Chronic Myelogenous Leukemia (CML)NCT01768845
Unrelated Umbilical Cord Blood (UBC)Transplantation
A study testing umbilical cord blood (UCB) in people with Chronic Myelogenous Leukemia (CML) and Acute Myelogenous Leukemia (AML), run by West Virginia University at 1 site in United States.
Trial summary
Hematopoietic progenitor cell (HPC- primitive cells in the blood, bone marrow and umbilical cord that can restore the bone marrow) transplant can be a curative therapy for the treatment of hematologic malignancies (a disease of the bone marrow and lymph nodes). Read more about this study
- Who is it for?
- Ages 16 to 70 years
- Conditions the study is for
- Chronic Myelogenous Leukemia (CML), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome
- What is being tested?
- umbilical cord blood (UCB)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 34 participants
- Where
- 1 siteUnited States
- Study dates
- 3 Feb 2009to 6 Apr 2021
- Phase
- Interventional
- 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 16 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age: 16-70 years
- Available 4/6, 5/6, or 6/6 HLA antigen match (using A, B, and DRB1) cord blood unit.
- Eastern Cooperative Oncology Group (ECOG) performance status of 0-2 (Karnofsky greater than or equal to 70%)
- Serum bilirubin less than 2 x upper limit of normal
- Serum creatinine less than 2 mg/dl
- DLCO or FEV1 greater than or equal to 50% predicted
Show all 21Show fewer
- Left ventricular ejection fraction greater than or equal to 35%
- no uncontrolled infection
- If female, not pregnant
- Informed consent given
- No major organ dysfunction precluding transplantation.
- One of the following malignancies or bone marrow failure syndromes:
- Chronic myelogenous leukemia (CML)
- Acute myelogenous leukemia (AML)
- Myelodysplastic syndrome
- Multiple myeloma
- Hodgkin lymphoma
- Non-Hodgkin lymphoma
- Chronic lymphocytic leukemia (CLL)
- Acute lymphocytic leukemia (ALL)
- Severe Aplastic Anemia
Who cannot join
- Patient pregnant
- Age less than 16, greater than 70
- ECOG performance status of greater than 2 (Karnofsky less than 70%)
- Psychiatric disorder or mental deficiency of the patient sufficiently severe as to make compliance with the BMT treatment unlikely, or making informed consent impossible
- Serum bilirubin greater than or equal to 2 x upper limit of normal, transaminases greater than 3 x upper limit of normal
- Serum creatinine greater than or equal to 2 mg/dl
Show all 9Show fewer
- DLCO less than 50% predicted
- Left ventricular ejection fraction less than 35%
- Major anticipated illness or organ failure incompatible with survival from Bone Marrow Transplant (BMT)
Trial locations and contacts
1 site in 1 country.
United States1 site
West Virginia University Hospitals Mary Babb Randolph Cancer Center
Morgantown, West Virginia, 26506
About this study
Official title: Unrelated Umbilical Cord Blood (UCB) Transplantation
Hematopoietic progenitor cell (HPC- primitive cells in the blood, bone marrow and umbilical cord that can restore the bone marrow) transplant can be a curative therapy for the treatment of hematologic malignancies (a disease of the bone marrow and lymph nodes). The source of cells used for the transplant comes from related (sibling) and in cases where there is no sibling match, from unrelated donors through the National Marrow Donor Program. The availability of a suitable donor can be a significant obstacle for patients who need a transplant but do not have a matched donor. Cord blood that has been harvested from an umbilical cord shortly after birth has a rich supply of cells needed for transplant. These stored cord bloods are now being used to transplant adults without a matched donor
Advantages to using cord blood includes a readily available source of cells with no risk to the donor during the collection process, immediate source of cells in urgent situations (no lengthy donor work-up)and a reduction in infectious disease transmission to the recipient.
Read more
One of the main disadvantages is the cord blood has a small number of cells needed for transplant. In an adult, usually two cords are needed and large recipients do not qualify because they need too many cells.
This study will use two different preparative regimens (chemotherapy and radiation) followed by one or two umbilical cord units (UBC). The preparative regimen used will be chosen by the physician and is based on patient's age, disease and medical condition at the time of transplant.
Multiple objectives for this study include disease-free and overall survival, treatment related mortality, rate of cells taking hold, and the incidence and severity of the transplant complication called graft versus host disease (GVHD).
