LeukemiaPhase 2NCT01020734
Donor Stem Cell Transplant or Bone Marrow Transplant in Treating Patients With Acute Myeloid Leukemia in Remission
A Phase 2 study testing anti-thymocyte globulin, busulfan and cyclophosphamide in people with Leukemia, run by Asan Medical Center at 2 sites in South Korea.
Trial summary
RATIONALE: Giving chemotherapy before a donor bone marrow or peripheral blood stem cell transplant helps stop the growth of cancer cells. It also helps stop the patient's immune system from rejecting the donor's stem cells. Read more about this study
- Who is it for?
- Ages 15 to 75 years
- Conditions the study is for
- Leukemia
- What is being tested?
- anti-thymocyte globulin, busulfan and cyclophosphamide
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 263 participants
- Where
- 2 sitesSouth Korea
- Study dates
- May 2011to Jul 2015
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 15 to 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
DISEASE CHARACTERISTICS:
- Diagnosis of acute myeloid leukemia (AML) meeting 1 of the following criteria:
- Achieved complete response (CR1) after induction chemotherapy
- Recurrent AML that went into second CR (CR2) after salvage chemotherapy, except those who have undergone prior allogeneic HSCT
- No acute promyelocytic leukemia or acute myeloid leukemia with chromosomal changes t(8;21), inv 16, or t(15;17)
- Must have a donor available meeting one of the following criteria:
- HLA-matched sibling of 65 years or younger
- 6/6 HLA-matched unrelated donor (younger than 55 years) for antigen A, B, and DR
- HLA-mismatched family member (offspring, parents, haploidentical sibling)
PATIENT CHARACTERISTICS:
- Karnofsky performance status 70-100%
- Bilirubin < 2.0 mg/dL
- AST < 3 times the upper limit of normal
- Creatinine < 2.0 mg/dL
- Ejection fraction > 40% on MUGA scan
- Negative pregnancy test
PRIOR CONCURRENT THERAPY:
- See Disease Characteristics
Trial locations and contacts
2 sites in 1 country.
South Korea2 sites
Inje University - Haeundae Paik Hospital
Busan, 612-030
Asan Medical Center - University of Ulsan College of Medicine
Seoul, 138-736
About this study
Official title: Allogeneic Hematopoietic Cell Transplantation for Patients With Acute Myelogenous Leukemia in Remission Using HLA-Matched Sibling Donors, HLA-Matched Unrelated Donors, or HLA-Mismatched Familial Donors - A Phase 2 Study
RATIONALE: Giving chemotherapy before a donor bone marrow or peripheral blood stem cell transplant helps stop the growth of cancer cells. It also helps stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving cyclosporine and methotrexate before and after transplant may stop this from happening.
PURPOSE: This phase II trial is studying how well donor stem cell transplant or bone marrow transplant works in treating patients with acute myeloid leukemia in remission.
Read the detailed description
OBJECTIVES:
Primary
- Evaluate the efficacy of allogeneic hematopoietic stem cell transplantation (HSCT) or bone marrow transplantation (BMT) from a HLA-matched sibling donor, HLA-matched unrelated donor, or HLA-mismatched familial donor, in terms of the frequency of relapse and duration of remission, in patients with acute myeloid leukemia (AML) who have either achieved complete remission (CR1) after induction chemotherapy or who experienced recurrent AML then achieved second CR (CR2) after salvage chemotherapy.
Secondary
- Determine the engraftment, donor chimerism, and secondary graft failure in these patients.
- Assess acute and chronic graft-vs-host disease, immune recovery, and infections in these patients.
- Determine transplantation-related mortality, leukemia-free survival, and overall survival of these patients.
