AnemiaPhase 2NCT00301912
Busulfan and Fludarabine Before Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer
A Phase 2 study testing filgrastim, busulfan and fludarabine phosphate in people with Anemia and Chronic Myeloproliferative Disorders, run by University of California, San Francisco at 1 site in United States.
Trial summary
RATIONALE: Drugs used in chemotherapy, such as busulfan and fludarabine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Read more about this study
- Who is it for?
- Ages 16 to 60 years
- Conditions the study is for
- Anemia, Chronic Myeloproliferative Disorders, Leukemia
- What is being tested?
- filgrastim, busulfan and fludarabine phosphate
- Type of treatment
- Surgery or procedure
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2002to Nov 2007
- 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 16 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
DISEASE CHARACTERISTICS:
- Diagnosis of 1 of the following hematopoietic disorders:
- Chronic myelogenous leukemia (CML), meeting 1 of the following criteria:
- Chronic phase disease failing imatinib mesylate therapy
- Progressive disease OR failed to achieve a major cytogenetic response at 1 year after initiation of therapy
- Accelerated phase disease, meeting 1 of the following criteria:
- Failed to achieve complete cytogenetic remission at 1 year after initiation of therapy
- Failed to achieve any cytogenetic response at 3 or 6 months during therapy
- Progressive disease, demonstrated by worsening cytogenetic response in 2 consecutive analyses separated by 4 weeks
- Blast crisis with < 10% blasts in bone marrow within 6 weeks of transplantation
- Acute myeloid leukemia (AML), meeting 1 of the following criteria:
- In second or greater remission
- In first remission with poor prognosis cytogenetics [-5, -5q, -7, -7q and ≥ 2 cytogenetic abnormalities, t(6,9), t(9,11), or Philadelphia chromosome]
- In hematologic remission but with persistent cytogenetic abnormalities
- Primary refractory AML with < 10% blasts in bone marrow within 6 weeks of transplantation
- Myelodysplasia with < 20% blasts in bone marrow within 6 weeks of transplantation and meeting 1 of the following criteria:
- Advanced disease (International Prognostic Scoring System [IPSS] score intermediate-1, intermediate-2, or high risk)
- Myelodysplastic syndromes (MDS) with progression to AML
- Treatment-related AML
- Acute lymphocytic leukemia (ALL), meeting 1 of the following criteria:
- In second or greater remission
- In first remission with high-risk cytogenetics [Philadelphia chromosome; t(4,11); and -7]
- Primary refractory ALL with < 10% blasts in the bone marrow
- Severe aplastic anemia that has failed immunosuppressive therapy
- Non-Hodgkin's lymphoma, meeting 1 of the following criteria:
- In second or greater remission
- Relapsed disease in a patient not eligible for autologous stem cell transplantation
- Lymphoproliferative disease (e.g., chronic lymphocytic leukemia or Waldenstrom's macroglobulinemia), meeting 1 of the following criteria:
- In second or greater remission
- Relapsed disease in a patient not eligible for autologous stem cell transplantation
- Multiple myeloma, meeting 1 of the following criteria:
- Stage II or III disease in first or greater relapse
- Refractory disease
- Newly diagnosed disease with chromosome 13 abnormalities
- Advanced myeloproliferative disease, meeting 1 of the following criteria:
- Myelofibrosis requiring > 2 units of packed red blood cells each month
- Essential thrombocythemia or polycythemia rubra vera that has progressed to AML
- Failed prior AML therapy
- No active, uncontrolled CNS leukemia
- Not eligible for autologous or mini-allogeneic transplantation
- No fully matched or single-antigen mismatched sibling donor available
- HLA-matched unrelated donor available
- HLA typed at HLA-A, -B, -C, -DRB1 and/or -DQB1 by high-resolution techniques
- For patients without HLA identical donors, mismatches at DQ (i.e., 8/8 match) and 1 additional mismatch at the allele level at HLA-A, -B, -C, or -DRB1 (i.e., 7/8 molecular match) allowed
PATIENT CHARACTERISTICS:
- ECOG performance status 0-2
- Creatinine < 2.0 mg/dL
- Pulmonary diffusing capacity > 40% of predicted
- Cardiac ejection fraction > 40% by MUGA or echocardiography
- No active liver disease
- Bilirubin ≤ 2.0 mg/dL
- Alkaline phosphatase < 3 times upper limit of normal (ULN)
- AST < 3 times ULN
- Hepatitis C or active hepatitis B (HBV) allowed provided a liver biopsy is performed and ≤ grade 2 inflammation is present
- Patients with active HBV viral replication must receive antiviral therapy
- HIV negative
- No ongoing active infection
- Not pregnant or nursing
- Negative pregnancy test
PRIOR CONCURRENT THERAPY:
- See Disease Characteristics
- More than 3 weeks since prior chemotherapy except for hydroxyurea or imatinib mesylate
- More than 3 months since prior interferon
Trial locations and contacts
1 site in 1 country.
