Leukemia, Lymphocytic, AcutePhase 2/3NCT00176839
Stem Cell Transplantation for Hematological Malignancies
A Phase 2/3 study testing Stem Cell Transplant, Busulfan and Cyclophosphamide in people with Leukemia, Lymphocytic, Acute and AML, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
Trial summary
This protocol using busulfan, cyclophosphamide and melphalan has been designed as conditioning therapy for patients receiving stem cell transplantation for acute leukemia or myelodysplastic syndrome (MDS). The hypothesis is that this new regimen will be well tolerated and will cure the patient. Read more about this study
- Who is it for?
- Ages Up to 35 years
- Conditions the study is for
- Leukemia, Lymphocytic, Acute, AML, MDS
- What is being tested?
- Stem Cell Transplant, Busulfan and Cyclophosphamide
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 1 siteUnited States
- Study dates
- 7 Jun 2000to Feb 2012
- Phase
- Phase 2/3
- 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 35 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients must have a diagnosis of acute lymphocytic leukemia (ALL), acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) and currently be in complete remission.
- Patients must be either:
- - <18 years of age who are at least 6 months after initial hematopoietic cell transplant (HCT),
- - 19-35 years of age and at least 18 months after initial HCT, or
- - <35 years of age and have received sufficient radiation treatment to be ineligible for total body irradiation (TBI) containing preparative therapy
- Adequate major organ function including:
Show all 12Show fewer
- - Cardiac: ejection fraction > or = 45%
- - Renal: creatinine clearance > or = 40 mL/min
- - Hepatic: no clinical evidence of hepatic failure (e.g. coagulopathy, ascites)
- - Karnofsky performance status > or = 70% or Lansky score > or = 50%
- Women of child bearing age must be using adequate birth control and have a negative pregnancy test.
- Written informed consent.
Who cannot join
- Eligible for TBI containing preparative regimen.
- Active uncontrolled infection within one week of HCT.
- Pregnant or lactating females.
Trial locations and contacts
1 site in 1 country.
United States1 site
Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, 55455
About this study
Official title: Busulfan, Cyclophosphamide, and Melphalan Followed by Allogeneic Hematopoietic Cell Transplantation in Patients With Hematological Malignancies
This protocol using busulfan, cyclophosphamide and melphalan has been designed as conditioning therapy for patients receiving stem cell transplantation for acute leukemia or myelodysplastic syndrome (MDS). The hypothesis is that this new regimen will be well tolerated and will cure the patient.
Read the detailed description
Subjects will be admitted to the bone marrow transplant unit and put in isolation to reduce exposure to infectious agents.
Prior to transplantation, they will receive BUSULFAN via the central venous line, four times a day for four days, CYCLOPHOSPHAMIDE via the central venous line once a day for two days, and MELPHALAN via the central venous line for one day. Busulfan, cyclophosphamide, and melphalan are given to destroy the subject's cancer. As well, these drugs will destroy their immune system to help ensure the new stem cells take and grow after transplantation.
On the day of transplantation, umbilical cord blood from the donor will be transfused via venous line. These new cells will replace the subject's bone marrow.
After transplantation, the subjects will receive Cyclosporin A and either MMF or MTX
Isolation will be continued until adequate numbers of cells are present in the blood to fight infection. Subjects will be discharged from the hospital when medically ready. They will be expected to return for follow-up to the blood and marrow transplant clinic at specific dates as determined by physicians.
- Conditions
- Leukemia, Lymphocytic, Acute, AML, MDS
- Being tested
- Stem Cell Transplant, Busulfan, Cyclophosphamide, Melphalan, G-CSF
- Keywords
- Stem Cell transplant, retransplant, hematological malignancies
- Medical subject headings
- Precursor Cell Lymphoblastic Leukemia-Lymphoma, Hematologic Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Stem Cell TransplantProcedure or surgery
Also called Bone Marrow Transplant, umbilical cord transplant, hematopoietic stem cell transplant
Certain cancers can be treated by giving patients stem cells that come from someone else. This is called a stem-cell transplant. As part of the transplant process, patients receive high doses of chemotherapy and/or radiation to treat their underlying disease, such as cancer. As one of its effects, this treatment also kills the healthy stem cells that are already in the marrow. The transplant provides new stem cells for the patient from a healthy donor; that replace the bone marrow and allow the blood counts to recover.
