LeukemiaPhase 2NCT00676806
A Phase II Study of Umbilical Cord Blood Transplantation
A Phase 2 study testing Umbilical Cord Blood After Myeloablative Conditioning and Umbilical Cord Blood After Reduced-Intensity Conditioning in people with Leukemia and Lymphoma, run by Tufts Medical Center at 1 site in United States.
Trial summary
This study is designed to determine whether Umbilical Cord Transplantation (UCB) can be substituted for adult bone marrow cells in the standard stem cell transplant regimens used at this hospital for subjects who do not have stem cell donors. Read more about this study
- Who is it for?
- Ages Up to 70 years
- Conditions the study is for
- Leukemia, Lymphoma, Multiple Myeloma
- What is being tested?
- Umbilical Cord Blood After Myeloablative Conditioning and Umbilical Cord Blood After Reduced-Intensity Conditioning
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2005to Jul 2014
- Phase
- Phase 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 Up to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients meeting eligibility criteria for unrelated allogeneic stem cell transplantation may be considered for participation on this clinical trial if and only if they meet each of the following criteria:
- Patients must have one of the following diagnoses
- Relapsed or refractory hematologic malignancy, or
- High risk hematologic malignancy in first remission, or
- Refractory acquired marrow failure state, or
- Inherited disorder of metabolism or marrow failure state without alternative curative therapy.
Show all 26Show fewer
- Patients must not have a 6/6 or 5/6 HLA-matched related donor.
- Patients must not have a HLA-A, -B and -DRB1 high resolution matched unrelated donor following registry search, or cannot (in the opinion of the treating physician) wait the median 3 months to receive a MUD unit.
- Patients must demonstrate an ability to understand and willingness to sign the informed consent document
- Patients considered for myeloablative conditioning must satisfy the following additional criteria:
- Patients must be up to age 55 (inclusive)
- Patients must have serum direct bilirubin ≤ 2.0 mg/dl and transaminases ≤ 2x institution upper limit of normal
- Patients must have serum creatinine ≤ 2 mg/dl with creatinine clearance ≥ 60 ml/min (either calculated or measured).
- Patients must have MUGA scan or echocardiogram normal for the institution, but not less than 45% left ventricular ejection fraction and no clinical evidence of cardiac dysfunction.
- Patients must have an ECOG performance status of 0 or 1 (see Appendix C).
- Patients must have adequate pulmonary function when corrected for hemoglobin and alveolar volume as evidenced by a diffusion capacity and FEV1 > 50% of predicted.
- Patients considered for reduced-intensity conditioning must satisfy the following additional criteria:
- Patients must not be candidates for myeloablative conditioning due to any one of the following: Prior myeloablative stem cell transplantation, Age > 50, Co morbid illness
- In opinion of treating physician, unable to comply with or withstand rigors of myeloablative conditioning
- Patients with leukemia must have circulating and bone marrow blast counts < 5%, all other patients must have chemotherapy responsive disease
- Patients must be between the ages of 18 and 70 (inclusive)
- Patients must have serum direct bilirubin ≤ 2.0 mg/dL and transaminases ≤ 3x institution upper limit of normal
- Patients must have creatinine clearance ≥ 30 ml/min (either calculated or measured).
- Patients must have MUGA scan or echocardiogram documenting left ventricular ejection fraction of no less than 35% and no clinical evidence of cardiac dysfunction.
- Patients must have an ECOG performance status of 0 or 1 (see Appendix C).
- Patients must have adequate pulmonary function when corrected for hemoglobin and alveolar volume as evidenced by a diffusion capacity and FEV1 ≥ 40% of predicted.
Who cannot join
- Patients are ineligible for participation on this trial if they meet any of the following criteria:
- Patients with a history of myocardial infarction within the preceding 6 months, significant arrhythmia within the preceding 3 months, uncontrolled hypertension or congestive heart failure are ineligible.
- Patients with unstable angina are not eligible.
- Pregnant or lactating women are ineligible.
- Male and female patients who do not agree to practice approved methods of birth control for the duration of the study are ineligible.
- Patients with uncontrolled infection are ineligible.
- Patients who are HIV positive or have evidence of chronic viral hepatitis are ineligible.
