LeukemiaPhase 1/2NCT00002831
Chemotherapy Plus Peripheral Stem Cell Transplantation in Treating Patients With Chronic Myelogenous or Acute Leukemia
A Phase 1/2 study testing Filgrastim, Busulfan and Cyclophosphamide in people with Leukemia, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more cancer cells. Read more about this study
- Who is it for?
- Ages 15 to 55 years
- Conditions the study is for
- Leukemia
- What is being tested?
- Filgrastim, Busulfan and Cyclophosphamide
- Type of treatment
- Pill or oral medicine, Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Aug 1995to 31 Dec 2002
- Phase
- Phase 1/2
- 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 55 years
- All sexes
- No healthy volunteers
- Child, Adult
DISEASE CHARACTERISTICS: Acute leukemia past first remission or induction failure Chronic myelogenous leukemia in accelerated phase or blast crisis
PATIENT CHARACTERISTICS: Age: 15 to 55 Performance status: Zubrod 0-2 Life expectancy: Life expectancy not severely limited by concurrent illness Hematopoietic: Not specified Hepatic: No evidence of chronic active hepatitis or cirrhosis Bilirubin no greater than 2 times upper limit of normal SGPT no greater than 4 times upper limit of normal Renal: Creatinine no greater than 1.5 mg/dL Cardiovascular: Left ventricular ejection fraction at least 50% No uncontrolled arrhythmias or symptomatic cardiac disease Pulmonary: FEV1, FVC, and DLCO at least 50% No symptomatic pulmonary disease Other: Related donor who is HLA-identical required No effusion or ascites greater than 1 L prior to drainage HIV negative Not pregnant No active CNS disease
PRIOR CONCURRENT THERAPY: Not specified
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Texas - MD Anderson Cancer Center
Houston, Texas, 77030
About this study
Official title: A Phase I/II Study of High-Dose Deoxyazacytidine, Busulfan, and Cyclophosphamide With Allogeneic Stem Cell Transplantation for Hematologic Malignancies
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more cancer cells.
PURPOSE: Phase I/II trial to study the effectiveness of high-dose chemotherapy plus peripheral stem cell transplantation in treating patients with chronic myelogenous or acute leukemia.
Read the detailed description
OBJECTIVES: I. Determine the maximum tolerated dose of decitabine in combination with busulfan and cyclophosphamide in patients with hematologic malignancies. II. Establish the pharmacokinetics of decitabine and busulfan in this patient population. III. Determine the effectiveness of this combination in achieving durable complete remission in patients with chronic myelogenous leukemia (CML) in blast crisis or acute myelogenous leukemia (AML) in relapse undergoing allogeneic stem cell transplantation.
OUTLINE: In cohorts of 3, patients receive escalating doses of decitabine (DAC) IV over 4 hours on days -8 and -7. Busulfan is administered orally every 6 hours on consecutive days -6 through -4. Cyclophosphamide is given by vein (IV) over 1 hour on consecutive days -3 and -2. The maximum tolerated dose of DAC is defined as the dose at which 2 patients experience dose limiting toxicity. Donors receive filgrastim subcutaneously (SQ) daily every 12 hours starting 2-4 days prior to the first stem cell collection and before DAC infusion. Leukapheresis is conducted daily. If insufficient number of cells are collected, blood marrow is harvested for supplementation. Stem cells are infused on day 0. For graft vs host disease prophylaxis (GVHD), patients receive tacrolimus IV beginning one day before stem cell infusion, then orally following tolerance to tacrolimus. Patients intolerant to tacrolimus receive cyclosporine IV beginning on day -2, then orally following tolerance and engraftment. All patients receive methylprednisolone given according to clinical grade of GVHD procedures. For CNS prophylaxis, methotrexate is given intrathecally or intraventricularly monthly, beginning on the second month through the eighth month of treatment. Allogeneic patients are followed until the end of 1 year.
PROJECTED ACCRUAL: An estimated 30 allogeneic recipients will be recruited in 2 years for the expected study duration of 2-3 years.
- Conditions
- Leukemia
- Being tested
- Filgrastim, Busulfan, Cyclophosphamide, Cyclosporine, Decitabine (DAC)
- Keywords
- recurrent adult acute myeloid leukemia, accelerated phase chronic myelogenous leukemia, blastic phase chronic myelogenous leukemia
- Medical subject headings
- Leukemia, Leukemia, Myeloid, Acute, Leukemia, Myeloid, Accelerated Phase, Blast Crisis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FilgrastimBiological
Also called C-CSF, Neupogen
Subcutaneously (SQ) daily every 12 hours starting 2-4 days prior to the first stem cell collection and before DAC infusion.
