LeukemiaPhase 1/2NCT00963872
Donor Umbilical Cord Blood Transplant After Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation in Treating Patients With High-Risk Hematologic Cancer
A Phase 1/2 study testing cyclophosphamide, fludarabine phosphate and Total body irradiation in people with Leukemia and Lymphoma, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
Trial summary
RATIONALE: Giving low doses of chemotherapy and total-body irradiation before a donor umbilical cord blood transplant helps stop the growth of cancer cells. It may also 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 75 years
- Conditions the study is for
- Leukemia, Lymphoma, Multiple Myeloma
- What is being tested?
- cyclophosphamide, fludarabine phosphate and Total body irradiation
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 31 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 2010to Aug 2013
- 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 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Disease Criteria
- Acute Leukemias: Must be in remission by morphology (<5% blasts). Note cytogenetic relapse or persistent disease without morphologic relapse is acceptable. Also a small percentage of blasts that is equivocal between marrow regeneration vs. early relapse are acceptable provided there are no associated cytogenetic markers consistent with relapse. (See exclusion criteria for more detailed definition)
- Acute myeloid leukemia: high risk CR1 (as evidenced by preceding myelodysplastic syndrome (MDS), high risk cytogenetics such as those associated with MDS or complex karyotype, > 2 cycles to obtain complete remission (CR) or erythroblastic and megakaryocytic); second or greater CR.
- Acute lymphoblastic leukemia/lymphoma: high risk CR1 as evidenced by high risk cytogenetics (e.g. t(9;22, t(1;19), t(4;11), other MLL rearrangements) or > 1 cycle to obtain CR; second or greater CR.
- Burkitt's lymphoma in CR2 or subsequent CR
- Natural Killer cell malignancies
Show all 23Show fewer
- Myeloproliferative syndromes/diseases: Chronic myelogenous leukemia (CML) in chronic or accelerated phase but patients must have failed or been intolerant to Imatinib mesylate. EXCLUDED: CML in refractory blast crisis, myelofibrosis, polycythemia vera, and essential thrombocytosis.
- Myelodysplastic Syndrome: any subtype including refractory anemia (RA) if severe pancytopenia, high risk complex cytogenetics or International Prognostic Scoring System (IPSS) ≥ intermediate-2 (Int-2). Blasts must be less than 5%. If 5% or more requires therapy pre-transplant to reduce blast count to ≤5%. Patients who receive single agent 5-azacytidine, decitabine or immunomodulating drugs are eligible.
- Large-cell lymphoma, Hodgkin's lymphoma and multiple myeloma: patients with chemotherapy sensitive disease that has failed or who are ineligible for an autologous transplant. Patients are eligible for umbilical cord blood (UCB) transplantation if there is no evidence of progressive disease by imaging modalities and/or biopsy. Persistent PET activity, though possibly related to lymphoma, IS NOT an exclusion criterion in the absence of computated tomography (CT) changes in size indicating progression. Large-cell and Hodgkin's lymphoma that is progressive on salvage therapy is NOT eligible. Patients with stable disease are eligible for transplantation if the largest residual nodal mass is < 5 cm (approximately). For patients who have responded to preceding therapy, the largest residual mass must represent a 50% reduction and be < 7.5 cm (approximately).
- Chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL), marginal zone B-cell lymphoma, follicular lymphoma which have progressed after at least two prior therapies. Patients with bulky disease should be considered for debulking chemotherapy before transplant. Patients with refractory disease are eligible, unless has bulky disease and an estimated tumor doubling time of less than one month. Patients with stable disease are eligible for transplantation if the largest residual nodal mass is < 5 cm (approximately). For patients who have responded to preceding therapy, the largest residual mass must represent a 50% reduction and be < 7.5 cm (approximately).
- Lymphoplasmacytic lymphoma, mantle-cell lymphoma, prolymphocytic leukemia are eligible after initial therapy if chemotherapy sensitive. Mantle-cell lymphoma that is progressive on salvage therapy is NOT eligible. Patients with stable disease are eligible for transplantation if the largest residual nodal mass is < 5 cm (approximately). For patients who have responded to preceding therapy, the largest residual mass must represent a 50% reduction and be < 7.5 cm (approximately).
