34 + E peripheral blood progenitors will then be administered to the research participants after they have completed cytoreduction. If the use of CD34 + E- PBSC is not possible, the alternative graft will consist of bone marrow derived stem cells T-cell depleted by soybean agglutinin and E-rosetting (SBA-E-).
Research participants will be carefully monitored for engraftment, chimerism, incidence and severity of acute and chronic GvHD, regimen-related toxicity, characteristics of hematopoietic and immune reconstitution and ultimate survival and disease-free survival. This phase II trial is designed to investigate the feasibility and safety of a chemotherapy-based cytoreductive regimen plus a T-cell depleted peripheral blood stem cell (PBSC) or bone marrow stem cell transplant (BMT) for the treatment of high risk patients with advanced stages of hematologic malignancies. The majority of research participants will receive grafts derived from PBSC and will be the focus of the trial. The study population will be segmented into three research participant groups based on the type of donors used for the hematopoietic stem cell graft: (1) HLA-identical sibling or related donor, (2) HLA-compatible unrelated donor, and (3) HLA-mismatched related donor.
A maximum of 25 PBSC research participants in both related groups will be accrued onto the study; a maximum of 70 PBSC research participants in the unrelated group will be accrued. In order to reduce patient risk, the study design includes early termination of any trial group in the event of excessive graft failure, grade 3-4 acute graft-versus-host disease, or early transplant related mortality during the accrual period. Excessive failure is defined differently in the three donor groups. Stopping rules for the three research participant populations will be utilized. In addition to the 120 PBSC research participants, we anticipate approximately 25 BMT research participants treated across the three donor groups. These research participants will be followed and at the conclusion of the trial descriptive statistics on this subgroup will be recorded.
- Being tested
- BUSULFAN, MELPHALAN, FLUDARABINE, G-CSF
- Keywords
- Cancer, Donors, Leukemia, Myelodysplastic Syndrome, Non-Hodgkin's Lymphoma, Lymphoma, AML, ALL, CML, Allogeneic marrow transplant, Bone marrow transplant
- Medical subject headings
- Leukemia, Myelodysplastic Syndromes, Lymphoma, Non-Hodgkin, Neoplasms, Lymphoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BUSULFAN, MELPHALAN, FLUDARABINE, G-CSFDrug
All research participants will be conditioned for transplantation with intravenous busulfan (busulfex®) (0.8- 1.0 mg/Kg/dose Q6H x 10 doses), melphalan (70 mg/m2/dose x 2 doses) and fludarabine (25 mg/m2/day x 5 doses). Doses of busulfan will be adjusted according to plasma levels. All research participants will also receive ATG (thymoglobulin®) prior to transplant to promote engraftment.All research participants will also receive G-CSF posttransplant to foster engraftment.
The preferred source of stem cells will be peripheral blood stem cells (PBSC) induced and mobilized by treatment of the donor with G-CSF for 5-6 days. PBSC obtained through 2-3 leukaphereses will be Isolex® 300i separated CD34+ stem cell column selected and E-rosette depleted (E-). The CD34+Eperipheral blood progenitors will be administered to the research participants after they have completed cytoreduction.
BUSULFAN, MELPHALAN, FLUDARABINE, G-CSFDrug
All research participants will be conditioned for transplantation with intravenous busulfan (busulfex®) (0.8- 1.0 mg/Kg/dose Q6H x 10 doses), melphalan (70 mg/m2/dose x 2 doses) and fludarabine (25mg/m2/day x 5 doses). Doses of busulfan will be adjusted according to plasma levels. All research participants will also receive ATG (Thymoglobulin®) prior to transplant to promote engraftment. No drug prophylaxis against GvHD will be administered post transplant. All research participants will also receive G-CSF posttransplant to foster engraftment.
The preferred source of stem cells will be peripheral blood stem cells (PBSC) induced and mobilized by treatment of the donor with G-CSF for 5-6 days. PBSC obtained through 2-3 leukaphereses will be Isolex® 300i separated CD34+ stem cell column selected and E-rosette depleted (E-). The CD34+Eperipheral blood progenitors will be administered to the research participants after they have completed cytoreduction.