BusulfanDrug
Also called Busulfex, Myleran
Administered orally every 6 hours on consecutive days -6 through -4.
CyclophosphamideDrug
Also called Cytoxan, Neosar
Given intravenously (IV) over 1 hour on consecutive days -3 and -2.
CyclosporineDrug
Also called Sandimmune, CYA, Cyclosporin A
Patients intolerant to tacrolimus receive cyclosporine IV beginning on day -2, then orally following tolerance and engraftment.
Decitabine (DAC)Drug
Also called Dacogen
IV over 4 hours on days -8 and -7.
MethotrexateDrug
Given intrathecally or intraventricularly monthly, beginning on the second month through the eighth month of treatment.
Also called Depo-Medrol, Medrol, Solu-Medrol
Given according to clinical grade of GVHD procedures.
TacrolimusDrug
Also called Prograf
IV beginning one day before stem cell infusion, then orally following tolerance to tacrolimus.
Allogeneic Bone Marrow TransplantationProcedure or surgery
Also called ABMT
Infusion of stem cells on Day 0.
Peripheral Blood Stem Cell TransplantationProcedure or surgery
Also called PBSCT
Stem cell infusion on Day 0.
| Study group | Role | What they receive |
|---|---|---|
| Deoxyazacytidine + Busulfan + Cyclophosphamide | Experimental | Deoxyazacytidine + Busulfan + Cyclophosphamide With Allogeneic Stem Cell Transplantation Receives: Biological: Filgrastim; Drug: Busulfan; Drug: Cyclophosphamide; Drug: Cyclosporine; Drug: Decitabine (DAC); Drug: Methotrexate; Drug: Methylprednisolone; Drug: Tacrolimus; Procedure: Allogeneic Bone Marrow Transplantation; Procedure: Peripheral Blood Stem Cell Transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum Tolerated Dose
Measured: Study Duration 3 Years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jul 2004 (estimated)
- Study start1 Aug 1995
- Primary completion31 Dec 2002
- Study completion31 Dec 2002
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 M.D. Anderson 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Sergio Giralt, MD, Study chair, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00002831
- Sponsor's protocol ID
- DM94-064
- Other IDs
- P30CA016672, MDA-DM-94064, NCI-G96-0999, CDR0000065033
- Status last verified
- Oct 2018
- First submitted
- 1 Nov 1999
- Last updated
- 26 Oct 2018
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Oct 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Study of High-Dose Deoxyazacytidine, Busulfan, and Cyclophosphamide With Allogeneic Stem Cell Transplantation for Hematologic Malignancies
- ClinicalTrials.gov ID
- NCT00002831
- Acronym / sponsor ID
- DM94-064
- Conditions
- Leukemia
- Keywords
- recurrent adult acute myeloid leukemia, accelerated phase chronic myelogenous leukemia, blastic phase chronic myelogenous leukemia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- 1 Aug 1995 (actual)
- Primary completion
- 31 Dec 2002 (actual)
- Study completion
- 31 Dec 2002 (actual)
- First posted
- 29 Jul 2004 (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: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more cancer cells.
PURPOSE: Phase I/II trial to study the effectiveness of high-dose chemotherapy plus peripheral stem cell transplantation in treating patients with chronic myelogenous or acute leukemia.
Detailed description
OBJECTIVES: I. Determine the maximum tolerated dose of decitabine in combination with busulfan and cyclophosphamide in patients with hematologic malignancies. II. Establish the pharmacokinetics of decitabine and busulfan in this patient population. III. Determine the effectiveness of this combination in achieving durable complete remission in patients with chronic myelogenous leukemia (CML) in blast crisis or acute myelogenous leukemia (AML) in relapse undergoing allogeneic stem cell transplantation.
