NeutropeniaPhase 2NCT00937053
Maintaining a Higher Level of Haemoglobin: Effect on the White Cells After Bone Marrow Transplantation in Children.
A Phase 2 study testing Transfusion level 120 g/dL, Transfusion level 70 g/dL and Platelet transfusion in people with Neutropenia, run by St. Justine's Hospital at 5 sites in Canada.
Trial summary
The purpose of this study is to determine if maintaining a high hemoglobin level in children that underwent bone marrow transplant will accelerate the neutrophil recovery. Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Neutropenia
- What is being tested?
- Transfusion level 120 g/dL, Transfusion level 70 g/dL and Platelet transfusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 5 sitesCanada
- Study dates
- Jun 2009to Jun 2015
- 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 1 year to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients between 1 year of age and 18 years of age at the time of transplantation (18 years is the upper limit of childhood in CIBMTR definitions)
- Patients planned to undergo a related or unrelated allogeneic bone marrow transplant for a malignant or benign disease (except for sickle cell disease)
- Conditioning regimen must be myeloablative, i.e. include busulfan greater or equal to 12 mg/kg or Total Body Irradiation greater or equal to 10 Gy, except for patients with acquired severe aplastic anemia
- Absence of any psychological, familial, sociological or geographical condition potentially hampering compliance with the study protocol and follow-up schedule; those conditions should be discussed with the patient before enrolment in the trial
- Patient must agree to receive blood transfusion
- Patient (or their legal guardians) must sign an Informed consent Form
Who cannot join
- Patients receiving autologous bone marrow transplant, cord blood transplant or peripheral stem cell transplant
- Sickle cell disease (since higher hemoglobin level increases blood viscosity and puts these patients at risk for stroke)
- Hematopoietic growth factor (G-CSF, GM-CSF, stem cell factor, erythropoietin) planned before transplantation (post-transplant decision of hematopoietic growth factors administration as required by the patient's condition will be accepted)
- Presence of an allo-antibody directed against red blood cells
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
Canada5 sites
Souther Alberta Children's Cancer Care Program, Calgary
Calgary, Alberta
Hematopoietic Stem Cell Transplantation Program, Sainte-Justine Hospital
Montreal, Quebec, H3T 1C5
Department of Hematology, The Montreal Children's Hospital
Montreal, Quebec
Section of Blood and Marrow Transplant, The Hospital for Sick Children
Toronto, Ontario
Pediatric Bone Marrow Transplant Unit, British Columbia Children's Hospital
Vancouver, British Columbia
About this study
Official title: The Effect of Maintaining a Higher Haemoglobin Level on Neutropenia Duration After Bone Marrow Transplantation in Children.
The purpose of this study is to determine if maintaining a high hemoglobin level in children that underwent bone marrow transplant will accelerate the neutrophil recovery.
Read the detailed description
The investigators know that children requiring bone marrow transplant need to first go through a myeloablative regimen, which induces a neutropenia. The length of the neutropenia has an incidence on the risk of contracting bacterial and fungal infections that could be lethal. It is then important to find ways to accelerate the neutrophil recovery, so patient survival can be improved.
Studies conducted in the '70s and '80s suggested that if the hemoglobin level could be kept at a higher level, then the neutrophil recovery would be accelerated. Other studies also support the hypothesis that if the stem cells do not need to produce red cells because these are being supplied through transfusions, then the stem cells would differentiate into non-erythroid cell lines.
As of now, for patients undergoing a bone marrow transplant, it is standard practice to transfuse with red cells based on the condition of the patient or if the hemoglobin level falls below 70 g/L. Hematopoietic growth factors have been used to increase the speed of the neutrophil recovery, but studies conducted so far do not demonstrate that mortality and length of hospitalization have been reduced by the specific use of G-CSF. In more recent studies, these agents have been shown to also have negative effects, such as delayed platelet recovery and impaired immune recovery. In addition, the prophylactic use of G-CSF was also associated with graft-versus-host disease, treatment-related mortality and death.
In conclusion, this study will determine if maintaining a higher hemoglobin level has an effect on the neutrophil recovery after allogenic bone marrow transplantation in children.
