Acute Myeloid LeukemiaNCT00967525
Intraosseous Infusion of Unrelated Cord Blood Grafts
A study testing cord blood infusion in people with Acute Myeloid Leukemia and Acute Lymphoblastic Leukemia, run by Emory University at 2 sites in United States.
Trial summary
In this trial the investigators seek to determine if injecting cord blood cells directly into the bone marrow (intraosseous injection), rather than infusing them intravenously, can improve engraftment. Read more about this study
- Who is it for?
- Ages 36 months to 60 years
- Conditions the study is for
- Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Chronic Myelogenous Leukemia
- What is being tested?
- cord blood infusion
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 2 sitesUnited States
- Study dates
- Mar 2007to Jul 2011
- Phase
- Interventional
- 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 36 months to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age 36 months to 60 years old (YO)
- No prior autologous or allogeneic transplant
- Karnofsky performance score or Lansky Play-Performance of at least 80
Who cannot join
- Age < 36 months or > 60 YO
- creatinine clearance or nuclear medicine GFR of < 50 mL/min
- cardiac ejection fraction < 50%
- bilirubin > 2 × upper limit of normal or ALT > 4 × upper limit of normal or unresolved veno-occlusive disease
- Pulmonary carbon monoxide diffusing capacity (DLCO), adjusted for Hgb < 50%
- Karnofsky performance score or Lansky Play-Performance Scale <80
Show all 11Show fewer
- Uncontrolled viral, bacterial, or fungal infection at the time of study enrollment
- Seropositive for HIV
- Availability of a willing and well HLA matched related (genotypically identical or mismatched at a single allele or antigen defined by typing at HLA A, B, C and DRB1 loci) donor
- Availability of a willing and well HLA matched unrelated (allele matched or mismatched at a single allele defined by allele level typing for HLA A, B, C and DRB1 loci) adult blood or marrow donor
- Availability of an umbilical cord blood unit, which provides at least a 4/6 HLA match as defined above and ≥ 5.0 * 107 NC/Kg (cryopreserved)
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
Emory University
Atlanta, Georgia, 30322
About this study
Official title: A Pilot Study of Intraosseous Infusion of Unrelated Cord Blood Grafts
In this trial the investigators seek to determine if injecting cord blood cells directly into the bone marrow (intraosseous injection), rather than infusing them intravenously, can improve engraftment. The rational for doing this is that most hematopoietic stem cells (HSCs) infused intravenously never reach the bone marrow, getting trapped by other organs, such as the lungs, instead. The potential advantage of intraosseous infusion is suggested by studies in rodents that have demonstrated that in HSC transplants where the cell dose is limiting intraosseous injection is a more effective route of administration. The safety of intraosseous injections, in general, is underscored by the vast experience using intraosseous injections for resuscitation of critically ill children. The safety of injecting HSCs intraosseously has been demonstrated in a clinical trial of transplanting bone marrow cells.
To safeguard against problems that might result, if intraosseous infusion fails to improve engraftment in this trial, the investigators will integrate a recently introduced strategy proven to improve engraftment-the transplantation of two cord blood units. Transplanting two unrelated cord blood units by intravenous infusion has been shown to improve engraftment (although there is still room for improvement). In this trial one unit will be injected intraosseously and the other unit will be infused intravenously.
This study is being conducted as a forerunner to a larger, multi-center trial. The investigators intend to enroll five patients over 1-2 years.
Read the detailed description
Hematopoietic (blood forming) stem cells (HSCs) reside primarily in the bone marrow. Traditionally, HSCs have been obtained directly from the bone marrow. Transplants using cells obtained this way are referred to as bone marrow transplants. HSCs also circulate in the blood. Transplants using cells obtained from the blood of children and adults are referred to as peripheral blood stem cell transplants. The blood of fetuses is especially rich in HSCs and these cells can be easily collected at birth from the placenta. Transplants using these cells are called cord blood transplants. Although HSCs can be collected from various sites, all HSC transplants, regardless of the source, are given to recipients by intravenous infusion. The transplanted HSCs then migrate to the bone marrow.
Over the past ten years unrelated cord blood transplantation has become an accepted alternative to bone marrow transplantation. African-Americans and other minorities, who are underrepresented in the National Marrow Donor Program, have benefited particularly from this. In infants and young children cord blood transplantation appears to be as effective as bone marrow transplantation. In older children, adolescents and adults, however, cord blood transplantation has not been as effective, primarily because most cord blood units provide an insufficient number of cells to ensure prompt and reliable engraftment ("taking" of the transplanted cells in the recipient's bone marrow).
- Conditions
- Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Chronic Myelogenous Leukemia, Myelodysplastic Syndrome
- Being tested
- cord blood infusion
- Keywords
- AML, ALL, leukemia, CML, Myelodysplastic Syndrome, malignancies, Undifferentiated leukemia, High risk malignancies
- Medical subject headings
- Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Myelodysplastic Syndromes, Leukemia, Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
cord blood infusionProcedure or surgery
Receive two cord blood units. One administered by intraosseous infusion and the other by intravenous infusion. The second unit is being given as a safeguard, but will also allow the researchers to directly compare engraftment between intravenously and intraosseously infused cord blood units.
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Receive two cord blood units. One administered by intraosseous infusion and the other by intravenous infusion. The second unit is being given as a safeguard, but will also allow the researchers to directly compare engraftment between intravenously and intraosseously infused cord blood units. Receives: Procedure: cord blood infusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Compare the rapidity of myeloid engraftment of intraosseously and intravenously administered unrelated cord blood grafts.
Measured: 1 year after last patient enrolled
Secondary outcomes (1)
Obtain preliminary data using flow cytometric analysis to assess the importance of graft associated variables that may affect engraftment.