OUTLINE:
- Conditioning chemotherapy and allogeneic bone marrow or hematopoietic stem cell transplantation (HSCT): After completion of induction chemotherapy and a resulting complete response (CR1) or salvage chemotherapy resulting in CR2, patients receive 1 of the following conditioning regimens and transplantations determined by age, co-morbidity, and type of available donor:
- 15 to 55 years of age without significant co-morbidity* undergoing HLA-matched sibling bone marrow transplantation (BMT) (BuCy conditioning): Patients receive busulfan IV once daily on days -7 to -4 and cyclophosphamide IV over 1-2 hours once daily on days -3 and -2. Patients then undergo an allogeneic BMT on day 0.
- Older than 55 years or younger than 55 years with co-morbidity* undergoing HLA-matched sibling BMT; patients of any age undergoing HLA-matched unrelated HSCT; and for patients of any age undergoing HLA-mismatched familial donor HSCT (BuFluATG conditioning): Patients receive busulfan IV once daily on days -7 and -6, fludarabine phosphate IV over 30 minutes once daily on days -7 to -2, anti-thymocyte globulin IV over 4 hours once daily on days -3 to -1, and methylprednisolone IV over 30 minutes once daily on days -4 to -1. Patients then undergo either an allogeneic BMT on day 0 or allogeneic peripheral blood hematopoietic stem cell infusions on days 0-1 or 0-2.
NOTE: *Significant co-morbidity is defined as residual fungal or other infections in the lung or other viscera and residual organ toxicities occurring during induction or consolidation chemotherapy.
- GVHD prophylaxis: Patients receive cyclosporine orally or IV over 2-4 hours twice daily beginning on day -1 followed by a taper starting on day 30 (BuFluATG conditioning) or day 60 (BuCy conditioning). Patients also receive methotrexate IV on days 1, 3, and 6 after the last day of donor cell infusion.
- CNS prophylaxis: Patients receive intrathecal (IT) methotrexate once before conditioning regimen. Patients receive IT methotrexate once every 2 weeks for 3 times after transplantation and platelet recovery. Patients also receive leucovorin calcium orally or IV over 4 hours after IT methotrexate and then once every 6 hours for a total of 8 doses after each dose of IT methotrexate.
After completion of study therapy, patients are followed every 3 months for 3 years and then annually.
- Conditions
- Leukemia
- Being tested
- anti-thymocyte globulin, busulfan, cyclophosphamide, cyclosporine, fludarabine phosphate
- Keywords
- adult acute myeloid leukemia with 11q23 (MLL) abnormalities, adult acute myeloid leukemia with del(5q), adult acute myeloid leukemia with t(16;16)(p13;q22), recurrent adult acute myeloid leukemia, adult acute myeloid leukemia in remission, childhood acute myeloid leukemia in remission, recurrent childhood acute myeloid leukemia, adult acute minimally differentiated myeloid leukemia (M0), childhood acute minimally differentiated myeloid leukemia (M0), adult acute myeloblastic leukemia without maturation (M1), childhood acute myeloblastic leukemia without maturation (M1), adult acute myeloblastic leukemia with maturation (M2), childhood acute myeloblastic leukemia with maturation (M2), adult acute myelomonocytic leukemia (M4), childhood acute myelomonocytic leukemia (M4), adult acute monoblastic leukemia (M5a), adult acute monocytic leukemia (M5b), childhood acute monoblastic leukemia (M5a), childhood acute monocytic leukemia (M5b), adult erythroleukemia (M6a)
- Medical subject headings
- Leukemia, Congenital Abnormalities, Leukemia, Myeloid, Acute, Leukemia, Myelomonocytic, Acute, Leukemia, Monocytic, Acute, Leukemia, Erythroblastic, Acute, Leukemia, Megakaryoblastic, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
anti-thymocyte globulinBiological
busulfanDrug
cyclophosphamideDrug
cyclosporineDrug
methotrexateDrug
allogeneic hematopoietic stem cell transplantationProcedure or surgery
| Study group | Role | What they receive |
|---|---|---|
| transplantation | Experimental | perform allogeneic HCT for patients with AML in CR1; then analyze various pre-transplantation variable, including donor type, for correlation to outcomes Receives: Biological: anti-thymocyte globulin; Drug: busulfan; Drug: cyclophosphamide; Drug: cyclosporine; Drug: fludarabine phosphate; Drug: methotrexate; Procedure: allogeneic hematopoietic stem cell transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Efficacy of the treatment measured in terms of frequency of relapse and duration of remission
duration of CR, leukemia recurrence
Measured: up to 2 years after transplantation
Secondary outcomes (4)
Engraftment
achievement of neutrophil count over 500/ul
Measured: up to 35 days after transplantation
Acute and chronic graft-versus-host disease
Measured: up to 100 days for acute GVHD and up to 2 years for chronic GVHD
Treatment-related mortality
Measured: up to 2 years after transplantation
Leukemia-free survival and overall survival
Measured: up to 2 years after transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Nov 2009 (estimated)
- Study startMay 2011
- Primary completionJul 2015
- Study completionJul 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for LeukemiaStudies of the same condition, open and under way.
