Acute Myeloid LeukemiaNCT00513318
Pilot Study of Reduced-Intensity Umbilical Cord Blood Transplantation in Adult Patients Wtih Advanced Hematopoietic Malignancies
An observational study of Acute Myeloid Leukemia and Myelodysplasia, run by University of California, San Francisco at 1 site in United States.
Trial summary
This is a pilot study designed to evaluate the safety and feasibility of performing umbilical cord blood transplants in older adults or younger infirm patients with high-risk hematopoeitic malignancies. A novel reduced-intensity preparative regimen for umbilical cord blood transplantation will be used. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Acute Myeloid Leukemia, Myelodysplasia, Acute Lymphoblastic Leukemia
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2004to Jun 2009
- Phase
- Observational
- 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 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Adults with hematopoietic malignancies ages 55 to 70.
Who may be able to join
- Age 55-70 years, or < 55 years if deemed ineligible for conventional high dose chemotherapy by the UCSF SCT Committee or by protocol eligibility requirements for myeloablative therapy. Reasons for ineligibility for myeloablative therapy include:
- Poor cardiac function (i.e. LVEF < 40%)
- Poor pulmonary function (i.e. DLCO < 50%)
- Hepatic dysfunction
- Prior myeloablative therapy
- Availability of donor cord blood (one to three units) matching at > or equal to 4 of 6 HLA antigens (A, B, and DR). HLA class I antigens will be determined by serologic methods, and Class II antigens will be determined by high-resolution DNA typing. Typing will be confirmed by the UCSF Immunogenetics Department. The target UCB TNC dose is > or equal to 3.5 x 10(7) TNC/kg recipient weight, however the absolute minimum TNC requirement is > 2.5 x 10(7) TNC per kilogram) based on cell counts prior to cryopreservation. Cord blood units will be obtained from all available international banks.
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- HLA identical or 1 antigen mismatched related donors or potential HLA-matched unrelated donors (MUD) must NOT be readily available
- Disease types include:
- Acute myeloid leukemia not expected to be curable with chemotherapy. This will include patients with high-risk cytogenetics (-7, -7q, -5, -5q, t(6, 9), t(9, 11), complex (> or equal to 3 abnormalities), Ph(+), evolution from prior myelodysplasia or AML secondary to prior chemotherapy, failure to achieve remission, second, or subsequent remission or refractory relapse. Marrow blasts must be < or equal to 10%. This may be achieved using standard chemotherapy treatment.
- Myelodysplasia with high-risk features. This will include patients with IPSS category INT2 or HI-risk MDS or CMML. Marrow blasts must be < or equal to 20%. If required, chemotherapy may be given to achieve target levels of blasts. Patients are expected to have disease control or not rapidly progressive disease regardless of blast count (but must be < or equal to 20%).
- Acute lymphoblastic leukemia not expected to be cured with chemotherapy. This will include patients with high-risk cytogenetics (Ph+, t(4,11), 11q23 abnormalities, and monosomy 7), patients requiring more than one induction course to achieve remission, as well as patients failing to enter remission or in second or subsequent remission. Marrow blasts must be < or equal to 10%. If required, chemotherapy may be given to achieve target level of blasts.
- Chronic myelogenous leukemia- chronic phase failing imatinib mesylate therapy (either progressive disease or failure to achieve a major cytogenetic response at 1 year following initiation of therapy), accelerated phase failing to achieve a complete cytogenetic remission at 1 year following initiation of therapy, accelerated phase failing to achieve any cytogenetic response at 6 months of therapy, accelerated phase with progressive disease as demonstrated by worsening cytogenetic response in two consecutive analyses separated by 4 weeks or CML in blast crisis. Patients with blast phase of CML must have < 10% blasts in bone marrow.
- Multiple myeloma stage I-III with relapse following autologous transplant, with disease refractory to at least two prior conventional; myeloma therapies or with chromosome 13 abnormalities. Patients with chromosome 13 abnormalities are eligible either at diagnosis or after initial progression.
