Myeloproliferative DisordersPhase 1/2NCT00295880
Donor Umbilical Cord Blood Transplant By Injection Into the Bone Marrow in Treating Patients With Hematologic Cancer
A Phase 1/2 study testing umbilical cord blood transplantation in people with Myeloproliferative Disorders and Leukemia, run by Masonic Cancer Center, University of Minnesota.
Trial summary
Rationale: Giving chemotherapy and total-body irradiation before a donor umbilical cord blood 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 12 to 45 years
- Conditions the study is for
- Myeloproliferative Disorders, Leukemia, Lymphoma
- What is being tested?
- umbilical cord blood transplantation
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- No sites listed yet
- Study dates
- Jun 2005to Aug 2008
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 12 to 45 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Acute myeloid leukemia (AML): high risk CR1
- Acute lymphocytic leukemia (ALL): high risk CR1 [t(9;22), t (1:19), t(4;11) or other MLL rearrangements] or > 1 cycle to obtain CR; CR2+. All patients must be in CR as defined by hematological recovery, AND <5% blasts by light microscopy within the bone marrow with a cellularity of ≥15%.
- Chronic myelogenous leukemia (CML) excluding refractory blast crisis. To be eligible in first chronic phase (CP1) patient must have failed or be intolerant to imatinib mesylate.
- Myelodysplasia (MDS) IPSS Int-2 or High risk (i.e. RAEB, RAEBt) or refractory anemia with severe pancytopenia or high risk cytogenetics.
- Chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL), marginal zone B-cell lymphoma or follicular lymphoma that have progressed after at least two prior therapies. Patients with bulky disease (nodal mass greater than 5 cm).
- Lymphoplasmacytic lymphoma, mantle-cell lymphoma, prolymphocytic leukemia are eligible after initial therapy in CR1+ or PR1+.
Show all 12Show fewer
- Large cell NHL > CR2/> PR2. Patients in CR2/PR2 with initial short remission (<6 months) are eligible.
- Lymphoblastic lymphoma.
- Multiple myeloma beyond PR2.
- Karnofsky performance status (PS) 90-100% (adults)
- Lansky PS 50-100% (children)
- Acceptable organ function
Who cannot join
- Active infection at time of transplantation
- History of HIV infection
- Pregnant or breast feeding.
- Chemotherapy refractory large cell and high grade NHL (ie progressive disease after > 2 salvage regimens)
- Extensive prior therapy including > 12 months alkylator therapy or > 6 months alkylator therapy with extensive radiation.
- Diffuse myelofibrosis of BM (bone marrow) (any severity) regardless of primary diagnosis (focal fibrosis acceptable provided it involves < 20% of BM volume).
- History of pelvic irradiation.
About this study
Official title: A Phase I/II Study of Double Unit Umbilical Cord Blood (UCB) Transplantation Utilizing Graft Administration Via Intra-Bone Marrow Injection (Companion Protocol to MT2000-25)
Rationale: Giving chemotherapy and total-body irradiation before a donor umbilical cord blood 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 the donor's umbilical cord blood are injected into the patient's bone marrow they may help make stem cells, red blood cells, white blood cells, and platelets.
Purpose: This phase I/II trial is studying the side effects of donor umbilical cord blood transplant when given directly into the bone marrow and to see how well it works in treating patients with hematologic cancer.
Read the detailed description
Objectives:
Primary
- Determine the safety, in terms of infusional toxicity, of myeloablative unrelated donor double-unit umbilical cord blood (UCB) transplantation via intra-bone marrow injection (IBMI) in patients with advanced or high-risk hematologic malignancy.
- Determine whether treatment with this regimen improves the time to neutrophil engraftment (compared to historical controls) in these patients.
Secondary
- Determine the incidence of sustained donor engraftment in patients treated with this regimen.
- Determine the relative contribution of each UCB unit to initial and sustained donor engraftment in these patients.
