CancerPhase 2NCT00597519
A Myeloablative Conditioning Regimen and Total Body Irradiation Followed by the Transplantation for Patients With Hematological Malignancy
A Phase 2 study testing Fludarabine, Cyclophosphamide and Transplantation in people with Cancer and Leukemia, run by Memorial Sloan Kettering Cancer Center at 1 site in United States.
Trial summary
In this study two cord blood collections will be used to increase the number of cord blood cells you will receive on transplant day. We call this a "double unit" cord blood transplant. A previous study suggests double unit cord blood transplant may have a better result. Read more about this study
- Who is it for?
- Ages 4 to 50 years
- Conditions the study is for
- Cancer, Leukemia, Myelodysplastic Syndrome
- What is being tested?
- Fludarabine, Cyclophosphamide and Transplantation
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 28 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 2006to Sep 2015
- 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 4 to 50 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age 4 - 50 years
- Patient should not have a related or unrelated volunteer donor that is suitably HLA matched and available in the required time period or be a suitable candidate for an autologous stem cell transplant.
- Patients will have one of the following hematological malignancies: Acute myelogenous leukemia (AML):
- Complete first remission (CR1) at high risk for relapse as defined by:
- known prior diagnosis of myelodysplasia (MDS); or therapy related AML; or White cell count at presentation > 100,000; or Presence of extramedullary leukemia at diagnosis; or Unfavorable FAB type (M0, M5-7); or High-risk cytogenetics (such as those associated with MDS, abnormalities of 5, 7, 8, Philadelphia chromosome, complex karyotype); or High risk molecular markers such as FLT3 mutations; or Requirement for 2 or more inductions to achieve CR1
- Complete second CR (CR2).
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- Acute lymphoblastic leukemia (ALL):
- Complete first remission (CR1) at high risk for relapse as defined by:
- White cell count at presentation as follows:
- > 100,000 if < 18 years
- > 50,000 if > 18 years; or
- Presence of extensive extra-medullary disease (excluding CNS disease); or Presence of high-risk cytogenetic abnormality such as t(9;22), t(1;19), t(4;11) or other MLL rearrangements (11q23), t(8;14) [excluding B-ALL in pediatric patients]; or
- Failed to achieve complete remission after four weeks of induction therapy Unable to receive required consolidation chemotherapy as would be needed to maintain remission
- Complete second or third remission (CR2 or CR3)
- Acute undifferentiated leukemia (AUL), infant leukemia, or biphenotypic leukemia in CR1, CR2 or CR3. Patients with infant leukemia must be eligible to receive total body irradiation.
- Juvenile Myelomonocytic leukemia (JMML) with < 30% bone marrow blasts.
- Chronic myelogenous leukemia (CML)
- GleevecTM failure in 1st or 2nd chronic phase;
- Gleevec failure in 1st or 2nd accelerated phase.
- Myelodysplastic Syndrome (MDS) with one of the following:
- Low (Score 0) International Prognostic Scoring System (IPSS) score with Life-threatening cytopenia(s); or Red cell or platelet transfusion dependent
- Intermediate (Score 1) or High (Score 2) International Prognostic Scoring System (IPSS) score.
- Patients with bone marrow blasts > 10% should have AML induction therapy with disease response to < 5% blasts and at least partial count recovery.
- Non-Hodgkin's Lymphoma
- Patients with high-grade disease following initial therapy and are not appropriate for further chemotherapy or autologous stem cell transplantation.
- Patients with high-grade or diffuse large cell NHL with recurrent disease after first remission and must have:
- Chemo-sensitivity as evidenced by > partial remission (PR) (defined as > 50% reduction in mass size after therapy).
- Patients with adequate organ function and performance status criteria as measured by:
- Karnofsky score > or = to 70 % or Lansky score > or = to 70%
- Renal: Calculated creatinine clearance > or = to 60 ml/min
- Hepatic: Total bilirubin < 2.5 mg/dL unless benign congenital hyperbilirubinemia (Gilbert's syndrome) and ALT/AST < 3 x upper limit of normal
- Albumin > or = to 2.5
- Pulmonary: Pulmonary function (spirometry and corrected DLCO) > or = to 60% normal if available (in small children use History and Physical and CT scan as necessary to determine pulmonary status)
- Cardiac: Left ventricular ejection fraction > or = to 50%
- Double Unit Umbilical Cord Blood Grafts:
- Units will be selected based on the HLA match to the patient and on the basis of the individual and combined cell doses of the units.