- More trials run by Asan Medical 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 263 participants (actual)
Sponsor and investigators
- Lead sponsor
- Asan Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Kyoo-Hyung Lee, Asan Medical Center
- Investigators
- Kyoo H. Lee, MD, Principal investigator, Asan Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01020734
- Sponsor's protocol ID
- CDR0000659891
- Other IDs
- AMC-UUCM-2009-0579
- Status last verified
- Dec 2015
- First submitted
- 24 Nov 2009
- Last updated
- 31 Dec 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 31 Dec 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Allogeneic Hematopoietic Cell Transplantation for Patients With Acute Myelogenous Leukemia in Remission Using HLA-Matched Sibling Donors, HLA-Matched Unrelated Donors, or HLA-Mismatched Familial Donors - A Phase 2 Study
- ClinicalTrials.gov ID
- NCT01020734
- Acronym / sponsor ID
- CDR0000659891
- Conditions
- Leukemia
- Keywords
- adult acute myeloid leukemia with 11q23 (MLL) abnormalities, adult acute myeloid leukemia with del(5q), adult acute myeloid leukemia with t(16;16)(p13;q22), recurrent adult acute myeloid leukemia, adult acute myeloid leukemia in remission, childhood acute myeloid leukemia in remission, recurrent childhood acute myeloid leukemia, adult acute minimally differentiated myeloid leukemia (M0)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 263 participants (actual)
- Lead sponsor
- Asan Medical CenterUniversity, hospital or other
- Study start
- May 2011
- Primary completion
- Jul 2015 (actual)
- Study completion
- Jul 2015 (actual)
- First posted
- 25 Nov 2009 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
RATIONALE: Giving chemotherapy before a donor bone marrow or peripheral blood stem cell transplant helps stop the growth of cancer cells. It also helps stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving cyclosporine and methotrexate before and after transplant may stop this from happening.
PURPOSE: This phase II trial is studying how well donor stem cell transplant or bone marrow transplant works in treating patients with acute myeloid leukemia in remission.
Detailed description
OBJECTIVES:
Primary
- Evaluate the efficacy of allogeneic hematopoietic stem cell transplantation (HSCT) or bone marrow transplantation (BMT) from a HLA-matched sibling donor, HLA-matched unrelated donor, or HLA-mismatched familial donor, in terms of the frequency of relapse and duration of remission, in patients with acute myeloid leukemia (AML) who have either achieved complete remission (CR1) after induction chemotherapy or who experienced recurrent AML then achieved second CR (CR2) after salvage chemotherapy.
Secondary
- Determine the engraftment, donor chimerism, and secondary graft failure in these patients.
- Assess acute and chronic graft-vs-host disease, immune recovery, and infections in these patients.
- Determine transplantation-related mortality, leukemia-free survival, and overall survival of these patients.
