LeukemiaPhase 2NCT00534430
Busulfan, Etoposide, and Total-Body Irradiation in Treating Patients Undergoing Donor Stem Cell or Bone Marrow Transplant for Advanced Hematologic Cancer
A Phase 2 study testing busulfan, cyclosporine and etoposide in people with Leukemia and Myelodysplastic Syndromes, run by City of Hope Medical Center.
Trial summary
RATIONALE: Giving chemotherapy and total-body irradiation before a donor stem cell transplant or a donor bone marrow transplant helps stop the growth of cancer and abnormal cells and helps stop the patient's immune system from rejecting the donor's stem cells. Read more about this study
- Who is it for?
- Ages 16 to 50 years
- Conditions the study is for
- Leukemia, Myelodysplastic Syndromes
- What is being tested?
- busulfan, cyclosporine and etoposide
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- No sites listed yet
- Study dates
- 29 Feb 2000to 2 Feb 2026
- Phase
- Phase 2
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 16 to 50 years
- All sexes
- No healthy volunteers
- Child, Adult
DISEASE CHARACTERISTICS:
- Diagnosis of 1 of the following:
- Acute myeloid leukemia (AML)
- Failed remission induction therapy or in relapse beyond second remission
- In first remission with poor risk cytogenetics (e.g., 11q abnormalities, -7, -5, complex abnormalities [i.e., > 3 abnormalities, 6;9 translocation and 3q abnormalities del (7q), del (5q), complex abnormalities ≥ abnormalities, 9q, 20q, 21q, 17q, t(9;21)])
- Acute lymphoblastic leukemia (ALL)
- Failed remission induction therapy or in relapse beyond second remission
- Blastic phase chronic myelogenous leukemia
- Refractory anemia with excess blasts
- Refractory anemia with excess blasts in transformation
- HLA -A, -B, -C, -DR identical sibling donor match available
- No relapse after prior bone marrow transplantation
PATIENT CHARACTERISTICS:
- Cardiac ejection fraction ≥ 50%
- Serum creatinine ≤ 1.2 times upper limit of normal (ULN) or creatinine clearance > 80 mL/min
- Bilirubin ≤ 1.5 times ULN
- AST and ALT < 5 times ULN
- FEV_1 ≥ 50% of predicted normal
- DLCO ≥ 50% of predicted normal
- No psychological or medical condition that would preclude allogeneic transplantation (in the opinion of the treating physician)
- Not pregnant
- Negative pregnancy test
PRIOR CONCURRENT THERAPY:
- See Disease Characteristics
- At least 28 days since prior induction or reinduction therapy
- Prior etoposide and busulfan allowed
- No prior radiation therapy that would exclude total-body irradiation
About this study
Official title: Phase II Study of IV Busulfan Combined With 12 cGy of Fractionated Total Body Irradiation (FTBI) and Etoposide (VP-16) as a Preparative Regimen for Allogeneic Bone Marrow Transplantation for Patients With Advanced Hematological Malignancies
RATIONALE: Giving chemotherapy and total-body irradiation before a donor stem cell transplant or a donor bone marrow transplant helps stop the growth of cancer and abnormal cells and 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 mycophenolate mofetil and cyclosporine before and after transplant may stop this from happening.
PURPOSE: This phase II trial is studying the side effects and best way to give busulfan together with etoposide and total-body irradiation and to see how well they work in treating patients who are undergoing a donor stem cell or bone marrow transplant for advanced hematologic cancer.
Read the detailed description
OBJECTIVES:
- To determine the efficacy of a preparative regimen comprising dose targeted busulfan, etoposide, and fractionated total-body irradiation followed by allogeneic hematopoietic stem cell or bone marrow transplantation in patients with advanced hematologic malignancies.
- To determine the efficacy of this regimen in patients with acute myeloid leukemia in first remission with unfavorable cytogenetics.
- To evaluate the early and late toxicities of this regimen.
OUTLINE:
- Preparative chemotherapy regimen: Patients receive busulfan IV over 2 hours once every 6 hours on days -14 to -8 for a total of 16 doses and etoposide IV on day -3.* NOTE: *Patients also receive oral or IV dilantin 1-3 times daily on days -18 to -5 for prophylaxis of grand mal seizures.
- Fractionated total-body irradiation (FTBI): Patients undergo FTBI on days -7 to -4 for a total of 10 fractions.