- Patient has a related or unrelated volunteer donor that is suitably HLA matched and available in the required time period.
- Patient is a candidate for an autologous stem cell transplant.
- Active CNS leukemia.
- Acute Myelogenous Leukemia in greater than CR2.
- Acute Myelogenous Leukemia evolved from myelofibrosis.
- Acute Lymphoblastic Leukemia, acute undifferentiated leukemia, biphenotypic leukemia or infant leukemia greater than CR3.
- Any acute leukemia with:
- Morphologic relapse or persistent disease in the BM (cytogenetic relapse without morphologic evidence of relapse or cytogenetic persistent disease in the BM is acceptable); or
- Active extra-medullary leukemia; or
- Requiring greater than 2 cycles of chemotherapy to obtain present remission status;
- Bone Marrow aplasia (defined as BM cellularity less than 5% at transplant work-up); or
- MDS with greater than 10% bone marrow blasts refractory to chemotherapy; or
- CML in blast crisis; or
- NHL refractory to chemotherapy (less than PR after 2 or more regimens); or
- Prior autologous or allogeneic HSC transplant at any time; or
- Prior radiation therapy rendering patient ineligible for TBI; or
- Uncontrolled viral, bacteria or fungal infection at time of study enrollment; or
- Seropositive or NAT positive for HIV; or
- Females who are pregnant or breast feeding; or
- Patient or guardian unable to give informed consent or unable to comply with the treatment protocol including appropriate supportive care, follow-up, and research tests.
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
United States1 site
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
About this study
Official title: A Myeloablative Conditioning Regimen Consisting of Cyclophosphamide, Fludarabine and Total Body Irradiation Followed by the Transplantation of Unrelated Donor Double Unit Umbilical Cord Blood Grafts for Patients With Hematological Malignancy.
In this study two cord blood collections will be used to increase the number of cord blood cells you will receive on transplant day. We call this a "double unit" cord blood transplant. A previous study suggests double unit cord blood transplant may have a better result. The main purpose of this study is to find out how good a cord blood transplant using two cord blood collections from two different babies is at curing you of your cancer. Double unit cord blood transplants are now being studied as a way to increase the number of cord blood cells given to bigger children and adult patients.
Based on studies that have already been done double unit cord blood transplant appears to be safer than if only one cord blood unit is used. However, double unit cord blood transplant is a fairly new form of treatment.
Read the detailed description
This is a single arm phase 2 study to obtain a preliminary estimate of efficacy of myeloablative double unit umbilical cord blood transplantation (UCBT) as measured by overall and disease-free survival at 1 year post transplantation. The UCB graft will consist of two (or double) units from two unrelated newborn donors. Patients with hematopoietic malignancy at high-risk for relapse or with advanced disease will receive myeloablative conditioning with cyclophosphamide (Cy),low dose fludarabine (Flu) and total body irradiation (TBI) with post transplantation cyclosporine (CSA) and mycophenolate mofetil (MMF) for GVHD prophylaxis.
- Conditions
- Cancer, Leukemia, Myelodysplastic Syndrome, Non-Hodgkin's Lymphoma
- Being tested
- Fludarabine, Cyclophosphamide, Transplantation
- Keywords
- Cancer, Leukemia, Myelodysplastic Syndrome, Non-Hodgkin's Lymphoma, Total Body Irradiation, Umbilical Cord Blood Grafts, 06-014
- Medical subject headings
- Neoplasms, Leukemia, Myelodysplastic Syndromes, Lymphoma, Non-Hodgkin
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Fludarabine, CyclophosphamideDrug
Fludarabine 25 mg/m2/day IV in the morning x 3 days (days -7, -6 and -5) for a total dose of 75 mg/m2 followed by Cyclophosphamide on days -6 and -5. 60mg/kg/day IV over 30-60 minutes x 2 days (days -6 and -5). High volume fluids should commence approximately 12 hours prior to drug and continue until 24 hours after second dose.