BUSULFAN, MELPHALAN, FLUDARABINE, G-CSFDrug
All research participants will be conditioned for transplantation with intravenous busulfan (busulfex®) (0.8- 1.0 mg/Kg/dose Q6H x 10 doses), melphalan (70 mg/m2/dose x 2 doses) and fludarabine (25mg/m2/day x 5 doses). Doses of busulfan will be adjusted according to plasma levels. All research participants will also receive ATG (Thymoglobulin®) prior to transplant to promote engraftment. No drug prophylaxis against GvHD will be administered post transplant. All research participants will also receive G-CSF posttransplant to foster engraftment.
The preferred source of stem cells will be peripheral blood stem cells (PBSC) induced and mobilized by treatment of the donor with G-CSF for 5-6 days. PBSC obtained through 2-3 leukaphereses will be Isolex® 300i separated CD34+ stem cell column selected and E-rosette depleted (E-). The CD34+Eperipheral blood progenitors will be administered to the research participants after they have completed cytoreduction.
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | 25 research participants with HLA Identical Related Donor using PBSC, 6 with BMT Receives: Drug: BUSULFAN, MELPHALAN, FLUDARABINE, G-CSF |
| 2 | Experimental | 70 research participants with HLA-Matched Unrelated Donor using PBSC, 17 with BMT Receives: Drug: BUSULFAN, MELPHALAN, FLUDARABINE, G-CSF |
| 3 | Experimental | 25 research participants with HLA-Mismatched Related Donor using PBSC, no BMT Receives: Drug: BUSULFAN, MELPHALAN, FLUDARABINE, G-CSF |
What is being measured
The primary outcome is the main question the study is designed to answer.
Death From GVHD
To establish the early transplant-related severe morbidity and mortality and 3-the incidence and severity of GvHD.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Dec 2007 (estimated)
- Study startMay 2001
- Primary completionApr 2008
- Study completionApr 2009
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 LeukemiaStudies of the same condition, open and under way.
- More trials run by Memorial Sloan Kettering Cancer CenterThe 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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 96 participants (actual)
Sponsor and investigators
- Lead sponsor
- Memorial Sloan Kettering Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Farid Boulad, MD, Principal investigator, Memorial Sloan Kettering Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00582933
- Sponsor's protocol ID
- 01-055
- Other IDs
- CA23766
- Status last verified
- Dec 2015
- First submitted
- 21 Dec 2007
- Last updated
- 1 Feb 2016
- Results posted
- Yes, on ClinicalTrials.gov
Study at a glance
From the registry record, last updated by the sponsor on 1 Feb 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Trial of a Chemotherapy Alone Regimen of IV Busulfan (Busulfex), Melphalan and Fludarabine as Myeloablative Regimen Followed by an Allogeneic T-Cell Depleted Hematopoietic Stem Cell Transplant From an HLA-Identical, or HLA-Non Identical Related or Unrelated Donor For the Treatment of Lymphohematopoietic Disorders
- ClinicalTrials.gov ID
- NCT00582933
- Acronym / sponsor ID
- 01-055
- Keywords
- Cancer, Donors, Leukemia, Myelodysplastic Syndrome, Non-Hodgkin's Lymphoma, Lymphoma, AML, ALL
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 96 participants (actual)
- Lead sponsor
- Memorial Sloan Kettering Cancer CenterUniversity, hospital or other
- Study start
- May 2001
- Primary completion
- Apr 2008 (actual)
- Study completion
- Apr 2009 (actual)
- First posted
- 28 Dec 2007 (estimated)
- Results
- Posted 1 Feb 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this research study is:(1) to determine if high doses of chemotherapy without total body irradiation can allow selected stem cells to take and grow,(2) to determine if selected stem cells from the blood or marrow can take and not cause a complication called graft-versus-host disease (GvHD) and (3) to evaluate the side effects of the combination of chemotherapy drugs used for these transplants. In the last 10 years we have developed chemotherapy combinations to be used for this T-cell depleted transplant protocol. By using three chemotherapy drugs (IV busulfan, melphalan and fludarabine), we hope to have a good chemotherapy combination to kill cancer cells, and to make the graft take, without the side effects of total body irradiation. The chemotherapy drugs to be tested in this protocol are busulfan, melphalan and fludarabine, all of which have been used successfully for stem cell transplantation, but not given together as in this specific regimen. This is what is being tested in this study.