United States1 site
UCSF Helen Diller Family Comprehensive Cancer Center
San Francisco, California, 94115
About this study
Official title: Phase II Study Evaluating Busulfan and Fludarabine as Preparative Therapy in Adults With Hematopoietic Disorders Undergoing Matched Unrelated Donor Stem Cell Transplantation
RATIONALE: Drugs used in chemotherapy, such as busulfan and fludarabine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Giving chemotherapy with a peripheral stem cell or bone marrow transplant may allow more chemotherapy to be given so that more cancer cells are killed. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Tacrolimus and methotrexate may stop this from happening.
PURPOSE: This phase II trial is studying how well giving busulfan together with fludarabine before donor stem cell transplant works in treating patients with hematologic cancer.
Read the detailed description
OBJECTIVES:
Primary
- Determine the safety, in terms of treatment-related mortality at 100 days post-transplantation, of a myeloablative preparative regimen comprising busulfan and fludarabine and graft-vs-host disease (GVHD) prophylaxis comprising tacrolimus and methotrexate in patients with hematopoietic disorders undergoing matched unrelated donor stem cell transplantation.
- Determine the efficacy, in terms of overall survival at 1-year post-transplantation, in patients treated with this regimen.
Secondary
- Determine organ toxicity in patients treated with this regimen.
- Determine neutrophil and platelet recovery in patients treated with this regimen.
- Determine the incidence and severity of acute and chronic GVHD in patients treated with this regimen.
OUTLINE:
- Myeloablative preparative regimen: Patients receive busulfan IV over 2 hours every 6 hours on days -7 to -4 and fludarabine IV over 30 minutes on days -7 to -3.
- Allogeneic stem cell transplantation: Patients undergo allogeneic peripheral blood stem cell or bone marrow transplantation on day 0. Patients also receive filgrastim (G-CSF) subcutaneously daily beginning on day 7 and continuing until blood counts recover.
- Graft-vs-host disease prophylaxis: Patients receive tacrolimus IV continuously beginning on day -2 and continuing until discharged from the hospital (may convert to oral dosing administered twice daily when tolerated) and methotrexate IV over 15-30 minutes on days 1, 3, 6, and 11.
After completion of study therapy, patients are followed periodically.
PROJECTED ACCRUAL: A total of 36 patients will be accrued for this study.
- Conditions
- Anemia, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes
- Being tested
- filgrastim, busulfan, fludarabine phosphate, methotrexate, tacrolimus
- Keywords
- chronic phase chronic myelogenous leukemia, accelerated phase chronic myelogenous leukemia, blastic phase chronic myelogenous leukemia, adult acute myeloid leukemia in remission, 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, secondary acute myeloid leukemia, de novo myelodysplastic syndromes, previously treated myelodysplastic syndromes, secondary myelodysplastic syndromes, atypical chronic myeloid leukemia, myelodysplastic/myeloproliferative disease, unclassifiable, adult acute lymphoblastic leukemia in remission, recurrent adult lymphoblastic lymphoma, anemia, refractory chronic lymphocytic leukemia
- Medical subject headings
- Anemia, Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma, Neoplasms, Plasma Cell, Myelodysplastic Syndromes, Myelodysplastic-Myeloproliferative Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
filgrastimBiological
busulfanDrug
methotrexateDrug
tacrolimusDrug
allogeneic bone marrow transplantationProcedure or surgery
allogeneic hematopoietic stem cell transplantationProcedure or surgery
peripheral blood stem cell transplantationProcedure or surgery
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment-related mortality in the first 100 days post-transplant
Overall survival at 1 year post-transplant
Secondary outcomes (4)
Incidence and severity of organ-specific toxicity
Engraftment including neutrophil and platelet recovery and donor chimerism at 3 and 12 months post-transplant
Rate of acute graft-vs-host disease (GVHD)
Rate of chronic GVHD
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Mar 2006 (estimated)
- Study startJan 2002
- Primary completionNov 2007
Why it stopped: Withdrawn because study never opened to accrual
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AnemiaStudies of the same condition, open and under way.