- Determine the incidence of grade II-IV and grade III-IV acute graft-vs-host disease (GVHD) and chronic GVHD in patients treated with this regimen.
- Determine the incidence of day 100 and 180 transplant-related mortality in patients treated with this regimen.
- Determine the probability of survival at 100 days and 1 year post-transplantation in these patients.
Outline: This is a nonrandomized study.
Patients receive a myeloablative conditioning regimen. Patients also receive immunosuppression, growth factor, and supportive care as in protocol MT2005-10 (NCT00309842).
Patients receive 2 units of donor umbilical cord blood (UCB) by intra-bone marrow injection (IBMI) over 10 minutes each on day 0. If the IBMI procedure is not possible, then the UCB units are given intravenously (IV.)
After completion of study therapy, patients are followed periodically for 5 years.
Projected Accrual: A total of 36 patients will be accrued for this study.
- Being tested
- umbilical cord blood transplantation
- Keywords
- adult acute myeloid leukemia in remission, adult acute myeloid leukemia, childhood acute myeloid leukemia in remission, secondary acute myeloid leukemia, accelerated phase chronic myelogenous leukemia, childhood chronic myelogenous leukemia, chronic phase chronic myelogenous leukemia, relapsing chronic myelogenous leukemia, refractory anemia with excess blasts, de novo myelodysplastic syndromes, myelodysplastic syndromes, adult diffuse large cell lymphoma, adult diffuse mixed cell lymphoma, adult diffuse small cleaved cell lymphoma, adult lymphoblastic lymphoma, grade 3 follicular lymphoma, mantle cell lymphoma, childhood large cell lymphoma, childhood lymphoblastic lymphoma, adult acute lymphoblastic leukemia
- Medical subject headings
- Myeloproliferative Disorders, Leukemia, Lymphoma, Myelodysplastic Syndromes, Leukemia, Myeloid, Acute, Leukemia, Myeloid, Accelerated Phase, Leukemia, Myeloid, Chronic-Phase, 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.
umbilical cord blood transplantationProcedure or surgery
Also called UCBT
The graft will be given by slow injection into each posterior iliac crest.
| Study group | Role | What they receive |
|---|---|---|
| Transplant Patients | Experimental | Patients receiving umbilical cord blood transplantation. Receives: Procedure: umbilical cord blood transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Median Number of Days to Neutrophil Engraftment
Number of days to neutrophil recovery observed in recipients of two umbilical cord blood units (UCB)administered i.v. Neutrophil recovery is defined as first of 3 consecutive days with ANC (absolute neutrophil count) greater than or equal to 500/ul.
Measured: Daily through Day 60 post transplant
Secondary outcomes (7)
Number of Patients Achieving Neutrophil Recovery
Number of patients with sustained neutrophil recovery with chimerism (evidence of engraftment of both cord blood transplants) at 6 months.
Measured: 6 months
Number of Patients With Evidence of Engraftment.
Number of patients who received both cord blood units and achieved sustained donor engraftment
Measured: 1 year
Number of Patients With Acute Graft-versus-host Disease (GVHD)
Number of patients who exhibited grade II-IV acute GVHD at 100 days post umbilical cord blood transplant.
Measured: 100 days post transplant
Number of Patients With Transplant-related Mortality (TRM)
Number of patients who were deceased at days 100 and 180 from any cause other than relapse.
Measured: Day 100 and Day 180
Number of Patients Surviving at Day 100 and 1 Year.
Overall survival of patients-Number of patients who were alive at Day 100 and 1 year post transplant.
Measured: Day 100 and 1 year
Number of Patients With Grade III-IV Acute Graft-versus-host Disease (GVHD)
Number of umbilical cord blood transplant patients developing severe GVHD at 100 days post transplant.
Measured: 100 days post transplant
Number of Patients With Chronic Graft-versus-host Disease (GVHD).
Number of umbilical cord blood transplant patients with limited and extensive chronic GVHD.
