Acute Myeloid Leukemia (AML)NCT01962636
Umbilical Cord Blood Transplantation Using a Myeloablative Preparative Regimen for Hematological Diseases
A study testing Fludarabine, Cyclophosphamide and Total Body Irradiation in people with Acute Myeloid Leukemia (AML) and Acute Lymphocytic Leukemia (ALL), run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
Trial summary
This is a treatment guideline for an unrelated umbilical cord blood transplant (UCBT) using a myeloablative preparative regimen for the treatment of hematological diseases, including, but not limited to acute leukemias. Read more about this study
- Who is it for?
- Ages Up to 55 years
- Conditions the study is for
- Acute Myeloid Leukemia (AML), Acute Lymphocytic Leukemia (ALL), Chronic Myelogenous Leukemia
- What is being tested?
- Fludarabine, Cyclophosphamide 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
- 200 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2016to 14 May 2026
- 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 Up to 55 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Eligible Disease Status
- Acute Myeloid Leukemia (AML): high risk CR1 (as evidenced by preceding MDS, high risk cytogenetics, ≥ 2 cycles to obtain CR, erythroblastic or megakaryocytic leukemia; 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%.
- Very high risk pediatric patients with AML: Patients <21 years, however, are eligible with (M2 marrow) with < 25% blasts in marrow after having failed one or more cycles of chemotherapy.
- Acute Lymphocytic Leukemia (ALL): high risk CR1 as defined by cytogenetics (such as t(9;22), t (1:19), t(4;11), other MLL rearrangements, hypodiploidy, or IKZF1 abnormalities), DNA index < 0.81, > 1 cycle to obtain CR or presence minimal residual disease (MRD). Patients in CR2+ are eligible. 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%.
- Very high risk pediatric patients with ALL: patients <21 years are also considered high risk CR1 if they had M2 or M3 marrow at day 42 from the initiation of induction or M3 marrow at the end of induction. They are eligible once they achieved a complete remission.
- Chronic Myelogenous Leukemia excluding refractory blast crisis: To be eligible in first chronic phase (CP1) patient must have failed or be intolerant to imatinib mesylate.
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- Plasma Cell Leukemia after initial therapy, who achieved at least a partial remission
- Advanced Myelofibrosis
- Myelodysplasia (MDS) IPSS INT-2 or High Risk (i.e. RAEB, RAEBt) or Refractory Anemia with severe pancytopenia or high risk cytogenetics: Blasts must be < 10% by a representative bone marrow aspirate morphology.
- Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CLL/SLL), Marginal Zone B-Cell Lymphoma or Follicular Lymphoma are eligible if there was disease progression/relapse within 12 of achieving a partial or complete remission. Patients who had remissions lasting > 12 months, are eligible after at least two prior therapies. Patients with bulky disease (nodal mass greater than 5 cm) should be considered for de-bulking chemotherapy before transplant.
- Lymphoplasmacytic Lymphoma, Mantle-Cell Lymphoma, Prolymphocytic Leukemia are eligible after initial therapy in CR1+ or PR1+.
- Large Cell NHL > CR2/> PR2: Patients in CR2/PR2 with initial short remission (<6 months) are eligible.
- Lymphoblastic Lymphoma, Burkitt's Lymphoma, and other high-grade NHL after initial therapy if stage III/IV in CR1/PR1 or after progression if stage I/II < 1 year.
- Multiple Myeloma beyond PR2: Patients with chromosome 13 abnormalities, first response lasting less than 6 months, or β-2 microglobulin > 3 mg/L, may be considered for this protocol after initial therapy.
- Myeloproliferative Syndromes
- Availability of suitable UCB unit(s)
- 0 to 55 years
- Voluntary written consent (adult or parental/guardian)
Who cannot join
- previous irradiation that precludes the safe administration of TBI - Radiation Oncology will evaluate all patients who have had previous radiation therapy
- chemotherapy refractory large cell and high grade NHL (ie progressive disease after > 2 salvage regimens)
- if ≤ 18 years old, prior myeloablative transplant within the last 6 months. If >18 years old prior myeloablative allotransplant or autologous transplant
- extensive prior therapy including > 12 months alkylator therapy or > 6 months alkylator therapy with extensive radiation
- pregnant or breastfeeding
- HIV positive
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Minnesota Masonic Cancer Center
Minneapolis, Minnesota, 55455
About this study
Official title: Umbilical Cord Blood Transplantation Using a Myeloablative Preparative Regimen for the Treatment of Hematological Diseases
This is a treatment guideline for an unrelated umbilical cord blood transplant (UCBT) using a myeloablative preparative regimen for the treatment of hematological diseases, including, but not limited to acute leukemias. The myeloablative preparative regimen will consist of cyclophosphamide (CY), fludarabine (FLU) and fractionated total body irradiation (TBI).