- More trials run by University of California, San FranciscoThe 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
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of California, San Francisco
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Investigators
- Thomas G. Martin, MD, Study chair, University of California, San Francisco
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00301912
- Sponsor's protocol ID
- CDR0000463168
- Other IDs
- UCSF-02257, UCSF-2214, UCSF-H24045-22163-04
- Status last verified
- Oct 2012
- First submitted
- 9 Mar 2006
- Last updated
- 2 Oct 2012
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Oct 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Study Evaluating Busulfan and Fludarabine as Preparative Therapy in Adults With Hematopoietic Disorders Undergoing Matched Unrelated Donor Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT00301912
- Acronym / sponsor ID
- CDR0000463168
- Conditions
- Anemia, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes
- Keywords
- chronic phase chronic myelogenous leukemia, accelerated phase chronic myelogenous leukemia, blastic phase chronic myelogenous leukemia, adult acute myeloid leukemia in remission, 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)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Withdrawn because study never opened to accrual
- Enrolment
- 0 participants (actual)
- Lead sponsor
- University of California, San FranciscoUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- Jan 2002
- Primary completion
- Nov 2007 (actual)
- First posted
- 13 Mar 2006 (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, such as busulfan and fludarabine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Giving chemotherapy with a peripheral stem cell or bone marrow transplant may allow more chemotherapy to be given so that more cancer cells are killed. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Tacrolimus and methotrexate may stop this from happening.
PURPOSE: This phase II trial is studying how well giving busulfan together with fludarabine before donor stem cell transplant works in treating patients with hematologic cancer.
Detailed description
OBJECTIVES:
Primary
- Determine the safety, in terms of treatment-related mortality at 100 days post-transplantation, of a myeloablative preparative regimen comprising busulfan and fludarabine and graft-vs-host disease (GVHD) prophylaxis comprising tacrolimus and methotrexate in patients with hematopoietic disorders undergoing matched unrelated donor stem cell transplantation.
- Determine the efficacy, in terms of overall survival at 1-year post-transplantation, in patients treated with this regimen.
Secondary
- Determine organ toxicity in patients treated with this regimen.
- Determine neutrophil and platelet recovery in patients treated with this regimen.
- Determine the incidence and severity of acute and chronic GVHD in patients treated with this regimen.
OUTLINE:
- Myeloablative preparative regimen: Patients receive busulfan IV over 2 hours every 6 hours on days -7 to -4 and fludarabine IV over 30 minutes on days -7 to -3.
- Allogeneic stem cell transplantation: Patients undergo allogeneic peripheral blood stem cell or bone marrow transplantation on day 0. Patients also receive filgrastim (G-CSF) subcutaneously daily beginning on day 7 and continuing until blood counts recover.
- Graft-vs-host disease prophylaxis: Patients receive tacrolimus IV continuously beginning on day -2 and continuing until discharged from the hospital (may convert to oral dosing administered twice daily when tolerated) and methotrexate IV over 15-30 minutes on days 1, 3, 6, and 11.
After completion of study therapy, patients are followed periodically.
PROJECTED ACCRUAL: A total of 36 patients will be accrued for this study.
Study design
- Primary purpose
- Treatment
- Masking
- None (open label)
Who may qualify
- Ages
- 16 to 60 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 |
|---|---|---|
| filgrastim | Biological | – |
| busulfan | Drug | – |
| fludarabine phosphate | Drug | – |
| methotrexate | Drug | – |
| tacrolimus | Drug | – |
| allogeneic bone marrow transplantation | Procedure or surgery | – |
| allogeneic hematopoietic stem cell transplantation | Procedure or surgery | – |
| peripheral blood stem cell transplantation | Procedure or surgery | – |
Primary outcome
Treatment-related mortality in the first 100 days post-transplant
Overall survival at 1 year post-transplant
4 secondary outcomes
Incidence and severity of organ-specific toxicity
Engraftment including neutrophil and platelet recovery and donor chimerism at 3 and 12 months post-transplant
Rate of acute graft-vs-host disease (GVHD)
Rate of chronic GVHD
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Oct 2012.
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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.
Thomas G. Martin, MD study chair
University of California, San Francisco
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 filgrastim, busulfan and fludarabine phosphate in people with Anemia and Chronic Myeloproliferative Disorders, run by University of California, San Francisco 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: withdrawn, as of the sponsor's last update on 2 Oct 2012. 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?
The study was withdrawn before enrolling participants, so there are no results.
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?
Treatment-related mortality in the first 100 days post-transplant. The study lists 2 primary outcomes in all. 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 2 Oct 2012.