Chronic Myeloproliferative DisordersPhase 3NCT00438958
Sibling Donor Peripheral Stem Cell Transplant or Sibling Donor Bone Marrow Transplant in Treating Patients With Hematologic Cancers or Other Diseases
A Phase 3 study testing filgrastim, allogeneic bone marrow transplantation and peripheral blood stem cell transplantation in people with Chronic Myeloproliferative Disorders and Graft Versus Host Disease, run by The Canadian Blood and Marrow Transplant Group at 16 sites in 5 countries.
Trial summary
RATIONALE: Giving chemotherapy before a donor peripheral stem cell transplant or bone marrow transplant using stem cells from a brother or sister that closely match the patient's stem cells, helps stop the growth of cancer or abnormal cells. Read more about this study
- Who is it for?
- Ages 16 to 65 years
- Conditions the study is for
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia
- What is being tested?
- filgrastim, allogeneic bone marrow transplantation and peripheral blood stem cell transplantation
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 230 participants
- Where
- 16 sitesin 5 countries
- Study dates
- Mar 2007to not given
- Phase
- Phase 3
- 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 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
DISEASE CHARACTERISTICS:
- Diagnosis of one of the following hematologic malignancies:
- Acute myeloid leukemia in first complete remission or second complete remission
- Chronic myeloid leukemia in chronic or accelerated phase
- Myelodysplasia, including any of the following:
- Refractory anemia (RA)
- RA with ringed sideroblasts
- RA with excess blasts (RAEB) I
- RAEB in transformation
- Chronic myelomonocytic leukemia
- Other hematologic malignancy for which sibling donor stem cell transplantation with a myeloablative conditioning regimen is appropriate, including any of the following:
- Indolent non-Hodgkin's lymphoma (NHL)
- Aggressive NHL
- Chronic lymphocytic leukemia
- Hodgkin's lymphoma
- Myelofibrosis
- Hematologic malignancy not otherwise specified
- HLA-matched sibling donor available meeting all of the following criteria:
- 6/6 HLA match
- HLA typing performed by serologic or DNA methodology for A and B and by DNA methodology for DRB1 (intermediate resolution)
- Not identical twin with patient
PATIENT CHARACTERISTICS:
- ECOG performance status 0-2
- Not pregnant or nursing
- Negative pregnancy test
- Fertile patients must use effective contraception
- No cognitive, linguistic, or emotional difficulty that would preclude participation in the quality-of-life component of the study
- Able to communicate in English or French
- No HIV antibody positivity
PRIOR CONCURRENT THERAPY:
- Not specified
Trial locations and contacts
16 sites in 5 countries.
16 sites in 5 countries
Canada11 sites
Cancer Care Nova Scotia
Halifax, Nova Scotia, B3H 2Y9
McMaster Children's Hospital at Hamilton Health Sciences
Hamilton, Ontario, L8N 3Z5
London Regional Cancer Program at London Health Sciences Centre
London, Ontario, N6A 465
Maisonneuve-Rosemont Hospital
Montreal, Quebec, H1T 2M4
Royal Victoria Hospital - Montreal
Montreal, Quebec, H3A 1A1
Ottawa Hospital Regional Cancer Centre - General Campus
Ottawa, Ontario, K1H 8L6
Hopital de L'Enfant Jesus
Québec, Quebec, G1J 1Z4
Centre Hospitalier Universitaire de Quebec
Québec, Quebec, G1R 2J6
Princess Margaret Hospital
Toronto, Ontario, M5G 2M9
Vancouver Hospital and Health Science Center
Vancouver, British Columbia, V5Z 4E3
CancerCare Manitoba
Winnipeg, Manitoba, R3E 0V9
Australia2 sites
Institute of Medical and Veterinary Science
Adelaide, South Australia, 5000
Royal Melbourne Hospital
Parkville, Victoria, 3050
United States1 site
Fred Hutchinson Cancer Research Center
Seattle, Washington, 98109-1024
Saudi Arabia1 site
King Faisal Specialist Hospital and Research Center
Riyadh, 11211
About this study
Official title: A Randomized Multicentre Study Comparing G-CSF Mobilized Peripheral Blood and G-CSF Stimulated Bone Marrow in Patients Undergoing Matched Sibling Transplantation for Hematologic Malignancies
RATIONALE: Giving chemotherapy before a donor peripheral stem cell transplant or bone marrow transplant using stem cells from a brother or sister that closely match the patient's stem cells, helps stop the growth of cancer or abnormal cells. It also helps stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining cancer or abnormal cells (graft-versus-tumor effect). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. 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. Giving methotrexate and cyclosporine before and after transplant may stop this from happening. It is not yet known whether a donor peripheral stem cell transplant is more effective than a donor bone marrow transplant in treating hematologic cancers or other diseases.