Our initial trials in the 1980's with T-cell depleted transplants showed less GvHD, but the overall results of the transplants were not better. The reason for this was that the stem cells did not take and engraft in 15% of our adult patients. This failure of the stem cells to take can leave patients without bone marrow or blood cells necessary for life. Most stem cell transplants were done using bone marrow (BMA) obtained from the donors. However, if we give a medication called G-CSF by shots to the donor, we can collect peripheral blood stem cells (PBSC) and use them for transplant. The advantage of this approach is that we can collect 2-20 times more stem cells than that obtained from the marrow. It has been proven that a larger number of stem cells in the graft make it more difficult for the patient to reject the stem cells. Some donors may be too small to provide peripheral blood stem cells or they may not want to take G-CSF shots. In these cases the donors will have their marrow collected in the operating room under general anesthesia.
Stem cell transplants can lead to a condition known as acute graft-versus-host disease or GvHD. This disease is caused by an assault by certain cells in the marrow or blood (T-cells) of the donor (graft) against your body (the host). These T-cells see your body as foreign and attack it. The disease causes a skin rash, liver disease, and diarrhea. Methods were developed at this institution to prevent GvHD. These methods take out most of the T-cells (responsible for GvHD) from the marrow or blood stem cells before transplant. This is called "T-cell depletion" or "stem cell selection". In this hospital, we use two types of methods of T-cell depletion: one method is used with peripheral blood stem cells and one for bone marrow. Both these techniques have been successful in preventing both acute and chronic GvHD. You will receive a T-cell depleted stem cell transplant.
Detailed description
The trial proposed is a single arm, phase II treatment protocol designed to examine the engraftment, toxicity, graft-versus-host disease and ultimate disease-free survival following transplants derived from (1) HLA-matched siblings or related donors, (2) HLA-compatible unrelated donors or (3) HLA haplo-type mismatched related donors using a new chemotherapeutic cytoreductive regimen. Candidates for transplant will be stratified according to their donor types.
Candidates for this trial will include patients with high risk forms of ALL, AML or CML, non-Hodgkin's lymphoma, or myelodysplastic syndrome for whom an allogeneic marrow transplant is clearly indicated, and patients with aplastic anemia refractory to ATG or cyclosporine treatment and who are transfusion dependent. All research participants will be conditioned for transplantation with intravenous busulfan (busulfex®) (0.8- 1.0 mg/Kg/dose Q6H x 10 doses), melphalan (70 mg/m2/dose x 2 doses) and fludarabine (25mg/m2/day x 5 doses). Doses of busulfan will be adjusted according to plasma levels. All research participants will also receive ATG (Thymoglobulin®) prior to transplant to promote engraftment. No drug prophylaxis againstGvHD will be administered post transplant. All research participants will also receive G-CSF post-transplant to foster engraftment.
The preferred source of stem cells will be peripheral blood stem cells (PBSC) induced and mobilized by treatment of the donor with G-CSF for 5-6 days. PBSC obtained through 2-3 leukaphereses will be Isolex® 300i separated CD34+ stem cell column selected and E-rosette depleted (E-). The CD34 + E peripheral blood progenitors will then be administered to the research participants after they have completed cytoreduction. If the use of CD34 + E- PBSC is not possible, the alternative graft will consist of bone marrow derived stem cells T-cell depleted by soybean agglutinin and E-rosetting (SBA-E-).