- Patients unable to comply with requirements for compliance with therapeutic plan and/or scheduled evaluations
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase II Study of Umbilical Cord Blood Transplantation Following Myeloablative or Reduced-Intensity Conditioning
This study is designed to determine whether Umbilical Cord Transplantation (UCB) can be substituted for adult bone marrow cells in the standard stem cell transplant regimens used at this hospital for subjects who do not have stem cell donors.
Read the detailed description
Allogeneic stem cell transplantation (SCT) following myeloablative and non-myeloablative conditioning therapy has proven curative treatment for a number of inherited and acquired hematologic disorders. The success of allogeneic transplantation is largely determined by compatibility between donor and recipient, which predicts the risk of fatal graft-versus-host disease (GVHD). Unfortunately, less than one third of patients needing an allogeneic transplant have an available compatible donor in their family. Registries have been established to match patients with compatible volunteer (unrelated) donors, but many patients, and in particular minority patients, still lack stem cell donors.
Umbilical cord blood (UCB) is a rich source of hematopoietic stem cells, which is readily available from the placenta following childbirth. Blood banks have been established in the United States and abroad to collect, process and store UCB for use in allogeneic transplantation. To date, more than 2000 UCB transplants have been performed in adults and children around the world.
Rationale for use of Umbilical Cord Blood in Transplantation
UCB has a number of proven and theoretical advantages as an alternative source of hematopoietic stem cells for transplantation:
- Placental or umbilical cord blood is an abundantly available source of stem cells, which is currently discarded and can be harvested at no risk to the mother or infant.
- Important infectious agents, particularly CMV, are much less common in the newborn than adults, and are less likely to contaminate UCB collections.
- UCB collections, typed, cryopreserved and banked, are available on demand, eliminating delays and uncertainties that now complicate marrow collection from unrelated donors. At present, UCB can be delivered for infusion within days of the initiation of a search. This compares with a median of 3 months from search to delivery of stem cells through the registries of volunteer adult donors.
- The intensity of graft-versus-host reactivity of fetal lymphocytes appears to be less than that of adult cells and consequently fetal lymphocytes are more tolerant of HLA incompatibility. Published studies have shown that transplantation of UCB matched at 4-5/6 antigens results in a comparable incidence of GVHD to transplantation of unrelated stem cells fully matched at 6/6 antigens.
- Frozen UCB can be easily shipped, stored at the treating institution, and thawed for use when needed, compared to freshly donated stem cells which have a limited shelf-life of one day or less, necessitating coordination between harvesting surgeons, transportation, and transplantation teams.
This research study has been designed for people who have been diagnosed with a blood tumor, which has not responded to treatment or has recurred, a bone marrow failure state such as aplastic anemia, or one of certain inherited metabolic disorders; and whose doctor feels the best treatment is an allogeneic stem cell transplant (alloSCT) but a related or unrelated adult donor is not available. Instead, a single unit of umbilical cord blood (UCB) will be used as the source of the subject's immune system. This study is designed to determine whether a single unit of UCB can be substituted for adult bone marrow cells in the standard stem cell transplant regimens used at this hospital for subjects who do not have stem cell donors.
- Conditions
- Leukemia, Lymphoma, Multiple Myeloma, Aplastic Anemia
- Being tested
- Umbilical Cord Blood After Myeloablative Conditioning, Umbilical Cord Blood After Reduced-Intensity Conditioning
- Keywords
- Umbilical cord blood transplantation, full ablation conditioning, reduced intensity conditioning
- Medical subject headings
- Leukemia, Lymphoma, Multiple Myeloma, Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Umbilical Cord Blood After Myeloablative ConditioningBiological
Also called Cytoxan, TBI, Fludara, Busulfex
cyclophosphamide (60mg/m2 days -6 \& -5), fludarabine (25 mg/m2 days -7, -6, \& -5) and total body irradiation (days -3, -2, \& -1, total 1200 cGy) followed by cord blood infusion on day 0.