Measured: 1 year post transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Feb 2006 (estimated)
- Study startJun 2005
- Primary completionAug 2008
- Study completionAug 2008
Why it stopped: Time to engraftment would not be improved compared to historical controls.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Myeloproliferative DisordersStudies of the same condition, open and under way.
- More trials run by Masonic Cancer Center, University of MinnesotaThe 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
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- John E. Wagner, MD, Study chair, Masonic Cancer Center, University of Minnesota
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00295880
- Sponsor's protocol ID
- 2004LS072
- Other IDs
- UMN-MT2004-26, UMN-0412M65789
- Status last verified
- Dec 2017
- First submitted
- 23 Feb 2006
- Last updated
- 28 Dec 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Brunstein CG, Barker JN, Weisdorf DJ, Defor TE, McKenna D, Chong SY, Miller JS, McGlave PB, Wagner JE. Intra-BM injection to enhance engraftment after myeloablative umbilical cord blood transplantation with two partially HLA-matched units. Bone Marrow Transplant. 2009 Jun;43(12):935-40. doi: 10.1038/bmt.2008.417. Epub 2009 Jan 12.
PubMed 19139736
Study at a glance
From the registry record, last updated by the sponsor on 28 Dec 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Study of Double Unit Umbilical Cord Blood (UCB) Transplantation Utilizing Graft Administration Via Intra-Bone Marrow Injection (Companion Protocol to MT2000-25)
- ClinicalTrials.gov ID
- NCT00295880
- Acronym / sponsor ID
- 2004LS072
- Keywords
- adult acute myeloid leukemia in remission, adult acute myeloid leukemia, childhood acute myeloid leukemia in remission, secondary acute myeloid leukemia, accelerated phase chronic myelogenous leukemia, childhood chronic myelogenous leukemia, chronic phase chronic myelogenous leukemia, relapsing chronic myelogenous leukemia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Time to engraftment would not be improved compared to historical controls.
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Masonic Cancer Center, University of MinnesotaUniversity, hospital or other
- Study start
- Jun 2005
- Primary completion
- Aug 2008 (actual)
- Study completion
- Aug 2008 (actual)
- First posted
- 24 Feb 2006 (estimated)
- Results
- Posted 3 Sep 2009
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 and total-body irradiation before a donor umbilical cord blood 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 the donor's umbilical cord blood are injected into the patient's bone marrow they may help make stem cells, red blood cells, white blood cells, and platelets.
Purpose: This phase I/II trial is studying the side effects of donor umbilical cord blood transplant when given directly into the bone marrow and to see how well it works in treating patients with hematologic cancer.
Detailed description
Objectives:
Primary
- Determine the safety, in terms of infusional toxicity, of myeloablative unrelated donor double-unit umbilical cord blood (UCB) transplantation via intra-bone marrow injection (IBMI) in patients with advanced or high-risk hematologic malignancy.
- Determine whether treatment with this regimen improves the time to neutrophil engraftment (compared to historical controls) in these patients.
Secondary
- Determine the incidence of sustained donor engraftment in patients treated with this regimen.
- Determine the relative contribution of each UCB unit to initial and sustained donor engraftment in these patients.
- Determine the incidence of grade II-IV and grade III-IV acute graft-vs-host disease (GVHD) and chronic GVHD in patients treated with this regimen.
- Determine the incidence of day 100 and 180 transplant-related mortality in patients treated with this regimen.
- Determine the probability of survival at 100 days and 1 year post-transplantation in these patients.
Outline: This is a nonrandomized study.
Patients receive a myeloablative conditioning regimen. Patients also receive immunosuppression, growth factor, and supportive care as in protocol MT2005-10 (NCT00309842).
Patients receive 2 units of donor umbilical cord blood (UCB) by intra-bone marrow injection (IBMI) over 10 minutes each on day 0. If the IBMI procedure is not possible, then the UCB units are given intravenously (IV.)