Read the detailed description
This is a study to collect routine clinical data from UCBT using unrelated single or double UCB units as an alternative, non-HLA-matched stem cell source for patients with hematological diseases.
- data collection from transplant preparative therapy consisting of treatments with chemotherapeutic regimens and total body irradiation.
- data collection from umbilical cord blood selection and infusion.
- data collection from standard supportive disease and transplant related care.
Pre- and post-transplant medication, UCB selection and infusion, supportive care, and follow-up will be according to the current University of Minnesota BMT guidelines.
An average of 18 patients are expected to be treated on this protocol per year.
- Conditions
- Acute Myeloid Leukemia (AML), Acute Lymphocytic Leukemia (ALL), Chronic Myelogenous Leukemia, Plasma Cell Leukemia, Myelofibrosis, Myelodysplasia
- Being tested
- Fludarabine, Cyclophosphamide, Total Body Irradiation, Cyclosporine A, Mycophenylate mofetil
- Keywords
- Umbilical Cord Transplant, Acute Myeloid Leukemia (AML), Acute Lymphocytic Leukemia (ALL), Chronic Myelogenous Leukemia, Plasma Cell Leukemia, Myelofibrosis, Myelodysplasia, Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma, Marginal Zone B-Cell Lymphoma, Follicular Lymphoma, Lymphoplasmacytic Lymphoma, Mantle-Cell Lymphoma, Prolymphocytic Leukemia, Large Cell Non-Hodgkin Lymphoma, Lymphoblastic Lymphoma, Burkitt's Lymphoma, Non-Hodgkin Lymphoma, Multiple Myeloma
- Medical subject headings
- Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Leukemia, Plasma Cell, Primary Myelofibrosis, Anemia, Refractory, with Excess of Blasts, Leukemia, Lymphocytic, Chronic, B-Cell, Lymphoma, B-Cell, Marginal Zone
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FludarabineDrug
Also called Fludara
25 mg/m^2 IV of Fludarabine will be given over 1 hour on days -8, -7, and -6 pre-UCB transplant.
CyclophosphamideDrug
Also called Cytoxan
60 mg/kg IV of Cyclophosphamide will be given over 2 hours on days -7 and -6 pre-UCB transplant.
Total Body IrradiationRadiation
165 cGy of total body irradiation will be given twice a day on days -4, -3, -2, and -1.
Cyclosporine ADrug
Also called CSA
Cyclosporine A (CSA) will start day -3 and will be administered PO/IV maintaining a trough level between 200 and 400 ng/mL. For adults the initial dose will be 2.5 mg/kg IV over 1 hour every 12 hours. For children < 40 kg the initial dose will be 2.5 mg/kg IV over 1 hour every 8 hours.
Mycophenylate mofetilDrug
Also called MMF
Mycophenylate mofetil (MMF) 3 gram/day IV/PO for patients who are ≥ 40 kg divided in 2 or 3 doses. Pediatric patient (<40 kilograms) will receive MMF at the dose of 15 mg/kg/dose every 8 hours beginning day -3.
Umbilical cord bloodBiological
Also called UCB
Pre-medications and UCB infusion will be per current institutional policies/guidelines. The infusion of the first UCB unit should begin within 15 minutes, and no later than 30 minutes after arrival on the Unit. If 2 units are used, both cords will be infused within 30-60 minutes of each other as deemed clinically safe by the BMT attending or designee.
| Study group | Role | What they receive |
|---|---|---|
| Umbilical Cord Blood Transplant | Experimental | The myeloablative preparative regimen will consist of cyclophosphamide (CY), fludarabine (FLU) and fractionated total body irradiation (TBI)followed by umbilical cord blood transplant. Immunosuppressive Cyclosporine and Mycophenylate Mofetil (MMF) will be administered pre- and post UCBT. Receives: Drug: Fludarabine; Drug: Cyclophosphamide; Radiation: Total Body Irradiation; Drug: Cyclosporine A; Drug: Mycophenylate mofetil; Biological: Umbilical cord blood |
What is being measured
The primary outcome is the main question the study is designed to answer.