BusulfanDrug
Also called Busulfex, Myleran
Prior to transplantation, subjects will receive BUSULFAN via the central venous line, four times a day for four days (days -7 through -4).
CyclophosphamideDrug
Also called Cytoxan, Neosar
Prior to stem cell transplantation, subjects will receive CYCLOPHOSPHAMIDE via the central venous line once a day for two days on days -3 and -2.
MelphalanDrug
Also called Alkeran
MELPHALAN will be given via the central venous line for one day, on day -1, prior to stem cell transplantation.
G-CSFDrug
Also called granulocyte colony-stimulating factor, Filgrastim, Neupogen
G-CSF is to be given daily IV beginning on day +1 until ANC 2.5 x 109/L.
ATGDrug
Also called antithymocyte globulin
ATG will be administered to umbilical cord blood recipients.
| Study group | Role | What they receive |
|---|---|---|
| Treatment Arm | Experimental | Patients treated with therapy plan consisting of Busulfan every 6 hours on days -7 through -4, Cyclophosphamide 60 mg/kg/day IV x 2 days, Melphalan 140 mg/m on day -1, antithymocyte globulin (ATG), G-CSF (granulocyte colony-stimulating factor) and stem cell transplantation on day 0. Receives: Procedure: Stem Cell Transplant; Drug: Busulfan; Drug: Cyclophosphamide; Drug: Melphalan; Drug: G-CSF; Drug: ATG |
What is being measured
The primary outcome is the main question the study is designed to answer.
Probability of Long-term Disease-free Survival (DFS)
Number of participants with long-term disease free survival after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies.
Measured: 1 year
Secondary outcomes (5)
Probability of Engraftment
Number of participants with engraftment after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies..
Measured: 1 year
Incidence of Acute Graft-versus-host Disease (GVHD)
Number of participants with acute GVHD after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies.
Measured: 100 days post-transplant
Incidence Chronic Graft-versus-host Disease (GVHD)
Number of participants with chronic GVHD after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies.
Measured: 1 year
Incidence of Regimen-related Toxicity 100 Days Post Transplant
Number of participants with regimen-related toxicity 100 days post transplant after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies.
Measured: 100 days post-transplant
Incidence of Relapse
Number of patients with relapse after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Sep 2005 (estimated)
- Study start7 Jun 2000
- Primary completionFeb 2012
- Study completionFeb 2012
Why it stopped: Replaced by a different study
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 Leukemia, Lymphocytic, AcuteStudies of the same condition, open and under way.
- 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 2/3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Margaret MacMillan, MD, Principal investigator, Masonic Cancer Center, University of Minnesota
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00176839
- Sponsor's protocol ID
- 2000LS040
- Other IDs
- MT2000-12, 0005M52481
- Status last verified
- Dec 2017
- First submitted
- 12 Sep 2005
- Last updated
- 5 Dec 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 5 Dec 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Busulfan, Cyclophosphamide, and Melphalan Followed by Allogeneic Hematopoietic Cell Transplantation in Patients With Hematological Malignancies
- ClinicalTrials.gov ID
- NCT00176839
- Acronym / sponsor ID
- 2000LS040
- Conditions
- Leukemia, Lymphocytic, Acute, AML, MDS
- Keywords
- Stem Cell transplant, retransplant, hematological malignancies
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Replaced by a different study
- Enrolment
- 11 participants (actual)
- Lead sponsor
- Masonic Cancer Center, University of MinnesotaUniversity, hospital or other
- Study start
- 7 Jun 2000 (actual)
- Primary completion
- Feb 2012 (actual)
- Study completion
- Feb 2012 (actual)
- First posted
- 15 Sep 2005 (estimated)
- Results
- Posted 13 Feb 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This protocol using busulfan, cyclophosphamide and melphalan has been designed as conditioning therapy for patients receiving stem cell transplantation for acute leukemia or myelodysplastic syndrome (MDS). The hypothesis is that this new regimen will be well tolerated and will cure the patient.
Detailed description
Subjects will be admitted to the bone marrow transplant unit and put in isolation to reduce exposure to infectious agents.
Prior to transplantation, they will receive BUSULFAN via the central venous line, four times a day for four days, CYCLOPHOSPHAMIDE via the central venous line once a day for two days, and MELPHALAN via the central venous line for one day. Busulfan, cyclophosphamide, and melphalan are given to destroy the subject's cancer. As well, these drugs will destroy their immune system to help ensure the new stem cells take and grow after transplantation.