- Allogeneic transplantation: Patients undergo allogeneic peripheral blood stem cell transplantation or bone marrow transplantation on day 0.
- Graft-versus-host disease (GVHD) prophylaxis: Patients receive cyclosporine IV or orally on days -1 to 50 followed by a taper to day 180 in the absence of GVHD. Patients also receive mycophenolate mofetil orally or IV over 2 hours twice daily on days 0-27, followed by a taper until day 56.
After completion of study treatment, patients are followed annually for 2 years.
- Conditions
- Leukemia, Myelodysplastic Syndromes
- Being tested
- busulfan, cyclosporine, etoposide, mycophenolate mofetil, allogeneic bone marrow transplantation
- Keywords
- adult acute myeloid leukemia with 11q23 (MLL) abnormalities, adult acute myeloid leukemia with inv(16)(p13;q22), adult acute myeloid leukemia with t(15;17)(q22;q12), adult acute myeloid leukemia with t(16;16)(p13;q22), adult acute myeloid leukemia with t(8;21)(q22;q22), recurrent adult acute myeloid leukemia, adult acute myeloid leukemia in remission, recurrent childhood acute myeloid leukemia, recurrent adult acute lymphoblastic leukemia, recurrent childhood acute lymphoblastic leukemia, blastic phase chronic myelogenous leukemia, refractory anemia with excess blasts in transformation, refractory anemia with excess blasts, previously treated myelodysplastic syndromes, secondary myelodysplastic syndromes
- Medical subject headings
- Leukemia, Myelodysplastic Syndromes, Congenital Abnormalities, Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Blast Crisis, Anemia, Refractory, with Excess of Blasts
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
busulfanDrug
cyclosporineDrug
etoposideDrug
allogeneic bone marrow transplantationProcedure or surgery
allogeneic hematopoietic stem cell transplantationProcedure or surgery
peripheral blood stem cell transplantationProcedure or surgery
total-body irradiationRadiation
| Study group | Role | What they receive |
|---|---|---|
| Busulfan, FTBI and VP16 | Experimental | IV Busulfan + 12 cGy FTBI + VP16 prior to allogeneic Bone Marrow Transplant Receives: Drug: busulfan; Drug: cyclosporine; Drug: etoposide; Drug: mycophenolate mofetil; Procedure: allogeneic bone marrow transplantation; Procedure: allogeneic hematopoietic stem cell transplantation; Procedure: peripheral blood stem cell transplantation; Radiation: total-body irradiation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Survival at 5 Years Post-Transplant.
Kaplan-Meier estimate of an event of death estimated at five years post-transplant. 95% confidence intervals were calculated from the logit transform of the Greenwood variance. The transformation was necessary to keep the estimate within the probability space of 0 to 100%.
Measured: Date of Transplant to Five Years post-Transplant
Disease-free Survival at Five Years Post-transplant
Kaplan-Meier estimate of an event of relapse or death estimated at five years post-transplant. 95% confidence intervals were calculated from the logit transform of the Greenwood variance. The transformation was necessary to keep the estimate within the probability space of 0 to 100%.
Measured: Date of transplant to five years post-transplant
Overall Survival Comparing Diagnosis Groups
Kaplan-Meier estimate of an event of death estimated at five years post-transplant. 95% confidence intervals were calculated from the logit transform of the Greenwood variance. The transformation was necessary to keep the estimate within the probability space of 0 to 100%.
Measured: Date of Transplant to Five Years post-Transplant
Cumulative Incidence of Relapse With Transplant-related Death as the Competing Risk: Diagnosis Groups Are Compared.
Fine and Gray estimate of cumulative incidence of Relapse, with Death as the competing risk. Estimate is at five years post-transplant. Ninety-five percent confidence interval is by logit transformation of Greenwood variance to keep the interval within the probability space of 0% to 100%.
Measured: Date of Transplant to Five Years post-Transplant. Estimate is at Five Years post-Transplant.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Sep 2007 (estimated)
- Study start29 Feb 2000
- Primary completion12 Apr 2010
- Study completion2 Feb 2026
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 City of Hope 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
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- City of Hope Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Anthony S. Stein, MD, Study chair, City of Hope Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00534430
- Sponsor's protocol ID
- 99041
- Other IDs
- P30CA033572, CHNMC-99041, CDR0000564777
- Status last verified
- May 2026
- First submitted
- 20 Sep 2007
- Last updated
- 3 Jun 2026
- Results posted
- Yes, on ClinicalTrials.gov