Survival at 1 year post-transplant
The number of patients that are still living 1 year after UCBT.
Measured: 1 year
Secondary outcomes (9)
Incidence of neutrophil engraftment at day 42.
Number of subjects with neutrophil engraftment at day 42 post UCBT.
Measured: 42 days
Platelet engraftment at 1 year.
Number of patients with platelet engraftment at 1 year post UCBT.
Measured: 1 year
Pattern of chimerism after transplant.
Pattern of chimerism after transplant. Chimerism will be plotted with box-plots and described over time.
Measured: 1 year
Incidence of graft failure.
Cumulative incidence of graft failure after UCBT.
Measured: 100 days
Incidence of acute graft versus host disease at 100 days.
Cumulative incidence will be used to estimate acute graft versus host disease 100 days after UCBT.
Measured: 100 days
Incidence of chronic graft versus host disease at 1 year.
Cumulative incidence will be used to estimate chronic GVHD at 1 year post UCBT.
Measured: 1 year
Incidence of transplant related mortality at 6 months.
Cumulative incidence will be used to estimate transplant related mortality at 6 months post UCBT.
Measured: 6 months
Incidence of disease free survival
Kaplan-Meier curves will be used to estimate disease-free survival at 1 and 2 years post UCBT.
Measured: 1, 2 years
Incidence of overall survival.
Kaplan-Meier curves will be used to estimate overall survival at 1 and 2 years post UCBT.
Measured: 1, 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Oct 2013 (estimated)
- Study startDec 2016
- Primary completion14 May 2026
- Study completion14 May 2026
Why it stopped: No longer a research question to answer.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid Leukemia (AML)Studies 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 200 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Claudio Brunstein, MD, Principal investigator, University of Minnesota
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01962636
- Sponsor's protocol ID
- 2013OC013
- Status last verified
- May 2026
- First submitted
- 10 Oct 2013
- Last updated
- 20 May 2026
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Umbilical Cord Blood Transplantation Using a Myeloablative Preparative Regimen for the Treatment of Hematological Diseases
- ClinicalTrials.gov ID
- NCT01962636
- Acronym / sponsor ID
- 2013OC013
- Conditions
- Acute Myeloid Leukemia (AML), Acute Lymphocytic Leukemia (ALL), Chronic Myelogenous Leukemia, Plasma Cell Leukemia, Myelofibrosis, Myelodysplasia
- Keywords
- Umbilical Cord Transplant, Acute Myeloid Leukemia (AML), Acute Lymphocytic Leukemia (ALL), Chronic Myelogenous Leukemia, Plasma Cell Leukemia, Myelofibrosis, Myelodysplasia, Chronic Lymphocytic Leukemia
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: No longer a research question to answer.
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- Masonic Cancer Center, University of MinnesotaUniversity, hospital or other
- Study start
- Dec 2016
- Primary completion
- 14 May 2026 (actual)
- Study completion
- 14 May 2026 (actual)
- First posted
- 14 Oct 2013 (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.
This is a treatment guideline for an unrelated umbilical cord blood transplant (UCBT) using a myeloablative preparative regimen for the treatment of hematological diseases, including, but not limited to acute leukemias. The myeloablative preparative regimen will consist of cyclophosphamide (CY), fludarabine (FLU) and fractionated total body irradiation (TBI).
Detailed description
This is a study to collect routine clinical data from UCBT using unrelated single or double UCB units as an alternative, non-HLA-matched stem cell source for patients with hematological diseases.
- data collection from transplant preparative therapy consisting of treatments with chemotherapeutic regimens and total body irradiation.
- data collection from umbilical cord blood selection and infusion.
- data collection from standard supportive disease and transplant related care.
Pre- and post-transplant medication, UCB selection and infusion, supportive care, and follow-up will be according to the current University of Minnesota BMT guidelines.