Read the detailed description
Allogeneic hematopoietic cell transplantation (allo- HCT) is a curative therapy for the treatment of hematological and non-hematological malignancies and certain non-malignant conditions. Bone marrow or peripheral blood from a Human Leukocyte Antigen (HLA) matched sibling donor is the most commonly used source of allogeneic stem cells. However, HLA matched siblings are available for less than one third of the patients who require allo-Stem Cell Transplant (allo-SCT). In the absence of an HLA matched sibling, volunteer unrelated donors or partially mismatched related donors (PMRD), stored cord blood may be used as a source of allogeneic stem cells. Stored cord blood has been used as a source of allogeneic stem cells in infants and children, but had early skepticism in adults because of concerns about the engraftment potential of the relatively limited number of stem cells. The number of stem cells in a unit of cord blood is generally one log less than the number of stem cells on an average collection of bone marrow from an adult for transplantation.
After the success of the first allogeneic umbilical cord blood transplantation in 1988, programs for banking screened unrelated donor CBSC have been initiated both in the United States and Europe. Dr Pablo Rubenstein started the first such bank at the New York Blood Center (NYBC) in 1993. Since its inception, the NYBC has provided unrelated donor cord blood stem cells for over 1000 transplants. Analysis of outcomes for the initial 562 transplant recipients from the NYBC revealed a cumulative rate of engraftment of 81% by day 42 for PMNs. and 85% by day 180 for platelets. Currently, approximately more than 100,000 cord blood units are available in cord blood banks worldwide and more than 2000 patients have received cord blood transplants from these banks. NetCord, an international cooperative group of cord blood banks, has developed a detailed set of standards for cord blood banking to facilitate international exchanges and to guarantee the quality of these products.
Cord Blood Unit Selection:
UCB units will be required to be a 4 to 6 of 6 HLA-A, -B antigen and -DRB1 allele match with the patient. Typing at HLA-C and -DQ will be obtained but not required in the match strategy. A minimum total nucleated cell (TNC) dose of >2.0 x 107/kg at the time of freezing will be utilized when possible. When using double units, each unit should contain a minimum pre-cryopreserved TNC dose of 1.5 x 107/kg.
UCB Transplant Procedure:
There will be a myeloablative and reduced-intensity preparative regimen that can be given prior to infusion of cord product. The myeloablative approach will be selected in younger patients (<50yo) with a HCT-CI score <3. The reduced-intensity regimen will be selected for all older patients (>50) or younger patients with a hematopoietic cell transplantation-specific comorbidity index (HCT-CI) score >3. The reduced-intensity regimen will also be chosen for any patients being transplanted for indolent/follicular lymphomas, CLL, myeloma, or Hodgkin lymphoma; irrelevant of age or HCT-CI score. On a case by case basis, patients may receive a preparative regimen outside of their designated category as noted above with the approval of the PI, if deemed in the patient's best interest.
- Conditions
- Chronic Myelogenous Leukemia (CML), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome, Multiple Myeloma, Hodgkin Lymphoma, Non-Hodgkin Lymphoma
- Being tested
- umbilical cord blood (UCB)
- Keywords
- Chronic myelogenous leukemia (CML), Acute myelogenous leukemia (AML), Myelodysplastic syndrome, Multiple myeloma, Hodgkin lymphoma, Non Hodgkin lymphoma, Chronic lymphocytic leukemia (CLL), Acute lymphocytic leukemia (ALL), Severe Aplastic Anemia, Cord Blood, Umbilical cord blood transplantation, UBC transplantation
- Medical subject headings
- Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Multiple Myeloma, Hodgkin Disease, Lymphoma, Non-Hodgkin, Leukemia, Lymphocytic, Chronic, B-Cell, Precursor Cell Lymphoblastic Leukemia-Lymphoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
umbilical cord blood (UCB)Genetic
Infusion will occur after preparative regimen in one or two UCB unit(s). If two products are used, they will be administered sequentially on the same day 1-6 hours apart. Tacrolimus and mycophenolate mofetil (MMF) will be used for GVHD prophylaxis.
| Study group | Role | What they receive |
|---|---|---|
| Transplant | Experimental | After a preparative regimen the patient will receive an infusion of one or two umbilical cord blood unit(s) (UBC). The UBC unit(s) will be thawed according to methods of Rubinstein et al. If two products are used, they will be administered sequentially on the same day 1-6 hours apart. Tacrolimus and mycophenolate mofetil (MMF) will be used for GVHD prophylaxis. On day +30, +60, +100, +180, and +365 the chimeric status of patients will be interpreted by variable number tandem repeat (VNTR) analysis. Immune reconstitution (Digeorge Panel) will also be checked at these time points. Receives: Genetic: umbilical cord blood (UCB) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Engraftment
Defined as neutrophil recovery associated with donor engraftment within the first 60 days of transplant
Measured: 60 days
Secondary outcomes (1)
Overall Survival at Day 180 Post-transplant
Number of Participants with Overall survival at day 180 transplant.