After completion of study therapy, patients are followed periodically for 5 years.
Projected Accrual: A total of 36 patients will be accrued for this study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Transplant Patients | Experimental | Procedure: umbilical cord blood transplantationPatients receiving umbilical cord blood transplantation. |
Who may qualify
- Ages
- 12 to 45 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (12 listed)
- Acute myeloid leukemia (AML): high risk CR1
- Acute lymphocytic leukemia (ALL): high risk CR1 [t(9;22), t (1:19), t(4;11) or other MLL rearrangements] or > 1 cycle to obtain CR; CR2+. All patients must be in CR as defined by hematological recovery, AND <5% blasts by light microscopy within the bone marrow with a cellularity of ≥15%.
- Chronic myelogenous leukemia (CML) excluding refractory blast crisis. To be eligible in first chronic phase (CP1) patient must have failed or be intolerant to imatinib mesylate.
- Myelodysplasia (MDS) IPSS Int-2 or High risk (i.e. RAEB, RAEBt) or refractory anemia with severe pancytopenia or high risk cytogenetics.
- Chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL), marginal zone B-cell lymphoma or follicular lymphoma that have progressed after at least two prior therapies. Patients with bulky disease (nodal mass greater than 5 cm).
Key exclusion criteria (7 listed)
- Active infection at time of transplantation
- History of HIV infection
- Pregnant or breast feeding.
- Chemotherapy refractory large cell and high grade NHL (ie progressive disease after > 2 salvage regimens)
- Extensive prior therapy including > 12 months alkylator therapy or > 6 months alkylator therapy with extensive radiation.
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 |
|---|---|---|
| umbilical cord blood transplantationAlso: UCBTThe graft will be given by slow injection into each posterior iliac crest. | Procedure or surgery | Transplant Patients |
Primary outcome
Median Number of Days to Neutrophil Engraftment
Time frame: Daily through Day 60 post transplant
Number of days to neutrophil recovery observed in recipients of two umbilical cord blood units (UCB)administered i.v. Neutrophil recovery is defined as first of 3 consecutive days with ANC (absolute neutrophil count) greater than or equal to 500/ul.
7 secondary outcomes
Number of Patients Achieving Neutrophil Recovery
Time frame: 6 months
Number of Patients With Evidence of Engraftment.
Time frame: 1 year
Number of Patients With Acute Graft-versus-host Disease (GVHD)
Time frame: 100 days post transplant
Number of Patients With Transplant-related Mortality (TRM)
Time frame: Day 100 and Day 180
Number of Patients Surviving at Day 100 and 1 Year.
Time frame: Day 100 and 1 year
Number of Patients With Grade III-IV Acute Graft-versus-host Disease (GVHD)
Time frame: 100 days post transplant
Number of Patients With Chronic Graft-versus-host Disease (GVHD).
Time frame: 1 year post transplant
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Dec 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 19139736Brunstein CG, Barker JN, Weisdorf DJ, Defor TE, McKenna D, Chong SY, Miller JS, McGlave PB, Wagner JE. Intra-BM injection to enhance engraftment after myeloablative umbilical cord blood transplantation with two partially HLA-matched units. Bone Marrow Transplant. 2009 Jun;43(12):935-40. doi: 10.1038/bmt.2008.417. Epub 2009 Jan 12.
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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 E. Wagner, MD study chair
Masonic Cancer Center, University of Minnesota
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing umbilical cord blood transplantation in people with Myeloproliferative Disorders and Leukemia, run by Masonic Cancer Center, University of Minnesota.
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 12 inclusion and 7 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 28 Dec 2017. Status can change between updates, so confirm with the study contact before making plans.
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?
Yes. Results are posted in the registry (first posted 3 Sep 2009). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Median Number of Days to Neutrophil Engraftment (time frame: Daily through Day 60 post transplant). 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 NCT00295880, 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 28 Dec 2017.