Chronic Myeloproliferative DisordersPhase 1/2NCT00049634
Donor Peripheral Stem Cell Transplant in Treating Patients With Myelodysplastic Syndrome, Acute Myeloid Leukemia, or Myeloproliferative Disorder
A Phase 1/2 study testing busulfan, cyclophosphamide and cyclosporine in people with Chronic Myeloproliferative Disorders and Leukemia, run by Fred Hutchinson Cancer Center at 2 sites in United States.
Trial summary
RATIONALE: Giving chemotherapy drugs before a donor peripheral blood stem cell 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 18 to 65 years
- Conditions the study is for
- Chronic Myeloproliferative Disorders, Leukemia, Myelodysplastic Syndromes
- What is being tested?
- busulfan, cyclophosphamide and cyclosporine
- Type of treatment
- Surgery or procedure
- How many people
- 30 participants
- Where
- 2 sitesUnited States
- Study dates
- Jan 2002to Feb 2007
- 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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
DISEASE CHARACTERISTICS:
- Diagnosis of 1 of the following:
- Myelodysplastic syndromes (MDS) that has advanced beyond refractory anemia (RA)
- RA with excess blasts (RAEB) (greater than 5% blasts)
- RAEB in transformation (greater than 20% but less than 30% blasts)
- Acute myeloid leukemia (greater than 30% blasts) that evolved from MDS
- Myeloproliferative disorder, including chronic myelomonocytic leukemia, agnogenic myeloid metaplasia, polycythemia vera, or essential thrombosis
- No chronic myelogenous leukemia with or without excess (greater than 5%) blasts
- Must have an HLA-identical, related donor
PATIENT CHARACTERISTICS:
Age
- 18 to 65
Performance status
- Not specified
Life expectancy
- At least 6 months
Hematopoietic
- Not specified
Hepatic
- Bilirubin less than 2 times upper limit of normal (ULN)*
- SGOT/SGPT less than 2 times ULN* NOTE: * Unless due to malignancy
Renal
- Creatinine no greater than 2.0 mg/dL OR
- Glomerular filtration rate at least 60 mL/min
Cardiovascular
- Cardiac ejection fraction at least 45%
Pulmonary
- DLCO at least 60% of predicted
Other
- HIV negative
- Human antimouse antibody negative
- Not pregnant or nursing
- Fertile patients must use effective contraception
- No other medical condition that would preclude study participation
- No hypersensitivity to cyclosporine
PRIOR CONCURRENT THERAPY:
Biologic therapy
- No prior marrow transplantation
- No concurrent growth factors for 21 days after study transplantation
Chemotherapy
- Not specified
Endocrine therapy
- Not specified
Radiotherapy
- Not specified
Surgery
- Not specified
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Fred Hutchinson Cancer Research Center
Seattle, Washington, 98104
Seattle Cancer Care Alliance
Seattle, Washington, 98109-1023
About this study
Official title: A Phase I/II Study of Immunologically Engineered rhG-CSF Mobilized Peripheral Blood Stem Cells (PBSC) for Allogeneic Transplant From HLA Identical, Related Donors for Treatment of Myeloid Malignancies
RATIONALE: Giving chemotherapy drugs before a donor peripheral blood stem cell 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. Giving colony-stimulating factors, such as G-CSF, to the donor helps the stem cells move from the bone marrow to the blood so they can be collected and stored.
PURPOSE: This phase I/II trial is studying how well donor peripheral stem cell transplant works in treating patients with myelodysplastic syndrome, acute myeloid leukemia, or myeloproliferative disorder.
Read the detailed description
OBJECTIVES:
- Determine the incidence of grades II, III, and IV graft-vs-host disease (GVHD) in patients with myelodysplastic syndromes (MDS), acute myeloid leukemia transformed from MDS, or myeloproliferative disorders treated with immunologically engineered, filgrastim (G-CSF)-mobilized, allogeneic peripheral blood stem cell transplantation.
- Determine the incidence of graft failure, relapse, and transplant-related mortality by day 100 in patients treated with this regimen.
- Determine the incidence of chronic GVHD, in terms of number and duration of immunosuppressant therapies, in patients treated with this regimen.
- Determine the feasibility of partial T-cell depletion in G-CSF-mobilized peripheral blood stem cells.
OUTLINE: Patients receive conditioning with oral busulfan every 6 hours on days -7 to -4 and cyclophosphamide IV on days -3 and -2. Immunologically engineered, filgrastim (G-CSF)-mobilized, allogeneic peripheral blood stem cells are infused on day 0.
Patients receive graft-vs-host disease prophylaxis comprising methotrexate IV on days 1, 3, 6, and 11 and cyclosporine IV over 1-4 hours (orally twice daily when tolerated) on days -1 to 80 and then gradually tapered over 5 months beginning on day 81.
Patients are followed regularly through day 100 and then at 1 year.