Research participants will be carefully monitored for engraftment, chimerism, incidence and severity of acute and chronic GvHD, regimen-related toxicity, characteristics of hematopoietic and immune reconstitution and ultimate survival and disease-free survival. This phase II trial is designed to investigate the feasibility and safety of a chemotherapy-based cytoreductive regimen plus a T-cell depleted peripheral blood stem cell (PBSC) or bone marrow stem cell transplant (BMT) for the treatment of high risk patients with advanced stages of hematologic malignancies. The majority of research participants will receive grafts derived from PBSC and will be the focus of the trial. The study population will be segmented into three research participant groups based on the type of donors used for the hematopoietic stem cell graft: (1) HLA-identical sibling or related donor, (2) HLA-compatible unrelated donor, and (3) HLA-mismatched related donor.
A maximum of 25 PBSC research participants in both related groups will be accrued onto the study; a maximum of 70 PBSC research participants in the unrelated group will be accrued. In order to reduce patient risk, the study design includes early termination of any trial group in the event of excessive graft failure, grade 3-4 acute graft-versus-host disease, or early transplant related mortality during the accrual period. Excessive failure is defined differently in the three donor groups. Stopping rules for the three research participant populations will be utilized. In addition to the 120 PBSC research participants, we anticipate approximately 25 BMT research participants treated across the three donor groups. These research participants will be followed and at the conclusion of the trial descriptive statistics on this subgroup will be recorded.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Drug: BUSULFAN, MELPHALAN, FLUDARABINE, G-CSF25 research participants with HLA Identical Related Donor using PBSC, 6 with BMT |
| 2 | Experimental | Drug: BUSULFAN, MELPHALAN, FLUDARABINE, G-CSF70 research participants with HLA-Matched Unrelated Donor using PBSC, 17 with BMT |
| 3 | Experimental | Drug: BUSULFAN, MELPHALAN, FLUDARABINE, G-CSF25 research participants with HLA-Mismatched Related Donor using PBSC, no BMT |
Who may qualify
- Ages
- Up to 54 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (10 listed)
- Diagnosis: Acute myelogenous leukemia (AML) Acute undifferentiated leukemia (AUL); Acute lymphoblastic leukemia (ALL); Acute biphenotypic leukemia (AbiL); Chronic myelogenous leukemia (CML); Non Hodgkin's lymphoma (NHL); Myelodysplastic syndrome (MDS) Aplastic anemia (AA)
- Status: CR1 first remission; CR2 second remission; CP chronic phase
- HLA-matched Related donors: Research participants who have an HLA-matched related donor are eligible for entry on this protocol. This will include a healthy related donor who is genotypically or phenotypically matched at all A, B and DRB1 loci, as tested by DNA analysis.
- HLA-compatible Unrelated donors: Research participants who do not have a related HLA-matched donor but have an unrelated donor who is either matched at all A, B and DRB1 loci or who is mismatched at 1/6 loci (A, B, or DRB1) as tested by DNA analysis, will be eligible for entry on this protocol.
- HLA-mismatched Related donors: Research participants who do not have a related or unrelated HLA-compatible donor must have a healthy family member who is at least HLA-haplotype identical to the recipient.
Key exclusion criteria (5 listed)
- Active CNS or skin leukemic/lymphomatous involvement
- Female research participants who are pregnant or breast-feeding
- Active viral, bacterial or fungal infection
- Research participant seropositive for HIV-I/II; HTLV -I/II
- Research participants who have undergone a prior allogeneic or autologous stem cell transplant within the previous six months.