On the day of transplantation, umbilical cord blood from the donor will be transfused via venous line. These new cells will replace the subject's bone marrow.
After transplantation, the subjects will receive Cyclosporin A and either MMF or MTX
Isolation will be continued until adequate numbers of cells are present in the blood to fight infection. Subjects will be discharged from the hospital when medically ready. They will be expected to return for follow-up to the blood and marrow transplant clinic at specific dates as determined by physicians.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment Arm | Experimental | Procedure: Stem Cell Transplant, Drug: Busulfan, Drug: Cyclophosphamide, Drug: Melphalan, Drug: G-CSF, Drug: ATGPatients treated with therapy plan consisting of Busulfan every 6 hours on days -7 through -4, Cyclophosphamide 60 mg/kg/day IV x 2 days, Melphalan 140 mg/m on day -1, antithymocyte globulin (ATG), G-CSF (granulocyte colony-stimulating factor) and stem cell transplantation on day 0. |
Who may qualify
- Ages
- Up to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (12 listed)
- Patients must have a diagnosis of acute lymphocytic leukemia (ALL), acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) and currently be in complete remission.
- Patients must be either:
- - <18 years of age who are at least 6 months after initial hematopoietic cell transplant (HCT),
- - 19-35 years of age and at least 18 months after initial HCT, or
- - <35 years of age and have received sufficient radiation treatment to be ineligible for total body irradiation (TBI) containing preparative therapy
Key exclusion criteria (3 listed)
- Eligible for TBI containing preparative regimen.
- Active uncontrolled infection within one week of HCT.
- Pregnant or lactating females.
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 |
|---|---|---|
| Stem Cell TransplantAlso: Bone Marrow Transplant, umbilical cord transplant, hematopoietic stem cell transplantCertain cancers can be treated by giving patients stem cells that come from someone else. This is called a stem-cell transplant. As part of the transplant process, patients receive high doses of chemotherapy and/or radiation to treat their underlying disease, such as cancer. As one of its effects, this treatment also kills the healthy stem cells that are already in the marrow. The transplant provides new stem cells for the patient from a healthy donor; that replace the bone marrow and allow the blood counts to recover. | Procedure or surgery | Treatment Arm |
| BusulfanAlso: Busulfex, MyleranPrior to transplantation, subjects will receive BUSULFAN via the central venous line, four times a day for four days (days -7 through -4). | Drug | Treatment Arm |
| CyclophosphamideAlso: Cytoxan, NeosarPrior to stem cell transplantation, subjects will receive CYCLOPHOSPHAMIDE via the central venous line once a day for two days on days -3 and -2. | Drug | Treatment Arm |
| MelphalanAlso: AlkeranMELPHALAN will be given via the central venous line for one day, on day -1, prior to stem cell transplantation. | Drug | Treatment Arm |
| G-CSFAlso: granulocyte colony-stimulating factor, Filgrastim, NeupogenG-CSF is to be given daily IV beginning on day +1 until ANC 2.5 x 109/L. | Drug | Treatment Arm |
| ATGAlso: antithymocyte globulinATG will be administered to umbilical cord blood recipients. | Drug | Treatment Arm |
Primary outcome
Probability of Long-term Disease-free Survival (DFS)
Time frame: 1 year
Number of participants with long-term disease free survival after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies.
5 secondary outcomes
Probability of Engraftment
Time frame: 1 year
Incidence of Acute Graft-versus-host Disease (GVHD)
Time frame: 100 days post-transplant
Incidence Chronic Graft-versus-host Disease (GVHD)
Time frame: 1 year
Incidence of Regimen-related Toxicity 100 Days Post Transplant
Time frame: 100 days post-transplant
Incidence of Relapse
Time frame: 1 year
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Dec 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.
Margaret MacMillan, MD principal investigator
Masonic Cancer Center, University of Minnesota
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Stem Cell Transplant, Busulfan and Cyclophosphamide in people with Leukemia, Lymphocytic, Acute and AML, run by Masonic Cancer Center, University of Minnesota 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 12 inclusion and 3 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 5 Dec 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.
What does this combined 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.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 13 Feb 2013). 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?
Probability of Long-term Disease-free Survival (DFS) (time frame: 1 year). 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 5 Dec 2017.