Umbilical Cord Blood After Reduced-Intensity ConditioningBiological
Also called Psoralen, Cytoxan, Nypent, TBI
Extracorporeal Photopheresis (days -8 \& -7), cyclophosphamide 50 mg/kg (day -6) pentostatin 4 mg/kg/d (continuous infusion days -5 \& -4), total body irradiation (days -3 \& -2, total 600cGy) followed by Umbilical Cord Blood Infusion day 0.
| Study group | Role | What they receive |
|---|---|---|
| Myeloablative conditioning | Experimental | Umbilical cord blood for hematopoietic rescue following myeloablative conditioning Receives: Biological: Umbilical Cord Blood After Myeloablative Conditioning |
| Reduced intensity conditioning | Experimental | Umbilical cord blood for hematopoietic rescue following non-myeloablative conditioning Receives: Biological: Umbilical Cord Blood After Reduced-Intensity Conditioning |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Neutrophil Engraftment
Number of participants with neutrophil engraftment receiving umbilical cord blood for hematopoietic rescue following myeloablative or non-myeloablative conditioning
Measured: +45 and 90 days
Secondary outcomes (5)
Proportion of Subjects With Platelet Engraftment
Proportion of patients engrafting by days +45, +90, and +180.
Measured: +45, 90, and 180 days
Incidence of Acute GVHD
Measured: Day +100
Infectious Complications in UCB Recipients.
Measured: Day +100
Incidence of Chronic GVHD
Measured: After Day +100
Compare Rates of Complications Between Patients Receiving Ablative vs. Non-myeloablative Conditioning Prior to UCB Transplantation
Composite endpoint of GVH or infection. Too few events to compare between arms.
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 posted13 May 2008 (estimated)
- Study startJul 2005
- Primary completionJul 2013
- Study completionJul 2014
Why it stopped: Slow accrual
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.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Tufts Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Andreas Klein, MD, Principal investigator, Tufts Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00676806
- Sponsor's protocol ID
- UCBT001
- Status last verified
- May 2017
- First submitted
- 9 May 2008
- Last updated
- 1 Jun 2017
- 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 1 Jun 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study of Umbilical Cord Blood Transplantation Following Myeloablative or Reduced-Intensity Conditioning
- ClinicalTrials.gov ID
- NCT00676806
- Acronym / sponsor ID
- UCBT001
- Conditions
- Leukemia, Lymphoma, Multiple Myeloma, Aplastic Anemia
- Keywords
- Umbilical cord blood transplantation, full ablation conditioning, reduced intensity conditioning
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Slow accrual
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Tufts Medical CenterUniversity, hospital or other
- Study start
- Jul 2005
- Primary completion
- Jul 2013 (actual)
- Study completion
- Jul 2014 (actual)
- First posted
- 13 May 2008 (estimated)
- Results
- Posted 22 Jul 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is designed to determine whether Umbilical Cord Transplantation (UCB) can be substituted for adult bone marrow cells in the standard stem cell transplant regimens used at this hospital for subjects who do not have stem cell donors.
Detailed description
Allogeneic stem cell transplantation (SCT) following myeloablative and non-myeloablative conditioning therapy has proven curative treatment for a number of inherited and acquired hematologic disorders. The success of allogeneic transplantation is largely determined by compatibility between donor and recipient, which predicts the risk of fatal graft-versus-host disease (GVHD). Unfortunately, less than one third of patients needing an allogeneic transplant have an available compatible donor in their family. Registries have been established to match patients with compatible volunteer (unrelated) donors, but many patients, and in particular minority patients, still lack stem cell donors.
Umbilical cord blood (UCB) is a rich source of hematopoietic stem cells, which is readily available from the placenta following childbirth. Blood banks have been established in the United States and abroad to collect, process and store UCB for use in allogeneic transplantation. To date, more than 2000 UCB transplants have been performed in adults and children around the world.
Rationale for use of Umbilical Cord Blood in Transplantation
UCB has a number of proven and theoretical advantages as an alternative source of hematopoietic stem cells for transplantation:
- Placental or umbilical cord blood is an abundantly available source of stem cells, which is currently discarded and can be harvested at no risk to the mother or infant.
- Important infectious agents, particularly CMV, are much less common in the newborn than adults, and are less likely to contaminate UCB collections.
- UCB collections, typed, cryopreserved and banked, are available on demand, eliminating delays and uncertainties that now complicate marrow collection from unrelated donors. At present, UCB can be delivered for infusion within days of the initiation of a search. This compares with a median of 3 months from search to delivery of stem cells through the registries of volunteer adult donors.