- Lymphomas including diffuse or follicular large cell lymphoma, mantle cell lymphoma, peripheral T-cell lymphoma, T-NK cell lymphoma, or Hodgkin's disease which has failed to respond to primary therapy, progressed, or recurred after prior therapy. Patients who have relapsed following autologous transplant are eligible.
- Low-grade NHL and Chronic Lymphocytic Leukemia with relapse or refractory disease following, at least, two chemotherapy-based treatment regimens; or with relapse following autologous transplantation.
- Patients must have an ECOG PS < or equal to 2.
- Laboratory requirements:
- Creatinine < 2.0mg/dL and creatinine clearance > 40/m/min (calculated or based on 24-hour urine collection)
- Bilirubin <2.0mg/dL, AST/alkphos <3x upper limit of normal
- Patients with hepatitis C and active hepatitis B (hepatitis B DNA detectable) are eligible only if a liver biopsy is performed and there is grade < or equal to 2 fibrosis and/or inflammation. Patients with a history of HBV infection should be tested for HBeAg, anti-HBe and HBV DNA (quantitative). Patients with active HBV viral replication should receive anti-viral therapy.
- Cardiac ejection fraction > 30%, DLCO > or equal to 40%.
- Negative pregnancy test (for females of reproductive age only).
- Signed informed consent
Who cannot join
- Active infection requiring ongoing antibiotic treatment
- HIV infection
- Poor performance status (ECOG > 2)
- Opinion of BMT Committee that autologous SCT or conventional therapy would be a preferable form of treatment
- Organ function is below requirements
- Pregnancy, or breast-feeding
Trial locations and contacts
1 site in 1 country.
United States1 site
University of California San Francisco
San Francisco, California, 94143
About this study
This is a pilot study designed to evaluate the safety and feasibility of performing umbilical cord blood transplants in older adults or younger infirm patients with high-risk hematopoeitic malignancies. A novel reduced-intensity preparative regimen for umbilical cord blood transplantation will be used. One to a maximum of three cord blood units, depending on cell count, will be administered to facilitate engraftment. Ten patients will be enrolled with an expected accrual rate of 3-4 patients per year and with a goal of completing accrual within 2-3 years.
Read the detailed description
Primary Objective:
- To assess the feasibility of performing umbilical cord blood transplants in older adults or younger infirm patients using a reduced-intensity preparative regimen. Feasibility of the procedure is defined as an engraftment rate of >80% at Day 180 post-transplantation and a transplant related mortality (TRM) of <50% at Day 100. A TRM of >50% will be considered unacceptable.
Secondary objectives:
- To describe the time to neutrophil and platelet recovery following mini-UCB transplantation.
- To assess lineage-specific chimerism following transplantation and to describe the contribution of each individual CB unit to post-transplantation hematopoeisis.
- To describe disease-specific, event-free and overall survival rates at 180 and 360 days.
- To describe the incidence, severity, and timing of acute and chronic GVHD following reduced-intensity UCB transplantation.
- To evaluate T-cell, B-cell, and NK cell recovery following reduced-intensity UCB transplantation.
- Conditions
- Acute Myeloid Leukemia, Myelodysplasia, Acute Lymphoblastic Leukemia, Chronic Myelogenous Leukemia, Multiple Myeloma, Lymphomas
- Keywords
- Umbilical cord blood transplantation, hematopoietic malignancies
- Medical subject headings
- Leukemia, Myeloid, Acute, Anemia, Refractory, with Excess of Blasts, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Multiple Myeloma, Lymphoma, Leukemia, Lymphocytic, Chronic, B-Cell, Hematologic Neoplasms
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Aug 2007 (estimated)
- Study startAug 2004
- Primary completionJun 2009
- Study completionJun 2009
Why it stopped: Insufficient enrollment to justify keeping open.