An average of 18 patients are expected to be treated on this protocol per year.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Umbilical Cord Blood Transplant | Experimental | Drug: Fludarabine, Drug: Cyclophosphamide, Radiation: Total Body Irradiation, Drug: Cyclosporine A, Drug: Mycophenylate mofetil, Biological: Umbilical cord bloodThe myeloablative preparative regimen will consist of cyclophosphamide (CY), fludarabine (FLU) and fractionated total body irradiation (TBI)followed by umbilical cord blood transplant. Immunosuppressive Cyclosporine and Mycophenylate Mofetil (MMF) will be administered pre- and post UCBT. |
Who may qualify
- Ages
- Up to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (18 listed)
- Eligible Disease Status
- Availability of suitable UCB unit(s)
- 0 to 55 years
- Voluntary written consent (adult or parental/guardian)
Key exclusion criteria (6 listed)
- previous irradiation that precludes the safe administration of TBI - Radiation Oncology will evaluate all patients who have had previous radiation therapy
- chemotherapy refractory large cell and high grade NHL (ie progressive disease after > 2 salvage regimens)
- if ≤ 18 years old, prior myeloablative transplant within the last 6 months. If >18 years old prior myeloablative allotransplant or autologous transplant
- extensive prior therapy including > 12 months alkylator therapy or > 6 months alkylator therapy with extensive radiation
- pregnant or breastfeeding
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 |
|---|---|---|
| FludarabineAlso: Fludara25 mg/m^2 IV of Fludarabine will be given over 1 hour on days -8, -7, and -6 pre-UCB transplant. | Drug | Umbilical Cord Blood Transplant |
| CyclophosphamideAlso: Cytoxan60 mg/kg IV of Cyclophosphamide will be given over 2 hours on days -7 and -6 pre-UCB transplant. | Drug | Umbilical Cord Blood Transplant |
| Total Body Irradiation165 cGy of total body irradiation will be given twice a day on days -4, -3, -2, and -1. | Radiation | Umbilical Cord Blood Transplant |
| Cyclosporine AAlso: CSACyclosporine A (CSA) will start day -3 and will be administered PO/IV maintaining a trough level between 200 and 400 ng/mL. For adults the initial dose will be 2.5 mg/kg IV over 1 hour every 12 hours. For children < 40 kg the initial dose will be 2.5 mg/kg IV over 1 hour every 8 hours. | Drug | Umbilical Cord Blood Transplant |
| Mycophenylate mofetilAlso: MMFMycophenylate mofetil (MMF) 3 gram/day IV/PO for patients who are ≥ 40 kg divided in 2 or 3 doses. Pediatric patient (<40 kilograms) will receive MMF at the dose of 15 mg/kg/dose every 8 hours beginning day -3. | Drug | Umbilical Cord Blood Transplant |
| Umbilical cord bloodAlso: UCBPre-medications and UCB infusion will be per current institutional policies/guidelines. The infusion of the first UCB unit should begin within 15 minutes, and no later than 30 minutes after arrival on the Unit. If 2 units are used, both cords will be infused within 30-60 minutes of each other as deemed clinically safe by the BMT attending or designee. | Biological | Umbilical Cord Blood Transplant |
Primary outcome
Survival at 1 year post-transplant
Time frame: 1 year
The number of patients that are still living 1 year after UCBT.
9 secondary outcomes
Incidence of neutrophil engraftment at day 42.
Time frame: 42 days
Platelet engraftment at 1 year.
Time frame: 1 year
Pattern of chimerism after transplant.
Time frame: 1 year
Incidence of graft failure.
Time frame: 100 days
Incidence of acute graft versus host disease at 100 days.
Time frame: 100 days
Incidence of chronic graft versus host disease at 1 year.
Time frame: 1 year
Incidence of transplant related mortality at 6 months.
Time frame: 6 months
Incidence of disease free survival
Time frame: 1, 2 years
Incidence of overall survival.
Time frame: 1, 2 years
Results and safety
The study was stopped early (reason given: No longer a research question to answer.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 May 2026.
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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 Brunstein, MD principal investigator
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 study testing Fludarabine, Cyclophosphamide and Total Body Irradiation in people with Acute Myeloid Leukemia (AML) and Acute Lymphocytic Leukemia (ALL), 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 18 inclusion and 6 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 20 May 2026. 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.
Are results available for this study?
The study was stopped early (reason given: No longer a research question to answer.). 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?
Survival at 1 year post-transplant (time frame: 1 year). 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 20 May 2026.