Measured: 180 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jan 2013 (estimated)
- Study start3 Feb 2009
- Primary completion14 May 2019
- Study completion6 Apr 2021
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 Chronic Myelogenous Leukemia (CML)Studies of the same condition, open and under way.
- More trials run by West Virginia 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
- 34 participants (actual)
Sponsor and investigators
- Lead sponsor
- West Virginia University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Michael Craig, MD, Principal investigator, West Virginia University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01768845
- Sponsor's protocol ID
- WVU 1909
- Status last verified
- Jun 2021
- First submitted
- 11 Jan 2013
- Last updated
- 14 Jun 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Unrelated Umbilical Cord Blood (UCB) Transplantation
- ClinicalTrials.gov ID
- NCT01768845
- Acronym / sponsor ID
- WVU 1909
- Conditions
- Chronic Myelogenous Leukemia (CML), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome, Multiple Myeloma, Hodgkin Lymphoma, Non-Hodgkin Lymphoma
- Keywords
- Chronic myelogenous leukemia (CML), Acute myelogenous leukemia (AML), Myelodysplastic syndrome, Multiple myeloma, Hodgkin lymphoma, Non Hodgkin lymphoma, Chronic lymphocytic leukemia (CLL), Acute lymphocytic leukemia (ALL)
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 34 participants (actual)
- Lead sponsor
- West Virginia UniversityUniversity, hospital or other
- Study start
- 3 Feb 2009 (actual)
- Primary completion
- 14 May 2019 (actual)
- Study completion
- 6 Apr 2021 (actual)
- First posted
- 15 Jan 2013 (estimated)
- Results
- Posted 8 Jun 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Hematopoietic progenitor cell (HPC- primitive cells in the blood, bone marrow and umbilical cord that can restore the bone marrow) transplant can be a curative therapy for the treatment of hematologic malignancies (a disease of the bone marrow and lymph nodes). The source of cells used for the transplant comes from related (sibling) and in cases where there is no sibling match, from unrelated donors through the National Marrow Donor Program. The availability of a suitable donor can be a significant obstacle for patients who need a transplant but do not have a matched donor. Cord blood that has been harvested from an umbilical cord shortly after birth has a rich supply of cells needed for transplant. These stored cord bloods are now being used to transplant adults without a matched donor
Advantages to using cord blood includes a readily available source of cells with no risk to the donor during the collection process, immediate source of cells in urgent situations (no lengthy donor work-up)and a reduction in infectious disease transmission to the recipient.
One of the main disadvantages is the cord blood has a small number of cells needed for transplant. In an adult, usually two cords are needed and large recipients do not qualify because they need too many cells.
This study will use two different preparative regimens (chemotherapy and radiation) followed by one or two umbilical cord units (UBC). The preparative regimen used will be chosen by the physician and is based on patient's age, disease and medical condition at the time of transplant.
Multiple objectives for this study include disease-free and overall survival, treatment related mortality, rate of cells taking hold, and the incidence and severity of the transplant complication called graft versus host disease (GVHD).
Detailed description
Allogeneic hematopoietic cell transplantation (allo- HCT) is a curative therapy for the treatment of hematological and non-hematological malignancies and certain non-malignant conditions. Bone marrow or peripheral blood from a Human Leukocyte Antigen (HLA) matched sibling donor is the most commonly used source of allogeneic stem cells. However, HLA matched siblings are available for less than one third of the patients who require allo-Stem Cell Transplant (allo-SCT). In the absence of an HLA matched sibling, volunteer unrelated donors or partially mismatched related donors (PMRD), stored cord blood may be used as a source of allogeneic stem cells. Stored cord blood has been used as a source of allogeneic stem cells in infants and children, but had early skepticism in adults because of concerns about the engraftment potential of the relatively limited number of stem cells. The number of stem cells in a unit of cord blood is generally one log less than the number of stem cells on an average collection of bone marrow from an adult for transplantation.