PURPOSE: This randomized phase III trial is studying filgrastim-mobilized sibling donor peripheral stem cell transplant to see how well it works compared with sibling donor bone marrow transplant in treating patients with hematologic cancers or other diseases.
Read the detailed description
OBJECTIVES:
Primary
- Compare the time to treatment failure in patients with hematologic malignancies or other diseases treated with filgrastim (G-CSF)-mobilized matched-sibling donor peripheral blood stem cell transplantation vs G-CSF-stimulated matched-sibling donor bone marrow transplantation.
Secondary
- Compare the hematological recovery and overall survival of patients treated with these regimens.
- Compare the quality of life, in terms of extensive graft-versus-host disease (GVHD), in patients treated with these regimens.
- Compare the economic impact associated with these treatment regimens.
Tertiary
- Compare the incidence and severity of acute GVHD in patients treated with these regimens.
- Compare organ involvement, symptomatology, and functional impact of chronic GVHD in patients treated with these regimens.
- Compare disease-free survival of patients treated with these regimens.
- Compare donor quality of life.
- Compare cost analysis, from a societal perspective, of these treatment regimens.
OUTLINE: This is a randomized, multicenter study. Patients are stratified according to treatment center, disease (chronic myelogenous leukemia vs acute myeloid leukemia vs myelodysplastic syndromes vs other hematologic malignancy), disease stage (early disease vs late disease), and conditioning regimen (busulfan and cyclophosphamide vs cyclophosphamide and total body irradiation vs other).
- Myeloablative conditioning regimen: Patients receive a myeloablative conditioning regimen that has been approved by the clinical chair.
- Stem cell transplantation (SCT): Patients are randomized to 1 of 2 SCT arms.
- Arm I: Patients undergo sibling donor filgrastim (G-CSF)-mobilized peripheral blood SCT on day 0.
- Arm II: Patients undergo sibling donor G-CSF- mobilized bone marrow transplantation on day 0.
- Graft-verus-host disease (GVHD) treatment: Patients receive methotrexate IV on days 1, 3, 6, and 11 and cyclosporine IV (or orally) every 12 hours beginning on day -2 and continuing until day 100.
Quality of life is assessed at baseline and at 1 and 3 years post-transplantation.
After completion of study therapy, patients are followed periodically for at least 4 years.
PROJECTED ACCRUAL: A total of 230 patients will be accrued for this study.
- Conditions
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma, Myelodysplastic Syndromes, Secondary Myelofibrosis
- Being tested
- filgrastim, allogeneic bone marrow transplantation, peripheral blood stem cell transplantation
- Keywords
- graft versus host disease, 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), accelerated phase chronic myelogenous leukemia, chronic phase chronic myelogenous leukemia, refractory anemia with excess blasts in transformation, refractory anemia with excess blasts, refractory anemia with ringed sideroblasts, refractory anemia, chronic myelomonocytic leukemia, de novo myelodysplastic syndromes, previously treated myelodysplastic syndromes, secondary myelodysplastic syndromes, refractory chronic lymphocytic leukemia, stage III chronic lymphocytic leukemia, stage IV chronic lymphocytic leukemia
- Medical subject headings
- Myeloproliferative Disorders, Graft vs Host Disease, Leukemia, Lymphoma, Myelodysplastic Syndromes, Congenital Abnormalities, Leukemia, Myeloid, Accelerated Phase, Leukemia, Myeloid, Chronic-Phase
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
filgrastimBiological
Given on day 0.