OUTLINE:
- Conditioning chemotherapy and allogeneic bone marrow or hematopoietic stem cell transplantation (HSCT): After completion of induction chemotherapy and a resulting complete response (CR1) or salvage chemotherapy resulting in CR2, patients receive 1 of the following conditioning regimens and transplantations determined by age, co-morbidity, and type of available donor:
- 15 to 55 years of age without significant co-morbidity* undergoing HLA-matched sibling bone marrow transplantation (BMT) (BuCy conditioning): Patients receive busulfan IV once daily on days -7 to -4 and cyclophosphamide IV over 1-2 hours once daily on days -3 and -2. Patients then undergo an allogeneic BMT on day 0.
- Older than 55 years or younger than 55 years with co-morbidity* undergoing HLA-matched sibling BMT; patients of any age undergoing HLA-matched unrelated HSCT; and for patients of any age undergoing HLA-mismatched familial donor HSCT (BuFluATG conditioning): Patients receive busulfan IV once daily on days -7 and -6, fludarabine phosphate IV over 30 minutes once daily on days -7 to -2, anti-thymocyte globulin IV over 4 hours once daily on days -3 to -1, and methylprednisolone IV over 30 minutes once daily on days -4 to -1. Patients then undergo either an allogeneic BMT on day 0 or allogeneic peripheral blood hematopoietic stem cell infusions on days 0-1 or 0-2.
NOTE: *Significant co-morbidity is defined as residual fungal or other infections in the lung or other viscera and residual organ toxicities occurring during induction or consolidation chemotherapy.
- GVHD prophylaxis: Patients receive cyclosporine orally or IV over 2-4 hours twice daily beginning on day -1 followed by a taper starting on day 30 (BuFluATG conditioning) or day 60 (BuCy conditioning). Patients also receive methotrexate IV on days 1, 3, and 6 after the last day of donor cell infusion.
- CNS prophylaxis: Patients receive intrathecal (IT) methotrexate once before conditioning regimen. Patients receive IT methotrexate once every 2 weeks for 3 times after transplantation and platelet recovery. Patients also receive leucovorin calcium orally or IV over 4 hours after IT methotrexate and then once every 6 hours for a total of 8 doses after each dose of IT methotrexate.
After completion of study therapy, patients are followed every 3 months for 3 years and then annually.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| transplantation | Experimental | Biological: anti-thymocyte globulin, Drug: busulfan, Drug: cyclophosphamide, Drug: cyclosporine, Drug: fludarabine phosphate, Drug: methotrexate, Procedure: allogeneic hematopoietic stem cell transplantationperform allogeneic HCT for patients with AML in CR1; then analyze various pre-transplantation variable, including donor type, for correlation to outcomes |
Who may qualify
- Ages
- 15 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
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 |
|---|---|---|
| anti-thymocyte globulin | Biological | transplantation |
| busulfan | Drug | transplantation |
| cyclophosphamide | Drug | transplantation |
| cyclosporine | Drug | transplantation |
| fludarabine phosphate | Drug | transplantation |
| methotrexate | Drug | transplantation |
| allogeneic hematopoietic stem cell transplantation | Procedure or surgery | transplantation |
Primary outcome
Efficacy of the treatment measured in terms of frequency of relapse and duration of remission
Time frame: up to 2 years after transplantation
duration of CR, leukemia recurrence
4 secondary outcomes
Engraftment
Time frame: up to 35 days after transplantation
Acute and chronic graft-versus-host disease
Time frame: up to 100 days for acute GVHD and up to 2 years for chronic GVHD
Treatment-related mortality
Time frame: up to 2 years after transplantation
Leukemia-free survival and overall survival
Time frame: up to 2 years after transplantation
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Dec 2015.
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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.
Kyoo H. Lee, MD principal investigator
Asan Medical Center
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing anti-thymocyte globulin, busulfan and cyclophosphamide in people with Leukemia, run by Asan Medical Center at 2 sites in South Korea.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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 31 Dec 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in South Korea. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Efficacy of the treatment measured in terms of frequency of relapse and duration of remission (time frame: up to 2 years after transplantation). 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 31 Dec 2015.