- The intensity of graft-versus-host reactivity of fetal lymphocytes appears to be less than that of adult cells and consequently fetal lymphocytes are more tolerant of HLA incompatibility. Published studies have shown that transplantation of UCB matched at 4-5/6 antigens results in a comparable incidence of GVHD to transplantation of unrelated stem cells fully matched at 6/6 antigens.
- Frozen UCB can be easily shipped, stored at the treating institution, and thawed for use when needed, compared to freshly donated stem cells which have a limited shelf-life of one day or less, necessitating coordination between harvesting surgeons, transportation, and transplantation teams.
This research study has been designed for people who have been diagnosed with a blood tumor, which has not responded to treatment or has recurred, a bone marrow failure state such as aplastic anemia, or one of certain inherited metabolic disorders; and whose doctor feels the best treatment is an allogeneic stem cell transplant (alloSCT) but a related or unrelated adult donor is not available. Instead, a single unit of umbilical cord blood (UCB) will be used as the source of the subject's immune system. This study is designed to determine whether a single unit of UCB can be substituted for adult bone marrow cells in the standard stem cell transplant regimens used at this hospital for subjects who do not have stem cell donors.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Myeloablative conditioning | Experimental | Biological: Umbilical Cord Blood After Myeloablative ConditioningUmbilical cord blood for hematopoietic rescue following myeloablative conditioning |
| Reduced intensity conditioning | Experimental | Biological: Umbilical Cord Blood After Reduced-Intensity ConditioningUmbilical cord blood for hematopoietic rescue following non-myeloablative conditioning |
Who may qualify
- Ages
- Up to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (26 listed)
- Patients meeting eligibility criteria for unrelated allogeneic stem cell transplantation may be considered for participation on this clinical trial if and only if they meet each of the following criteria:
- Patients must have one of the following diagnoses
- Relapsed or refractory hematologic malignancy, or
- High risk hematologic malignancy in first remission, or
- Refractory acquired marrow failure state, or
Key exclusion criteria (8 listed)
- Patients are ineligible for participation on this trial if they meet any of the following criteria:
- Patients with a history of myocardial infarction within the preceding 6 months, significant arrhythmia within the preceding 3 months, uncontrolled hypertension or congestive heart failure are ineligible.
- Patients with unstable angina are not eligible.
- Pregnant or lactating women are ineligible.
- Male and female patients who do not agree to practice approved methods of birth control for the duration of the study are ineligible.
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 After Myeloablative ConditioningAlso: Cytoxan, TBI, Fludara, Busulfexcyclophosphamide (60mg/m2 days -6 \& -5), fludarabine (25 mg/m2 days -7, -6, \& -5) and total body irradiation (days -3, -2, \& -1, total 1200 cGy) followed by cord blood infusion on day 0. | Biological | Myeloablative conditioning |
| Umbilical Cord Blood After Reduced-Intensity ConditioningAlso: Psoralen, Cytoxan, Nypent, TBIExtracorporeal Photopheresis (days -8 \& -7), cyclophosphamide 50 mg/kg (day -6) pentostatin 4 mg/kg/d (continuous infusion days -5 \& -4), total body irradiation (days -3 \& -2, total 600cGy) followed by Umbilical Cord Blood Infusion day 0. | Biological | Reduced intensity conditioning |
Primary outcome
Number of Participants With Neutrophil Engraftment
Time frame: +45 and 90 days
Number of participants with neutrophil engraftment receiving umbilical cord blood for hematopoietic rescue following myeloablative or non-myeloablative conditioning
5 secondary outcomes
Proportion of Subjects With Platelet Engraftment
Time frame: +45, 90, and 180 days
Incidence of Acute GVHD
Time frame: Day +100
Infectious Complications in UCB Recipients.
Time frame: Day +100
Incidence of Chronic GVHD
Time frame: After Day +100
Compare Rates of Complications Between Patients Receiving Ablative vs. Non-myeloablative Conditioning Prior to UCB Transplantation
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 1 Jun 2017.
- 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.
Andreas Klein, MD principal investigator
Tufts Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Umbilical Cord Blood After Myeloablative Conditioning and Umbilical Cord Blood After Reduced-Intensity Conditioning in people with Leukemia and Lymphoma, run by Tufts Medical Center 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 26 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 1 Jun 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 phase mean?
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 22 Jul 2016). 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 Neutrophil Engraftment (time frame: +45 and 90 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 1 Jun 2017.