- Umbilical Cord Blood Graft Selection - Two UCB units will be compose the graft, and each unit must be a 4-6 HLA-A, B, DRB1 antigen match to each other, as well as a 4-6 antigen match to the recipient. The combined cryopreserved nucleated cell dose of the 2 units must be ≥ 3 X 10^7/kg with each unit having a minimum cell dose of 1.5 X 10^7/kg. UCB units will be selected according to a common umbilical cord blood graft selection algorithm
- Performance Status - adequate performance status defined as Karnofsky score ≥ 60
- Age 18 to 70 years of age; patients ≥ 70 but ≤ 75 years are eligible if the co-morbidity score is ≤ 2
- Organ Function
- Cardiac: Left ventricular ejection fraction > 35%; absence of decompensated congestive heart failure; absence of uncontrolled arrhythmia
- Pulmonary: DLCO > 30% of predicted; absence of O2 requirements
- Hepatic: ALT, AST, alkaline phosphatase and bilirubin < 5 x upper limit of normal
- Renal: Creatinine ≤ 2 mg/dl (patients with a creatinine > 1.2 or history of renal dysfunction must have calculated glomerular filtration rate (GFR) > 40 mL/min/1.73m2)
- If recent mold infection e.g. Aspergillus - must have minimum of 30 days of appropriate treatment before transplant and infection controlled and be cleared by Infectious Disease.
- The following conditions must be met:
- If prior myeloablative autologous transplant, must be > 3 months but ≤ 12 months from transplant OR have received at least 2 cycles of multi-agent or highly immunosuppressive chemotherapy (i.e. induction for acute leukemia) within the 3 months preceding this study. OR
- If neither prior myeloablative autologous transplant ≤ 12 months from transplant nor have received at least 2 cycles of multi-agent or highly immunosuppressive chemotherapy (i.e. induction for acute leukemia) within the 3 months preceding this study, patients are eligible as long as they receive equine anti-thymocyte globulin as part of the conditioning regimen.
Who cannot join
- Patients who have an available, medically suitable, 5-6/6 HLA-A, B, DRB1 matched sibling donor
- Patients who are eligible for autologous transplantation
- Prior allogeneic transplant
- Acquired or inherited bone marrow failure syndromes such as aplastic anemia and Fanconi anemia
- Pregnant or breast feeding
- Evidence of HIV infection or known HIV positive serology
- Current uncontrolled serious infection
- Active central nervous system malignancy
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Minnesota Medical Center - Fairview
Minneapolis, Minnesota, 55455
About this study
Official title: MT2008-15: Transplantation of Unrelated Donor Umbilical Cord Blood in Patients With Hematological Malignancies Using a Nonmyeloablative Preparative Regimen and Priming With Complement 3a Fragment (C3a)
RATIONALE: Giving low doses of chemotherapy and total-body irradiation before a donor umbilical cord blood transplant helps stop the growth of cancer cells. It may also 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 cells (graft-versus-tumor effect).
PURPOSE: This phase I trial is studying the safety of donor umbilical cord blood transplant after fludarabine phosphate, cyclophosphamide, and total-body irradiation in treating patients with high-risk hematologic cancer (now closed).
The Phase II part of this trial is studying whether priming one of two UCB units with C3a facilitates engraftment of the treated unit.
Read the detailed description
OUTLINE:
- Nonmyeloablative preparative regimen: Patients receive fludarabine phosphate IV over 1 hour on days -6 to -2 and cyclophosphamide IV over 2 hours on day -6. Patients then undergo total-body irradiation on day -1. Some patients also receive anti-thymocyte globulin IV every 12 hours on days -6, -5, and -4.
- Umbilical cord blood (UCB) transplantation: Patients undergo unmanipulated UCB transplantation followed by complement 3a fragment primed UCB transplantation on day 0.
Treatment for graft-vs-host disease prophylaxis is also given.