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 University of California, San FranciscoThe 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
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of California, San Francisco
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Thomas G. Martin, M.D., Principal investigator, University of California, San Francisco
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00513318
- Sponsor's protocol ID
- UC2407
- Status last verified
- Aug 2013
- First submitted
- 7 Aug 2007
- Last updated
- 2 Aug 2013
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 2 Aug 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study of Reduced-Intensity Umbilical Cord Blood Transplantation in Adult Patients Wtih Advanced Hematopoietic Malignancies
- ClinicalTrials.gov ID
- NCT00513318
- Acronym / sponsor ID
- UC2407
- Conditions
- Acute Myeloid Leukemia, Myelodysplasia, Acute Lymphoblastic Leukemia, Chronic Myelogenous Leukemia, Multiple Myeloma, Lymphomas
- Keywords
- Umbilical cord blood transplantation, hematopoietic malignancies
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Insufficient enrollment to justify keeping open.
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- University of California, San FranciscoUniversity, hospital or other
- Study start
- Aug 2004
- Primary completion
- Jun 2009 (actual)
- Study completion
- Jun 2009 (actual)
- First posted
- 8 Aug 2007 (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 pilot study designed to evaluate the safety and feasibility of performing umbilical cord blood transplants in older adults or younger infirm patients with high-risk hematopoeitic malignancies. A novel reduced-intensity preparative regimen for umbilical cord blood transplantation will be used. One to a maximum of three cord blood units, depending on cell count, will be administered to facilitate engraftment. Ten patients will be enrolled with an expected accrual rate of 3-4 patients per year and with a goal of completing accrual within 2-3 years.
Detailed description
Primary Objective:
- To assess the feasibility of performing umbilical cord blood transplants in older adults or younger infirm patients using a reduced-intensity preparative regimen. Feasibility of the procedure is defined as an engraftment rate of >80% at Day 180 post-transplantation and a transplant related mortality (TRM) of <50% at Day 100. A TRM of >50% will be considered unacceptable.
Secondary objectives:
- To describe the time to neutrophil and platelet recovery following mini-UCB transplantation.
- To assess lineage-specific chimerism following transplantation and to describe the contribution of each individual CB unit to post-transplantation hematopoeisis.
- To describe disease-specific, event-free and overall survival rates at 180 and 360 days.
- To describe the incidence, severity, and timing of acute and chronic GVHD following reduced-intensity UCB transplantation.
- To evaluate T-cell, B-cell, and NK cell recovery following reduced-intensity UCB transplantation.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Adults with hematopoietic malignancies ages 55 to 70.
Key inclusion criteria (23 listed)
- Age 55-70 years, or < 55 years if deemed ineligible for conventional high dose chemotherapy by the UCSF SCT Committee or by protocol eligibility requirements for myeloablative therapy. Reasons for ineligibility for myeloablative therapy include:
- Availability of donor cord blood (one to three units) matching at > or equal to 4 of 6 HLA antigens (A, B, and DR). HLA class I antigens will be determined by serologic methods, and Class II antigens will be determined by high-resolution DNA typing. Typing will be confirmed by the UCSF Immunogenetics Department. The target UCB TNC dose is > or equal to 3.5 x 10(7) TNC/kg recipient weight, however the absolute minimum TNC requirement is > 2.5 x 10(7) TNC per kilogram) based on cell counts prior to cryopreservation. Cord blood units will be obtained from all available international banks.
- HLA identical or 1 antigen mismatched related donors or potential HLA-matched unrelated donors (MUD) must NOT be readily available
- Disease types include:
- Patients must have an ECOG PS < or equal to 2.
Key exclusion criteria (6 listed)
- Active infection requiring ongoing antibiotic treatment
- HIV infection
- Poor performance status (ECOG > 2)
- Opinion of BMT Committee that autologous SCT or conventional therapy would be a preferable form of treatment
- Organ function is below requirements
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Not reported in the source record.
Results and safety
The study was stopped early (reason given: Insufficient enrollment to justify keeping open.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Aug 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.
Thomas G. Martin, M.D. principal investigator
University of California, San Francisco
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Acute Myeloid Leukemia and Myelodysplasia, run by University of California, San Francisco 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 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 2 Aug 2013. 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: Insufficient enrollment to justify keeping open.). 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.
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 2 Aug 2013.