PROJECTED ACCRUAL: A total of 30 patients will be accrued for this study within 3 years.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
- Being tested
- busulfan, cyclophosphamide, cyclosporine, methotrexate, in vitro-treated peripheral blood stem cell transplantation
- Keywords
- chronic myelomonocytic leukemia, de novo myelodysplastic syndromes, essential thrombocythemia, polycythemia vera, previously treated myelodysplastic syndromes, refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, secondary acute myeloid leukemia, secondary myelodysplastic syndromes, chronic eosinophilic leukemia, chronic neutrophilic leukemia, atypical chronic myeloid leukemia, myelodysplastic/myeloproliferative disease, unclassifiable
- Medical subject headings
- Myeloproliferative Disorders, Leukemia, Myelodysplastic Syndromes, Myelodysplastic-Myeloproliferative Diseases, Leukemia, Myelomonocytic, Chronic, Thrombocythemia, Essential, Polycythemia Vera, 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
cyclophosphamideDrug
cyclosporineDrug
methotrexateDrug
in vitro-treated peripheral blood stem cell transplantationProcedure or surgery
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of grade II, III, and IV graft-versus-host disease
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jan 2003 (estimated)
- Study startJan 2002
- Primary completionFeb 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Myeloproliferative DisordersStudies of the same condition, open and under way.
- More trials run by Fred Hutchinson 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
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Fred Hutchinson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Investigators
- Ann E. Woolfrey, MD, Study chair, Fred Hutchinson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00049634
- Sponsor's protocol ID
- 1628.00
- Other IDs
- FHCRC-1628.00, NCI-H02-0099, CDR0000258137
- Status last verified
- Sep 2010
- First submitted
- 12 Nov 2002
- Last updated
- 16 Sep 2010
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Study of Immunologically Engineered rhG-CSF Mobilized Peripheral Blood Stem Cells (PBSC) for Allogeneic Transplant From HLA Identical, Related Donors for Treatment of Myeloid Malignancies
- ClinicalTrials.gov ID
- NCT00049634
- Acronym / sponsor ID
- 1628.00
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
- Keywords
- chronic myelomonocytic leukemia, de novo myelodysplastic syndromes, essential thrombocythemia, polycythemia vera, previously treated myelodysplastic syndromes, refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, secondary acute myeloid leukemia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Fred Hutchinson Cancer CenterUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- Jan 2002
- Primary completion
- Feb 2007 (actual)
- First posted
- 27 Jan 2003 (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 drugs before a donor peripheral blood stem cell 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. Giving colony-stimulating factors, such as G-CSF, to the donor helps the stem cells move from the bone marrow to the blood so they can be collected and stored.
PURPOSE: This phase I/II trial is studying how well donor peripheral stem cell transplant works in treating patients with myelodysplastic syndrome, acute myeloid leukemia, or myeloproliferative disorder.
Detailed description
OBJECTIVES:
- Determine the incidence of grades II, III, and IV graft-vs-host disease (GVHD) in patients with myelodysplastic syndromes (MDS), acute myeloid leukemia transformed from MDS, or myeloproliferative disorders treated with immunologically engineered, filgrastim (G-CSF)-mobilized, allogeneic peripheral blood stem cell transplantation.
- Determine the incidence of graft failure, relapse, and transplant-related mortality by day 100 in patients treated with this regimen.
- Determine the incidence of chronic GVHD, in terms of number and duration of immunosuppressant therapies, in patients treated with this regimen.
- Determine the feasibility of partial T-cell depletion in G-CSF-mobilized peripheral blood stem cells.
OUTLINE: Patients receive conditioning with oral busulfan every 6 hours on days -7 to -4 and cyclophosphamide IV on days -3 and -2. Immunologically engineered, filgrastim (G-CSF)-mobilized, allogeneic peripheral blood stem cells are infused on day 0.
Patients receive graft-vs-host disease prophylaxis comprising methotrexate IV on days 1, 3, 6, and 11 and cyclosporine IV over 1-4 hours (orally twice daily when tolerated) on days -1 to 80 and then gradually tapered over 5 months beginning on day 81.
Patients are followed regularly through day 100 and then at 1 year.
PROJECTED ACCRUAL: A total of 30 patients will be accrued for this study within 3 years.
Study design
- Primary purpose
- Treatment
- Masking
- None (open label)
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- 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 |
|---|---|---|
| busulfan | Drug | – |
| cyclophosphamide | Drug | – |
| cyclosporine | Drug | – |
| methotrexate | Drug | – |
| in vitro-treated peripheral blood stem cell transplantation | Procedure or surgery | – |
Primary outcome
Incidence of grade II, III, and IV graft-versus-host disease
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 16 Sep 2010.
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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.
Ann E. Woolfrey, MD study chair
Fred Hutchinson Cancer Center
2 sites 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 busulfan, cyclophosphamide and cyclosporine in people with Chronic Myeloproliferative Disorders and Leukemia, run by Fred Hutchinson Cancer Center at 2 sites 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 16 Sep 2010. 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 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?
Incidence of grade II, III, and IV graft-versus-host disease. 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 16 Sep 2010.