After completion of study therapy, patients are followed up periodically for up to 2 years.
- Conditions
- Leukemia, Lymphoma, Multiple Myeloma, Myelodysplastic Syndromes
- Being tested
- cyclophosphamide, fludarabine phosphate, Total body irradiation, Umbilical cord blood unit with C3a fragment, Unmanipulated UCB Unit
- Medical subject headings
- Leukemia, Lymphoma, Multiple Myeloma, Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
cyclophosphamideDrug
Also called Cytoxan
50 mg/kg intravenously (IV) over 2 hours on Day -6.
Also called Fludara
40 mg/m^2 over 1 hour on Days -6 through -2.
Total body irradiationRadiation
200 cGy on Day -1
Umbilical cord blood unit with C3a fragmentBiological
On Day 0, the C3a primed UCB unit will be infused intravenously SECOND, within 30 minutes of the completion of the infusion of the unmanipulated UCB unit, through a central line without in-line filtration in a manner identical to the unmanipulated UCB unit.
Unmanipulated UCB UnitBiological
On Day 0, the unmanipulated UCB unit will be infused FIRST through a central line without in-line filtration per institutional guidelines.
| Study group | Role | What they receive |
|---|---|---|
| Complement Fragment 3A - Small Cell Dose | Experimental | Patients who received complement fragment 3a (C3a) priming of the UCB unit with the smaller cell dose following preparation regimen and radiation. Receives: Drug: cyclophosphamide; Drug: fludarabine phosphate; Radiation: Total body irradiation; Biological: Umbilical cord blood unit with C3a fragment; Biological: Unmanipulated UCB Unit |
| Complement Fragment A - Larger Cell Dose | Experimental | Patients who received complement fragment 3a (C3a) priming of the UCB unit with the larger cell dose. Receives: Drug: cyclophosphamide; Drug: fludarabine phosphate; Radiation: Total body irradiation; Biological: Umbilical cord blood unit with C3a fragment; Biological: Unmanipulated UCB Unit |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Patients With the Complement 3a (C3a) Unit Predominating
Efficacy of the pre-incubation of one of two umbilical cord blood (UCB) units with C3a as part of a unrelated donor double UCB nonmyeloablative transplantation in patients with high-risk hematological malignancies.
Measured: Day 180
Secondary outcomes (19)
Neutrophil Engraftment
Achieving 500 neutrophils/uL by day 42.
Measured: Day 42
Donor Chimerism in Blood
Percentage of donor DNA present in the peripheral blood
Measured: Day 28
Incidence of Grades II-IV Graft-vs-host Disease
Development of graft-versus-host disease through day 100.
Measured: Day 0 through Day 100
Non-Relapse Mortality
Deaths not due to relapse.
Measured: Day 180
Overall Survival
Survival (alive) from transplantation to last follow-up.
Measured: Day 360
Bone Marrow Chimerism
Percentage of donor DNA in the bone marrow.
Measured: Day 21
Chronic Graft-Versus-Host Disease
Patients who developed chronic graft-versus-host disease.
Measured: Day 360
Relapse of Disease
Patients who developed disease relapse after transplantation.
Measured: Day 360
Disease Progression
Patients who developed disease progression after transplantation.
Measured: Day 360
Platelet Recovery
Number of patients with >20,000 platelets/uL by day 180
Measured: Day 180
Donor Chimerism in Blood
Percentage of donor DNA present in the peripheral blood
Measured: Day 60
Incidence of Grades III-IV Graft-vs-host Disease
Development of graft-versus-host disease by day 100.
Measured: 0 to 100 days
Non-relapse Mortality
Deaths not due to relapse.
Measured: Day 360
Overall Survival at Day 720
Survival (alive) from transplantation to last follow-up at day 720.
Measured: 720 days
Relapse of Disease
Patients who developed disease relapse after transplantation.
Measured: Day 720
Disease Progression
Patients who developed disease progression after transplantation.
Measured: Day 720
Donor Chimerism
Percentage of donor DNA in the bone marrow.