allogeneic bone marrow transplantationProcedure or surgery
Given on day 0
peripheral blood stem cell transplantationProcedure or surgery
Given on day 0
| Study group | Role | What they receive |
|---|---|---|
| Arm I | Active comparator | Patients undergo filgrastim (G-CSF)-mobilized sibling donor peripheral blood SCT on day 0. Receives: Biological: filgrastim; Procedure: peripheral blood stem cell transplantation |
| Arm II | Experimental | Patients undergo G-CSF-mobilized sibling donor bone marrow transplantation on day 0. Receives: Biological: filgrastim; Procedure: allogeneic bone marrow transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to treatment failure (extensive chronic graft-versus-host disease [GVHD], relapse, death)
Secondary outcomes (9)
Time to neutrophil recovery
Primary graft failure
Overall survival
Quality of life
Time to acute GVHD
Time to chronic GVHD
Chronic GVHD details
Cost
Detailed donor and patient self-reported outcomes
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Feb 2007 (estimated)
- Study startMar 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Chronic Myeloproliferative DisordersStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Enrolment
- 230 participants (estimated)
Sponsor and investigators
- Lead sponsor
- The Canadian Blood and Marrow Transplant Group
- Sponsor type
- Research network
- Collaborators
- National Cancer Institute (NCI)
- Investigators
- Stephen Couban, MD, Study chair, Cancer Care Nova ScotiaJeffrey H. Lipton, MD, PhD, Princess Margaret Hospital, Canada
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00438958
- Sponsor's protocol ID
- CDR0000528289
- Other IDs
- CBMTG-0601
- Status last verified
- Jul 2009
- First submitted
- 20 Feb 2007
- Last updated
- 5 Mar 2014
- Results posted
- No
Publications
Kariminia A, Ivison S, Ng B, Rozmus J, Sung S, Varshney A, Aljurf M, Lachance S, Walker I, Toze C, Lipton J, Lee SJ, Szer J, Doocey R, Lewis I, Smith C, Chaudhri N, Levings MK, Broady R, Devins G, Szwajcer D, Foley R, Mostafavi S, Pavletic S, Wall DA, Couban S, Panzarella T, Schultz KR. CD56bright natural killer regulatory cells in filgrastim primed donor blood or marrow products regulate chronic graft-versus-host disease: the Canadian Blood and Marrow Transplant Group randomized 0601 study results. Haematologica. 2017 Nov;102(11):1936-1946. doi: 10.3324/haematol.2017.170928. Epub 2017 Sep 21.
PubMed 28935847
Study at a glance
From the registry record, last updated by the sponsor on 5 Mar 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized Multicentre Study Comparing G-CSF Mobilized Peripheral Blood and G-CSF Stimulated Bone Marrow in Patients Undergoing Matched Sibling Transplantation for Hematologic Malignancies
- ClinicalTrials.gov ID
- NCT00438958
- Acronym / sponsor ID
- CDR0000528289
- Conditions
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma, Myelodysplastic Syndromes, Secondary Myelofibrosis
- Keywords
- graft versus host disease, 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), accelerated phase chronic myelogenous leukemia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 230 participants (estimated)
- Lead sponsor
- The Canadian Blood and Marrow Transplant GroupResearch network
- Collaborators
- National Cancer Institute (NCI)
- Study start
- Mar 2007
- First posted
- 22 Feb 2007 (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 before a donor peripheral stem cell transplant or bone marrow transplant using stem cells from a brother or sister that closely match the patient's stem cells, helps stop the growth of cancer or abnormal cells. It also helps stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining cancer or abnormal cells (graft-versus-tumor effect). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. 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. Giving methotrexate and cyclosporine before and after transplant may stop this from happening. It is not yet known whether a donor peripheral stem cell transplant is more effective than a donor bone marrow transplant in treating hematologic cancers or other diseases.
PURPOSE: This randomized phase III trial is studying filgrastim-mobilized sibling donor peripheral stem cell transplant to see how well it works compared with sibling donor bone marrow transplant in treating patients with hematologic cancers or other diseases.
Detailed description
OBJECTIVES:
Primary
- Compare the time to treatment failure in patients with hematologic malignancies or other diseases treated with filgrastim (G-CSF)-mobilized matched-sibling donor peripheral blood stem cell transplantation vs G-CSF-stimulated matched-sibling donor bone marrow transplantation.
Secondary
- Compare the hematological recovery and overall survival of patients treated with these regimens.
- Compare the quality of life, in terms of extensive graft-versus-host disease (GVHD), in patients treated with these regimens.