- Conditions
- Neutropenia
- Being tested
- Transfusion level 120 g/dL, Transfusion level 70 g/dL, Platelet transfusion
- Keywords
- Neutropenia, Transfusions, Hemoglobin, Bone marrow transplant, Hematopoietic stem cell transplant
- Medical subject headings
- Neutropenia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Transfusion level 120 g/dLOther
Patients whose hemoglobin falls below 120 g/dL will be transfused with red cells within 24 hours.
Transfusion level 70 g/dLOther
Patients whose hemoglobin falls below 70 g/dL will be transfused with red cells within 24 hours
Platelet transfusionOther
Patients whose platelets fall below 10 x 10*9 will be transfused with platelets
| Study group | Role | What they receive |
|---|---|---|
| Hemoglobin below 120 g/dL | Experimental | Not described Receives: Other: Transfusion level 120 g/dL; Other: Platelet transfusion |
| Hemoglobin below 70 g/dL | Active comparator | Not described Receives: Other: Transfusion level 70 g/dL; Other: Platelet transfusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to neutrophil engraftment (defined as the time from transplantation to the first of three consecutive days with a neutrophil count > 0,5 x 109/L, as used in the International Bone Marrow Transplant Registry (IBMTR) criteria).
Measured: First 100 days post HSCT
Secondary outcomes (9)
Time to platelet engraftment (defined as the time from transplantation to the first of three consecutive days with a platelet count > 20 x 109/L, without platelet transfusion 7 days prior (IBMTR criteria)).
Measured: First 100 days post HSCT
Transfusions given (red cells and platelets)
Measured: First 100 days post HSCT
Hospitalization length
Measured: 2 years
Immune reconstitution (lymphoid subsets)
Measured: First 100 days post HSCT
Overall survival
Measured: 5 years
Graft vs host disease (GVHD)
Measured: 2 years
Treatment-related mortality (death without relapse)
Measured: 2 years
Relapse
Measured: 2 years
Chimerism
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jul 2009 (estimated)
- Study startJun 2009
- Primary completionJun 2010 (estimated)
- Study completionJun 2015 (estimated)
Why it stopped: 3 unexpected Serious Adverse Events (veno-occlusive disease (VOD))
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NeutropeniaStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- St. Justine's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Nancy Robitaille, MD
- Investigators
- Michel Duval, MD, Principal investigator, St. Justine's HospitalNancy Robitaille, MD, Principal investigator, St. Justine's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00937053
- Sponsor's protocol ID
- 9967
- Status last verified
- May 2009
- First submitted
- 9 Jul 2009
- Last updated
- 4 Aug 2010
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 4 Aug 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Effect of Maintaining a Higher Haemoglobin Level on Neutropenia Duration After Bone Marrow Transplantation in Children.
- ClinicalTrials.gov ID
- NCT00937053
- Acronym / sponsor ID
- 9967
- Conditions
- Neutropenia
- Keywords
- Neutropenia, Transfusions, Hemoglobin, Bone marrow transplant, Hematopoietic stem cell transplant
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: 3 unexpected Serious Adverse Events (veno-occlusive disease (VOD))
- Enrolment
- 6 participants (actual)
- Lead sponsor
- St. Justine's HospitalUniversity, hospital or other
- Collaborators
- Nancy Robitaille, MD
- Study start
- Jun 2009
- Primary completion
- Jun 2010 (estimated)
- Study completion
- Jun 2015 (estimated)
- First posted
- 10 Jul 2009 (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.
The purpose of this study is to determine if maintaining a high hemoglobin level in children that underwent bone marrow transplant will accelerate the neutrophil recovery.
Detailed description
The investigators know that children requiring bone marrow transplant need to first go through a myeloablative regimen, which induces a neutropenia. The length of the neutropenia has an incidence on the risk of contracting bacterial and fungal infections that could be lethal. It is then important to find ways to accelerate the neutrophil recovery, so patient survival can be improved.
Studies conducted in the '70s and '80s suggested that if the hemoglobin level could be kept at a higher level, then the neutrophil recovery would be accelerated. Other studies also support the hypothesis that if the stem cells do not need to produce red cells because these are being supplied through transfusions, then the stem cells would differentiate into non-erythroid cell lines.
As of now, for patients undergoing a bone marrow transplant, it is standard practice to transfuse with red cells based on the condition of the patient or if the hemoglobin level falls below 70 g/L. Hematopoietic growth factors have been used to increase the speed of the neutrophil recovery, but studies conducted so far do not demonstrate that mortality and length of hospitalization have been reduced by the specific use of G-CSF. In more recent studies, these agents have been shown to also have negative effects, such as delayed platelet recovery and impaired immune recovery. In addition, the prophylactic use of G-CSF was also associated with graft-versus-host disease, treatment-related mortality and death.