Measured: Day 100
Donor Chimerism
Percentage of donor DNA in the bone marrow.
Measured: Day 180
Donor Chimerism
Percentage of donor DNA in the bone marrow.
Measured: Day 360
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Aug 2009 (estimated)
- Study startMar 2010
- Primary completionAug 2012
- Study completionAug 2013
Why it stopped: Lack of efficacy after interim analysis
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.
- Other trials for LeukemiaStudies 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
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 31 participants (actual)
Sponsor and investigators
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Sponsor
- Investigators
- Claudio G. Brunstein, MD, PhD, Principal investigator, Masonic Cancer Center, University of Minnesota
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00963872
- Sponsor's protocol ID
- 2008UC097
- Other IDs
- MT2008-15, 0809M46361, RC1HL099447
- Status last verified
- Dec 2017
- First submitted
- 21 Aug 2009
- Last updated
- 28 Dec 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
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
- MT2008-15: Transplantation of Unrelated Donor Umbilical Cord Blood in Patients With Hematological Malignancies Using a Nonmyeloablative Preparative Regimen and Priming With Complement 3a Fragment (C3a)
- ClinicalTrials.gov ID
- NCT00963872
- Acronym / sponsor ID
- 2008UC097
- Conditions
- Leukemia, Lymphoma, Multiple Myeloma, Myelodysplastic Syndromes
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Lack of efficacy after interim analysis
- Enrolment
- 31 participants (actual)
- Lead sponsor
- Masonic Cancer Center, University of MinnesotaUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- Mar 2010
- Primary completion
- Aug 2012 (actual)
- Study completion
- Aug 2013 (actual)
- First posted
- 24 Aug 2009 (estimated)
- Results
- Posted 10 Nov 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
RATIONALE: Giving low doses of chemotherapy and total-body irradiation before a donor umbilical cord blood transplant helps stop the growth of cancer cells. It may also 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 cells (graft-versus-tumor effect).
PURPOSE: This phase I trial is studying the safety of donor umbilical cord blood transplant after fludarabine phosphate, cyclophosphamide, and total-body irradiation in treating patients with high-risk hematologic cancer (now closed).
The Phase II part of this trial is studying whether priming one of two UCB units with C3a facilitates engraftment of the treated unit.
Detailed description
OUTLINE:
- Nonmyeloablative preparative regimen: Patients receive fludarabine phosphate IV over 1 hour on days -6 to -2 and cyclophosphamide IV over 2 hours on day -6. Patients then undergo total-body irradiation on day -1. Some patients also receive anti-thymocyte globulin IV every 12 hours on days -6, -5, and -4.
- Umbilical cord blood (UCB) transplantation: Patients undergo unmanipulated UCB transplantation followed by complement 3a fragment primed UCB transplantation on day 0.
Treatment for graft-vs-host disease prophylaxis is also given.