OUTLINE: In cohorts of 3, patients receive escalating doses of decitabine (DAC) IV over 4 hours on days -8 and -7. Busulfan is administered orally every 6 hours on consecutive days -6 through -4. Cyclophosphamide is given by vein (IV) over 1 hour on consecutive days -3 and -2. The maximum tolerated dose of DAC is defined as the dose at which 2 patients experience dose limiting toxicity. Donors receive filgrastim subcutaneously (SQ) daily every 12 hours starting 2-4 days prior to the first stem cell collection and before DAC infusion. Leukapheresis is conducted daily. If insufficient number of cells are collected, blood marrow is harvested for supplementation. Stem cells are infused on day 0. For graft vs host disease prophylaxis (GVHD), patients receive tacrolimus IV beginning one day before stem cell infusion, then orally following tolerance to tacrolimus. Patients intolerant to tacrolimus receive cyclosporine IV beginning on day -2, then orally following tolerance and engraftment. All patients receive methylprednisolone given according to clinical grade of GVHD procedures. For CNS prophylaxis, methotrexate is given intrathecally or intraventricularly monthly, beginning on the second month through the eighth month of treatment. Allogeneic patients are followed until the end of 1 year.
PROJECTED ACCRUAL: An estimated 30 allogeneic recipients will be recruited in 2 years for the expected study duration of 2-3 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Deoxyazacytidine + Busulfan + Cyclophosphamide | Experimental | Biological: Filgrastim, Drug: Busulfan, Drug: Cyclophosphamide, Drug: Cyclosporine, Drug: Decitabine (DAC), Drug: Methotrexate, Drug: Methylprednisolone, Drug: Tacrolimus, Procedure: Allogeneic Bone Marrow Transplantation, Procedure: Peripheral Blood Stem Cell TransplantationDeoxyazacytidine + Busulfan + Cyclophosphamide With Allogeneic Stem Cell Transplantation |
Who may qualify
- Ages
- 15 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, 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 |
|---|---|---|
| FilgrastimAlso: C-CSF, NeupogenSubcutaneously (SQ) daily every 12 hours starting 2-4 days prior to the first stem cell collection and before DAC infusion. | Biological | Deoxyazacytidine + Busulfan + Cyclophosphamide |
| BusulfanAlso: Busulfex, MyleranAdministered orally every 6 hours on consecutive days -6 through -4. | Drug | Deoxyazacytidine + Busulfan + Cyclophosphamide |
| CyclophosphamideAlso: Cytoxan, NeosarGiven intravenously (IV) over 1 hour on consecutive days -3 and -2. | Drug | Deoxyazacytidine + Busulfan + Cyclophosphamide |
| CyclosporineAlso: Sandimmune, CYA, Cyclosporin APatients intolerant to tacrolimus receive cyclosporine IV beginning on day -2, then orally following tolerance and engraftment. | Drug | Deoxyazacytidine + Busulfan + Cyclophosphamide |
| Decitabine (DAC)Also: DacogenIV over 4 hours on days -8 and -7. | Drug | Deoxyazacytidine + Busulfan + Cyclophosphamide |
| MethotrexateGiven intrathecally or intraventricularly monthly, beginning on the second month through the eighth month of treatment. | Drug | Deoxyazacytidine + Busulfan + Cyclophosphamide |
| MethylprednisoloneAlso: Depo-Medrol, Medrol, Solu-MedrolGiven according to clinical grade of GVHD procedures. | Drug | Deoxyazacytidine + Busulfan + Cyclophosphamide |
| TacrolimusAlso: PrografIV beginning one day before stem cell infusion, then orally following tolerance to tacrolimus. | Drug | Deoxyazacytidine + Busulfan + Cyclophosphamide |
| Allogeneic Bone Marrow TransplantationAlso: ABMTInfusion of stem cells on Day 0. | Procedure or surgery | Deoxyazacytidine + Busulfan + Cyclophosphamide |
| Peripheral Blood Stem Cell TransplantationAlso: PBSCTStem cell infusion on Day 0. | Procedure or surgery | Deoxyazacytidine + Busulfan + Cyclophosphamide |
Primary outcome
Maximum Tolerated Dose
Time frame: Study Duration 3 Years
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 26 Oct 2018.
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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.
Sergio Giralt, MD study chair
M.D. Anderson 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 1/2 study testing Filgrastim, Busulfan and Cyclophosphamide in people with Leukemia, run by M.D. Anderson 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?
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 26 Oct 2018. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Maximum Tolerated Dose (time frame: Study Duration 3 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 26 Oct 2018; AzTrials last refreshed its copy on 6 Nov 2026.