After the success of the first allogeneic umbilical cord blood transplantation in 1988, programs for banking screened unrelated donor CBSC have been initiated both in the United States and Europe. Dr Pablo Rubenstein started the first such bank at the New York Blood Center (NYBC) in 1993. Since its inception, the NYBC has provided unrelated donor cord blood stem cells for over 1000 transplants. Analysis of outcomes for the initial 562 transplant recipients from the NYBC revealed a cumulative rate of engraftment of 81% by day 42 for PMNs. and 85% by day 180 for platelets. Currently, approximately more than 100,000 cord blood units are available in cord blood banks worldwide and more than 2000 patients have received cord blood transplants from these banks. NetCord, an international cooperative group of cord blood banks, has developed a detailed set of standards for cord blood banking to facilitate international exchanges and to guarantee the quality of these products.
Cord Blood Unit Selection:
UCB units will be required to be a 4 to 6 of 6 HLA-A, -B antigen and -DRB1 allele match with the patient. Typing at HLA-C and -DQ will be obtained but not required in the match strategy. A minimum total nucleated cell (TNC) dose of >2.0 x 107/kg at the time of freezing will be utilized when possible. When using double units, each unit should contain a minimum pre-cryopreserved TNC dose of 1.5 x 107/kg.
UCB Transplant Procedure:
There will be a myeloablative and reduced-intensity preparative regimen that can be given prior to infusion of cord product. The myeloablative approach will be selected in younger patients (<50yo) with a HCT-CI score <3. The reduced-intensity regimen will be selected for all older patients (>50) or younger patients with a hematopoietic cell transplantation-specific comorbidity index (HCT-CI) score >3. The reduced-intensity regimen will also be chosen for any patients being transplanted for indolent/follicular lymphomas, CLL, myeloma, or Hodgkin lymphoma; irrelevant of age or HCT-CI score. On a case by case basis, patients may receive a preparative regimen outside of their designated category as noted above with the approval of the PI, if deemed in the patient's best interest.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Transplant | Experimental | Genetic: umbilical cord blood (UCB)After a preparative regimen the patient will receive an infusion of one or two umbilical cord blood unit(s) (UBC). The UBC unit(s) will be thawed according to methods of Rubinstein et al. If two products are used, they will be administered sequentially on the same day 1-6 hours apart. Tacrolimus and mycophenolate mofetil (MMF) will be used for GVHD prophylaxis. On day +30, +60, +100, +180, and +365 the chimeric status of patients will be interpreted by variable number tandem repeat (VNTR) analysis. Immune reconstitution (Digeorge Panel) will also be checked at these time points. |
Who may qualify
- Ages
- 16 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (21 listed)
- Age: 16-70 years
- Available 4/6, 5/6, or 6/6 HLA antigen match (using A, B, and DRB1) cord blood unit.
- Eastern Cooperative Oncology Group (ECOG) performance status of 0-2 (Karnofsky greater than or equal to 70%)
- Serum bilirubin less than 2 x upper limit of normal
- Serum creatinine less than 2 mg/dl
Key exclusion criteria (9 listed)
- Patient pregnant
- Age less than 16, greater than 70
- ECOG performance status of greater than 2 (Karnofsky less than 70%)
- Psychiatric disorder or mental deficiency of the patient sufficiently severe as to make compliance with the BMT treatment unlikely, or making informed consent impossible
- Serum bilirubin greater than or equal to 2 x upper limit of normal, transaminases greater than 3 x upper limit of normal
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 (UCB)Infusion will occur after preparative regimen in one or two UCB unit(s). If two products are used, they will be administered sequentially on the same day 1-6 hours apart. Tacrolimus and mycophenolate mofetil (MMF) will be used for GVHD prophylaxis. | Genetic | Transplant |
Primary outcome
Number of Participants With Engraftment
Time frame: 60 days
Defined as neutrophil recovery associated with donor engraftment within the first 60 days of transplant
1 secondary outcome
Overall Survival at Day 180 Post-transplant
Time frame: 180 days
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jun 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
Michael Craig, MD principal investigator
West Virginia University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing umbilical cord blood (UCB) in people with Chronic Myelogenous Leukemia (CML) and Acute Myelogenous Leukemia (AML), run by West Virginia University 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 21 inclusion and 9 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: finished, as of the sponsor's last update on 14 Jun 2021. 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?
Yes. Results are posted in the registry (first posted 8 Jun 2021). 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 Participants With Engraftment (time frame: 60 days). 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 14 Jun 2021.