- Compare the economic impact associated with these treatment regimens.
Tertiary
- Compare the incidence and severity of acute GVHD in patients treated with these regimens.
- Compare organ involvement, symptomatology, and functional impact of chronic GVHD in patients treated with these regimens.
- Compare disease-free survival of patients treated with these regimens.
- Compare donor quality of life.
- Compare cost analysis, from a societal perspective, of these treatment regimens.
OUTLINE: This is a randomized, multicenter study. Patients are stratified according to treatment center, disease (chronic myelogenous leukemia vs acute myeloid leukemia vs myelodysplastic syndromes vs other hematologic malignancy), disease stage (early disease vs late disease), and conditioning regimen (busulfan and cyclophosphamide vs cyclophosphamide and total body irradiation vs other).
- Myeloablative conditioning regimen: Patients receive a myeloablative conditioning regimen that has been approved by the clinical chair.
- Stem cell transplantation (SCT): Patients are randomized to 1 of 2 SCT arms.
- Arm I: Patients undergo sibling donor filgrastim (G-CSF)-mobilized peripheral blood SCT on day 0.
- Arm II: Patients undergo sibling donor G-CSF- mobilized bone marrow transplantation on day 0.
- Graft-verus-host disease (GVHD) treatment: Patients receive methotrexate IV on days 1, 3, 6, and 11 and cyclosporine IV (or orally) every 12 hours beginning on day -2 and continuing until day 100.
Quality of life is assessed at baseline and at 1 and 3 years post-transplantation.
After completion of study therapy, patients are followed periodically for at least 4 years.
PROJECTED ACCRUAL: A total of 230 patients will be accrued for this study.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm I | Active comparator | Biological: filgrastim, Procedure: peripheral blood stem cell transplantationPatients undergo filgrastim (G-CSF)-mobilized sibling donor peripheral blood SCT on day 0. |
| Arm II | Experimental | Biological: filgrastim, Procedure: allogeneic bone marrow transplantationPatients undergo G-CSF-mobilized sibling donor bone marrow transplantation on day 0. |
Who may qualify
- Ages
- 16 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, 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 |
|---|---|---|
| filgrastimGiven on day 0. | Biological | Arm I, Arm II |
| allogeneic bone marrow transplantationGiven on day 0 | Procedure or surgery | Arm II |
| peripheral blood stem cell transplantationGiven on day 0 | Procedure or surgery | Arm I |
Primary outcome
Time to treatment failure (extensive chronic graft-versus-host disease [GVHD], relapse, death)
9 secondary outcomes
Time to neutrophil recovery
Primary graft failure
Overall survival
Quality of life
Time to acute GVHD
Time to chronic GVHD
Chronic GVHD details
Cost
Detailed donor and patient self-reported outcomes
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 5 Mar 2014.
- Derived from this study: PubMed 28935847Kariminia A, Ivison S, Ng B, Rozmus J, Sung S, Varshney A, Aljurf M, Lachance S, Walker I, Toze C, Lipton J, Lee SJ, Szer J, Doocey R, Lewis I, Smith C, Chaudhri N, Levings MK, Broady R, Devins G, Szwajcer D, Foley R, Mostafavi S, Pavletic S, Wall DA, Couban S, Panzarella T, Schultz KR. CD56bright natural killer regulatory cells in filgrastim primed donor blood or marrow products regulate chronic graft-versus-host disease: the Canadian Blood and Marrow Transplant Group randomized 0601 study results. Haematologica. 2017 Nov;102(11):1936-1946. doi: 10.3324/haematol.2017.170928. Epub 2017 Sep 21.
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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.
Stephen Couban, MD study chair
Cancer Care Nova Scotia
Jeffrey H. Lipton, MD, PhD
Princess Margaret Hospital, Canada
16 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing filgrastim, allogeneic bone marrow transplantation and peripheral blood stem cell transplantation in people with Chronic Myeloproliferative Disorders and Graft Versus Host Disease, run by The Canadian Blood and Marrow Transplant Group at 16 sites in 5 countries.
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 5 Mar 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in Canada, Australia, United States, New Zealand, Saudi Arabia. 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 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?
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?
Time to treatment failure (extensive chronic graft-versus-host disease [GVHD], relapse, death). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00438958, which also lists 1 linked publication.
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 Mar 2014.