Total Body Irradiation: 125 cGy x 11 doses (TID on days -3, -2, -1 and BID on day 0) for a total TBI dose of 1375 cGy. Pediatric patients unable to tolerate a TID dosing schedule can receive 150 cGy x 8 doses (BID on days -3, -2, -1, and 0). All patients will receive GVHD prophylaxis with 2 drugs: Cyclosporine A and Mycophenolate mofetil (MMF).
Units should be given consecutively each over approximately 10-30 minutes.Pre-medication should include acetaminophen and diphenhydramine or hydroxyzine.G-CSF 5 mcg/kg/day IV/SQ (dose rounded to vial size to a maximum of 480 mcg) will be given from day +1 until ANC recovery.
TransplantationProcedure or surgery
The UCB ( Umbilical Cord Blood) collection known as a unit is processed to remove excess plasma and red cells, tested for sterility, HLA-typed, cryopreserved and stored. This protocol involves the administration of two UCB units from two different donors. The units will be thawed in the Cytotherapy Laboratory as per the current standard operating procedure.
| Study group | Role | What they receive |
|---|---|---|
| Treatment | Experimental | Patients with hematopoietic malignancy at high-risk for relapse or with advanced disease will receive myeloablative conditioning with cyclophosphamide (Cy), low dose fludarabine (Flu) and total body irradiation (TBI) with post transplantation cyclosporine (CSA) and mycophenolate mofetil (MMF) for GVHD prophylaxis. Receives: Drug: Fludarabine, Cyclophosphamide; Procedure: Transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Response
To obtain a preliminary estimate of efficacy of double unit UCBT as measured by overall response.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jan 2008 (estimated)
- Study startMar 2006
- Primary completionSep 2015
- Study completionSep 2015
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 CancerStudies of the same condition, open and under way.
- More trials run by Memorial Sloan Kettering Cancer CenterThe 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 28 participants (actual)
Sponsor and investigators
- Lead sponsor
- Memorial Sloan Kettering Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Juliet Barker, MBBS, Principal investigator, Memorial Sloan Kettering Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00597519
- Sponsor's protocol ID
- 06-014
- Other IDs
- CA23766
- Status last verified
- Jan 2016
- First submitted
- 26 Dec 2007
- Last updated
- 12 Feb 2016
- Results posted
- Yes, on ClinicalTrials.gov
Study at a glance
From the registry record, last updated by the sponsor on 12 Feb 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Myeloablative Conditioning Regimen Consisting of Cyclophosphamide, Fludarabine and Total Body Irradiation Followed by the Transplantation of Unrelated Donor Double Unit Umbilical Cord Blood Grafts for Patients With Hematological Malignancy.
- ClinicalTrials.gov ID
- NCT00597519
- Acronym / sponsor ID
- 06-014
- Conditions
- Cancer, Leukemia, Myelodysplastic Syndrome, Non-Hodgkin's Lymphoma
- Keywords
- Cancer, Leukemia, Myelodysplastic Syndrome, Non-Hodgkin's Lymphoma, Total Body Irradiation, Umbilical Cord Blood Grafts, 06-014
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 28 participants (actual)
- Lead sponsor
- Memorial Sloan Kettering Cancer CenterUniversity, hospital or other
- Study start
- Mar 2006
- Primary completion
- Sep 2015 (actual)
- Study completion
- Sep 2015 (actual)
- First posted
- 18 Jan 2008 (estimated)
- Results
- Posted 12 Feb 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
In this study two cord blood collections will be used to increase the number of cord blood cells you will receive on transplant day. We call this a "double unit" cord blood transplant. A previous study suggests double unit cord blood transplant may have a better result. The main purpose of this study is to find out how good a cord blood transplant using two cord blood collections from two different babies is at curing you of your cancer. Double unit cord blood transplants are now being studied as a way to increase the number of cord blood cells given to bigger children and adult patients.