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 |
|---|---|---|
| BUSULFAN, MELPHALAN, FLUDARABINE, G-CSFAll research participants will be conditioned for transplantation with intravenous busulfan (busulfex®) (0.8- 1.0 mg/Kg/dose Q6H x 10 doses), melphalan (70 mg/m2/dose x 2 doses) and fludarabine (25 mg/m2/day x 5 doses). Doses of busulfan will be adjusted according to plasma levels. All research participants will also receive ATG (thymoglobulin®) prior to transplant to promote engraftment.All research participants will also receive G-CSF posttransplant to foster engraftment. The preferred source of stem cells will be peripheral blood stem cells (PBSC) induced and mobilized by treatment of the donor with G-CSF for 5-6 days. PBSC obtained through 2-3 leukaphereses will be Isolex® 300i separated CD34+ stem cell column selected and E-rosette depleted (E-). The CD34+Eperipheral blood progenitors will be administered to the research participants after they have completed cytoreduction. | Drug | 1 |
| BUSULFAN, MELPHALAN, FLUDARABINE, G-CSFAll research participants will be conditioned for transplantation with intravenous busulfan (busulfex®) (0.8- 1.0 mg/Kg/dose Q6H x 10 doses), melphalan (70 mg/m2/dose x 2 doses) and fludarabine (25mg/m2/day x 5 doses). Doses of busulfan will be adjusted according to plasma levels. All research participants will also receive ATG (Thymoglobulin®) prior to transplant to promote engraftment. No drug prophylaxis against GvHD will be administered post transplant. All research participants will also receive G-CSF posttransplant to foster engraftment. The preferred source of stem cells will be peripheral blood stem cells (PBSC) induced and mobilized by treatment of the donor with G-CSF for 5-6 days. PBSC obtained through 2-3 leukaphereses will be Isolex® 300i separated CD34+ stem cell column selected and E-rosette depleted (E-). The CD34+Eperipheral blood progenitors will be administered to the research participants after they have completed cytoreduction. | Drug | 2 |
| BUSULFAN, MELPHALAN, FLUDARABINE, G-CSFAll research participants will be conditioned for transplantation with intravenous busulfan (busulfex®) (0.8- 1.0 mg/Kg/dose Q6H x 10 doses), melphalan (70 mg/m2/dose x 2 doses) and fludarabine (25mg/m2/day x 5 doses). Doses of busulfan will be adjusted according to plasma levels. All research participants will also receive ATG (Thymoglobulin®) prior to transplant to promote engraftment. No drug prophylaxis against GvHD will be administered post transplant. All research participants will also receive G-CSF posttransplant to foster engraftment. The preferred source of stem cells will be peripheral blood stem cells (PBSC) induced and mobilized by treatment of the donor with G-CSF for 5-6 days. PBSC obtained through 2-3 leukaphereses will be Isolex® 300i separated CD34+ stem cell column selected and E-rosette depleted (E-). The CD34+Eperipheral blood progenitors will be administered to the research participants after they have completed cytoreduction. | Drug | 3 |
Primary outcome
Death From GVHD
Time frame: 2 years
To establish the early transplant-related severe morbidity and mortality and 3-the incidence and severity of GvHD.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Feb 2016.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Memorial Sloan-Kettering web siteLink provided by the sponsor.
Related trials for Leukemia
RecruitingPhase 2
Tafasitamab With Acalabrutinib and Venetoclax for the Treatment of Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma
Chronic Lymphocytic Leukemia, Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma
Enrolling by invitationObservational study
Natural History Study of Early Life Exposures in Agriculture (ELEA)
Prostate Cancer, Leukemia
RecruitingPhase 1
A Study of CHM-029 in Participants With NPM1 Mutated, KMT2A or NUP98 Rearranged AML
AML (Acute Myeloid Leukemia)
RecruitingPhase 1
Vididencel in CML-CP Patients With MRD
CML (Chronic Myelogenous Leukemia)
RecruitingPhase 2
Testing New Therapies for Patients With Acute Myeloid Leukemia Who Are Newly Diagnosed and Have Not Yet Started Treatment (A MyeloMATCH Treatment Trial)
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
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.
Farid Boulad, MD principal investigator
Memorial Sloan Kettering Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing BUSULFAN, MELPHALAN, FLUDARABINE, G-CSF in people with Leukemia and Myelodysplastic Syndrome, run by Memorial Sloan Kettering Cancer Center 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 10 inclusion and 5 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 1 Feb 2016. 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 1 Feb 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?
Death From GVHD (time frame: 2 years). 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 1 Feb 2016.