Measured: 1 year after last patient enrolled
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Aug 2009 (estimated)
- Study startMar 2007
- Primary completionJul 2011
- Study completionJul 2011
Why it stopped: poor enrollment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- More trials run by Emory UniversityThe 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, John Horan, Emory University
- Investigators
- John Horan, MD, Principal investigator, Emory University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00967525
- Sponsor's protocol ID
- IRB00000113
- Other IDs
- Intraosseous Infusion
- Status last verified
- Nov 2013
- First submitted
- 11 Aug 2009
- Last updated
- 27 Nov 2013
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 27 Nov 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study of Intraosseous Infusion of Unrelated Cord Blood Grafts
- ClinicalTrials.gov ID
- NCT00967525
- Acronym / sponsor ID
- IRB00000113
- Conditions
- Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Chronic Myelogenous Leukemia, Myelodysplastic Syndrome
- Keywords
- AML, ALL, leukemia, CML, Myelodysplastic Syndrome, malignancies, Undifferentiated leukemia, High risk malignancies
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: poor enrollment
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Study start
- Mar 2007
- Primary completion
- Jul 2011 (actual)
- Study completion
- Jul 2011 (actual)
- First posted
- 28 Aug 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.
In this trial the investigators seek to determine if injecting cord blood cells directly into the bone marrow (intraosseous injection), rather than infusing them intravenously, can improve engraftment. The rational for doing this is that most hematopoietic stem cells (HSCs) infused intravenously never reach the bone marrow, getting trapped by other organs, such as the lungs, instead. The potential advantage of intraosseous infusion is suggested by studies in rodents that have demonstrated that in HSC transplants where the cell dose is limiting intraosseous injection is a more effective route of administration. The safety of intraosseous injections, in general, is underscored by the vast experience using intraosseous injections for resuscitation of critically ill children. The safety of injecting HSCs intraosseously has been demonstrated in a clinical trial of transplanting bone marrow cells.
To safeguard against problems that might result, if intraosseous infusion fails to improve engraftment in this trial, the investigators will integrate a recently introduced strategy proven to improve engraftment-the transplantation of two cord blood units. Transplanting two unrelated cord blood units by intravenous infusion has been shown to improve engraftment (although there is still room for improvement). In this trial one unit will be injected intraosseously and the other unit will be infused intravenously.
This study is being conducted as a forerunner to a larger, multi-center trial. The investigators intend to enroll five patients over 1-2 years.
Detailed description
Hematopoietic (blood forming) stem cells (HSCs) reside primarily in the bone marrow. Traditionally, HSCs have been obtained directly from the bone marrow. Transplants using cells obtained this way are referred to as bone marrow transplants. HSCs also circulate in the blood. Transplants using cells obtained from the blood of children and adults are referred to as peripheral blood stem cell transplants. The blood of fetuses is especially rich in HSCs and these cells can be easily collected at birth from the placenta. Transplants using these cells are called cord blood transplants. Although HSCs can be collected from various sites, all HSC transplants, regardless of the source, are given to recipients by intravenous infusion. The transplanted HSCs then migrate to the bone marrow.
Over the past ten years unrelated cord blood transplantation has become an accepted alternative to bone marrow transplantation. African-Americans and other minorities, who are underrepresented in the National Marrow Donor Program, have benefited particularly from this. In infants and young children cord blood transplantation appears to be as effective as bone marrow transplantation. In older children, adolescents and adults, however, cord blood transplantation has not been as effective, primarily because most cord blood units provide an insufficient number of cells to ensure prompt and reliable engraftment ("taking" of the transplanted cells in the recipient's bone marrow).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Procedure: cord blood infusionReceive two cord blood units. One administered by intraosseous infusion and the other by intravenous infusion. The second unit is being given as a safeguard, but will also allow the researchers to directly compare engraftment between intravenously and intraosseously infused cord blood units. |
Who may qualify
- Ages
- 36 months to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Age 36 months to 60 years old (YO)
- No prior autologous or allogeneic transplant
- Karnofsky performance score or Lansky Play-Performance of at least 80
Key exclusion criteria (11 listed)
- Age < 36 months or > 60 YO
- creatinine clearance or nuclear medicine GFR of < 50 mL/min
- cardiac ejection fraction < 50%
- bilirubin > 2 × upper limit of normal or ALT > 4 × upper limit of normal or unresolved veno-occlusive disease
- Pulmonary carbon monoxide diffusing capacity (DLCO), adjusted for Hgb < 50%
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 |
|---|---|---|
| cord blood infusionReceive two cord blood units. One administered by intraosseous infusion and the other by intravenous infusion. The second unit is being given as a safeguard, but will also allow the researchers to directly compare engraftment between intravenously and intraosseously infused cord blood units. | Procedure or surgery | 1 |
Primary outcome
Compare the rapidity of myeloid engraftment of intraosseously and intravenously administered unrelated cord blood grafts.
Time frame: 1 year after last patient enrolled
1 secondary outcome
Obtain preliminary data using flow cytometric analysis to assess the importance of graft associated variables that may affect engraftment.
Time frame: 1 year after last patient enrolled
Results and safety
The study was stopped early (reason given: poor enrollment). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Nov 2013.
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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.
John Horan, MD principal investigator
Emory University
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing cord blood infusion in people with Acute Myeloid Leukemia and Acute Lymphoblastic Leukemia, run by Emory University 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?
The sponsor lists 3 inclusion and 11 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 27 Nov 2013. 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.
Are results available for this study?
The study was stopped early (reason given: poor enrollment). 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?
Compare the rapidity of myeloid engraftment of intraosseously and intravenously administered unrelated cord blood grafts. (time frame: 1 year after last patient enrolled). 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 27 Nov 2013.