In conclusion, this study will determine if maintaining a higher hemoglobin level has an effect on the neutrophil recovery after allogenic bone marrow transplantation in children.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Hemoglobin below 120 g/dL | Experimental | Other: Transfusion level 120 g/dL, Other: Platelet transfusion |
| Hemoglobin below 70 g/dL | Active comparator | Other: Transfusion level 70 g/dL, Other: Platelet transfusion |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- Patients between 1 year of age and 18 years of age at the time of transplantation (18 years is the upper limit of childhood in CIBMTR definitions)
- Patients planned to undergo a related or unrelated allogeneic bone marrow transplant for a malignant or benign disease (except for sickle cell disease)
- Conditioning regimen must be myeloablative, i.e. include busulfan greater or equal to 12 mg/kg or Total Body Irradiation greater or equal to 10 Gy, except for patients with acquired severe aplastic anemia
- Absence of any psychological, familial, sociological or geographical condition potentially hampering compliance with the study protocol and follow-up schedule; those conditions should be discussed with the patient before enrolment in the trial
- Patient must agree to receive blood transfusion
Key exclusion criteria (4 listed)
- Patients receiving autologous bone marrow transplant, cord blood transplant or peripheral stem cell transplant
- Sickle cell disease (since higher hemoglobin level increases blood viscosity and puts these patients at risk for stroke)
- Hematopoietic growth factor (G-CSF, GM-CSF, stem cell factor, erythropoietin) planned before transplantation (post-transplant decision of hematopoietic growth factors administration as required by the patient's condition will be accepted)
- Presence of an allo-antibody directed against red blood cells
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 |
|---|---|---|
| Transfusion level 120 g/dLPatients whose hemoglobin falls below 120 g/dL will be transfused with red cells within 24 hours. | Other | Hemoglobin below 120 g/dL |
| Transfusion level 70 g/dLPatients whose hemoglobin falls below 70 g/dL will be transfused with red cells within 24 hours | Other | Hemoglobin below 70 g/dL |
| Platelet transfusionPatients whose platelets fall below 10 x 10*9 will be transfused with platelets | Other | Hemoglobin below 120 g/dL, Hemoglobin below 70 g/dL |
Primary outcome
Time to neutrophil engraftment (defined as the time from transplantation to the first of three consecutive days with a neutrophil count > 0,5 x 109/L, as used in the International Bone Marrow Transplant Registry (IBMTR) criteria).
Time frame: First 100 days post HSCT
9 secondary outcomes
Time to platelet engraftment (defined as the time from transplantation to the first of three consecutive days with a platelet count > 20 x 109/L, without platelet transfusion 7 days prior (IBMTR criteria)).
Time frame: First 100 days post HSCT
Transfusions given (red cells and platelets)
Time frame: First 100 days post HSCT
Hospitalization length
Time frame: 2 years
Immune reconstitution (lymphoid subsets)
Time frame: First 100 days post HSCT
Overall survival
Time frame: 5 years
Graft vs host disease (GVHD)
Time frame: 2 years
Treatment-related mortality (death without relapse)
Time frame: 2 years
Relapse
Time frame: 2 years
Chimerism
Time frame: 2 years
Results and safety
The study was stopped early (reason given: 3 unexpected Serious Adverse Events (veno-occlusive disease (VOD))). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Aug 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.
Michel Duval, MD principal investigator
St. Justine's Hospital
Nancy Robitaille, MD principal investigator
St. Justine's Hospital
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Transfusion level 120 g/dL, Transfusion level 70 g/dL and Platelet transfusion in people with Neutropenia, run by St. Justine's Hospital at 5 sites in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 4 exclusion criteria. 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: stopped early, as of the sponsor's last update on 4 Aug 2010. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in Canada. 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 stopped early (reason given: 3 unexpected Serious Adverse Events (veno-occlusive disease (VOD))). Results are not available in the source record.
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 neutrophil engraftment (defined as the time from transplantation to the first of three consecutive days with a neutrophil count > 0,5 x 109/L, as used in the International Bone Marrow Transplant Registry (IBMTR) criteria). (time frame: First 100 days post HSCT). 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 4 Aug 2010.