Based on studies that have already been done double unit cord blood transplant appears to be safer than if only one cord blood unit is used. However, double unit cord blood transplant is a fairly new form of treatment.
Detailed description
This is a single arm phase 2 study to obtain a preliminary estimate of efficacy of myeloablative double unit umbilical cord blood transplantation (UCBT) as measured by overall and disease-free survival at 1 year post transplantation. The UCB graft will consist of two (or double) units from two unrelated newborn donors. Patients with hematopoietic malignancy at high-risk for relapse or with advanced disease will receive myeloablative conditioning with cyclophosphamide (Cy),low dose fludarabine (Flu) and total body irradiation (TBI) with post transplantation cyclosporine (CSA) and mycophenolate mofetil (MMF) for GVHD prophylaxis.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment | Experimental | Drug: Fludarabine, Cyclophosphamide, Procedure: TransplantationPatients with hematopoietic malignancy at high-risk for relapse or with advanced disease will receive myeloablative conditioning with cyclophosphamide (Cy), low dose fludarabine (Flu) and total body irradiation (TBI) with post transplantation cyclosporine (CSA) and mycophenolate mofetil (MMF) for GVHD prophylaxis. |
Who may qualify
- Ages
- 4 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (56 listed)
- Age 4 - 50 years
- Patient should not have a related or unrelated volunteer donor that is suitably HLA matched and available in the required time period or be a suitable candidate for an autologous stem cell transplant.
- Patients will have one of the following hematological malignancies: Acute myelogenous leukemia (AML):
- known prior diagnosis of myelodysplasia (MDS); or therapy related AML; or White cell count at presentation > 100,000; or Presence of extramedullary leukemia at diagnosis; or Unfavorable FAB type (M0, M5-7); or High-risk cytogenetics (such as those associated with MDS, abnormalities of 5, 7, 8, Philadelphia chromosome, complex karyotype); or High risk molecular markers such as FLT3 mutations; or Requirement for 2 or more inductions to achieve CR1
- Complete second CR (CR2).
Key exclusion criteria (0 listed)
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 |
|---|---|---|
| Fludarabine, CyclophosphamideFludarabine 25 mg/m2/day IV in the morning x 3 days (days -7, -6 and -5) for a total dose of 75 mg/m2 followed by Cyclophosphamide on days -6 and -5. 60mg/kg/day IV over 30-60 minutes x 2 days (days -6 and -5). High volume fluids should commence approximately 12 hours prior to drug and continue until 24 hours after second dose. Total Body Irradiation: 125 cGy x 11 doses (TID on days -3, -2, -1 and BID on day 0) for a total TBI dose of 1375 cGy. Pediatric patients unable to tolerate a TID dosing schedule can receive 150 cGy x 8 doses (BID on days -3, -2, -1, and 0). All patients will receive GVHD prophylaxis with 2 drugs: Cyclosporine A and Mycophenolate mofetil (MMF). Units should be given consecutively each over approximately 10-30 minutes.Pre-medication should include acetaminophen and diphenhydramine or hydroxyzine.G-CSF 5 mcg/kg/day IV/SQ (dose rounded to vial size to a maximum of 480 mcg) will be given from day +1 until ANC recovery. | Drug | Treatment |
| TransplantationThe UCB ( Umbilical Cord Blood) collection known as a unit is processed to remove excess plasma and red cells, tested for sterility, HLA-typed, cryopreserved and stored. This protocol involves the administration of two UCB units from two different donors. The units will be thawed in the Cytotherapy Laboratory as per the current standard operating procedure. | Procedure or surgery | Treatment |
Primary outcome
Overall Response
Time frame: 1 year
To obtain a preliminary estimate of efficacy of double unit UCBT as measured by overall response.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Feb 2016.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Memorial Sloan Kettering web siteLink provided by the sponsor.
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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.
Juliet Barker, MBBS principal investigator
Memorial Sloan Kettering Cancer 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 Fludarabine, Cyclophosphamide and Transplantation in people with Cancer and Leukemia, run by Memorial Sloan Kettering Cancer 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 56 inclusion and 0 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 12 Feb 2016. 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 12 Feb 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?
Overall Response (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 12 Feb 2016.