After completion of study therapy, patients are followed up periodically for up to 2 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Complement Fragment 3A - Small Cell Dose | Experimental | Drug: cyclophosphamide, Drug: fludarabine phosphate, Radiation: Total body irradiation, Biological: Umbilical cord blood unit with C3a fragment, Biological: Unmanipulated UCB UnitPatients who received complement fragment 3a (C3a) priming of the UCB unit with the smaller cell dose following preparation regimen and radiation. |
| Complement Fragment A - Larger Cell Dose | Experimental | Drug: cyclophosphamide, Drug: fludarabine phosphate, Radiation: Total body irradiation, Biological: Umbilical cord blood unit with C3a fragment, Biological: Unmanipulated UCB UnitPatients who received complement fragment 3a (C3a) priming of the UCB unit with the larger cell dose. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (23 listed)
- Disease Criteria
- Umbilical Cord Blood Graft Selection - Two UCB units will be compose the graft, and each unit must be a 4-6 HLA-A, B, DRB1 antigen match to each other, as well as a 4-6 antigen match to the recipient. The combined cryopreserved nucleated cell dose of the 2 units must be ≥ 3 X 10^7/kg with each unit having a minimum cell dose of 1.5 X 10^7/kg. UCB units will be selected according to a common umbilical cord blood graft selection algorithm
- Performance Status - adequate performance status defined as Karnofsky score ≥ 60
- Age 18 to 70 years of age; patients ≥ 70 but ≤ 75 years are eligible if the co-morbidity score is ≤ 2
- Organ Function
Key exclusion criteria (8 listed)
- Patients who have an available, medically suitable, 5-6/6 HLA-A, B, DRB1 matched sibling donor
- Patients who are eligible for autologous transplantation
- Prior allogeneic transplant
- Acquired or inherited bone marrow failure syndromes such as aplastic anemia and Fanconi anemia
- Pregnant or breast feeding
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 |
|---|---|---|
| cyclophosphamideAlso: Cytoxan50 mg/kg intravenously (IV) over 2 hours on Day -6. | Drug | Complement Fragment 3A - Small Cell Dose, Complement Fragment A - Larger Cell Dose |
| fludarabine phosphateAlso: Fludara40 mg/m^2 over 1 hour on Days -6 through -2. | Drug | Complement Fragment 3A - Small Cell Dose, Complement Fragment A - Larger Cell Dose |
| Total body irradiation200 cGy on Day -1 | Radiation | Complement Fragment 3A - Small Cell Dose, Complement Fragment A - Larger Cell Dose |
| Umbilical cord blood unit with C3a fragmentOn Day 0, the C3a primed UCB unit will be infused intravenously SECOND, within 30 minutes of the completion of the infusion of the unmanipulated UCB unit, through a central line without in-line filtration in a manner identical to the unmanipulated UCB unit. | Biological | Complement Fragment 3A - Small Cell Dose, Complement Fragment A - Larger Cell Dose |
| Unmanipulated UCB UnitOn Day 0, the unmanipulated UCB unit will be infused FIRST through a central line without in-line filtration per institutional guidelines. | Biological | Complement Fragment 3A - Small Cell Dose, Complement Fragment A - Larger Cell Dose |
Primary outcome
Number of Patients With the Complement 3a (C3a) Unit Predominating
Time frame: Day 180
Efficacy of the pre-incubation of one of two umbilical cord blood (UCB) units with C3a as part of a unrelated donor double UCB nonmyeloablative transplantation in patients with high-risk hematological malignancies.
19 secondary outcomes
Neutrophil Engraftment
Time frame: Day 42
Donor Chimerism in Blood
Time frame: Day 28
Incidence of Grades II-IV Graft-vs-host Disease
Time frame: Day 0 through Day 100
Non-Relapse Mortality
Time frame: Day 180
Overall Survival
Time frame: Day 360
Bone Marrow Chimerism
Time frame: Day 21
Chronic Graft-Versus-Host Disease
Time frame: Day 360
Relapse of Disease
Time frame: Day 360
Disease Progression
Time frame: Day 360
Platelet Recovery
Time frame: Day 180
Donor Chimerism in Blood
Time frame: Day 60
Incidence of Grades III-IV Graft-vs-host Disease
Time frame: 0 to 100 days
Non-relapse Mortality
Time frame: Day 360
Overall Survival at Day 720
Time frame: 720 days
Relapse of Disease
Time frame: Day 720
Disease Progression
Time frame: Day 720
Donor Chimerism
Time frame: Day 100
Donor Chimerism
Time frame: Day 180
Donor Chimerism
Time frame: Day 360
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.
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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.
Claudio G. Brunstein, MD, PhD principal investigator
Masonic Cancer Center, University of Minnesota
1 site 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 cyclophosphamide, fludarabine phosphate and Total body irradiation in people with Leukemia and Lymphoma, run by Masonic Cancer Center, University of Minnesota at 1 site 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 23 inclusion and 8 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.
Where is the study taking place?
The record lists 1 site 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?
Yes. Results are posted in the registry (first posted 10 Nov 2015). 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?
Number of Patients With the Complement 3a (C3a) Unit Predominating (time frame: Day 180). 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 28 Dec 2017.