Chronic Myeloproliferative DisordersNCT00255684
Fludarabine, Cyclophosphamide, and Total-Body Irradiation Followed by Cyclosporine and Mycophenolate Mofetil in Treating Patients Who Are Undergoing a Donor Umbilical Cord Blood Transplant for Hematologic Cancer
A study testing graft-versus-tumor induction therapy, cyclophosphamide and cyclosporine in people with Chronic Myeloproliferative Disorders and Graft Versus Host Disease, run by University of Rochester at 1 site in United States.
Trial summary
RATIONALE: Giving low doses of chemotherapy, such as fludarabine and cyclophosphamide, and radiation therapy before a donor umbilical cord blood stem cell transplant helps stop the growth of cancer cells. It also stops the patient's immune system from rejecting the donor's stem cells. Read more about this study
- Who is it for?
- Ages Up to 75 years
- Conditions the study is for
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia
- What is being tested?
- graft-versus-tumor induction therapy, cyclophosphamide and cyclosporine
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2003to Jun 2016
- 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 Up to 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
DISEASE CHARACTERISTICS:
- Diagnosis of 1 of the following hematologic malignancies:
- Acute myeloid leukemia (AML) with or without history of myelodysplastic syndromes, meeting 1 of the following criteria:
- In first complete remission (CR-1) with unfavorable cytogenetics and/or achieved CR-1 after ≥ 1 course of induction therapy
- Secondary or treatment-related AML
- In second or further complete remission
- Relapsed with ≤ 20% blasts in the bone marrow AND no circulating blasts
- Acute lymphoblastic leukemia (ALL), meeting 1 of the following criteria:
- In CR-1 with unfavorable cytogenetics or elevated WBC at presentation OR failed to achieve CR-1 after ≥ 4 weeks of induction therapy
- In second or further complete remission
- Relapsed with ≤ 20% blasts in the bone marrow AND no circulating blasts
- Other acute leukemic variants allowed at the discretion of the principal investigator
- Chronic myelogenous leukemia (CML), meeting 1 of the following criteria:
- In first chronic phase AND refractory to or unable to tolerate imatinib mesylate
- In second or further chronic phase
- In first or second accelerated phase
- Myelodysplastic syndromes with intermediate 2- or high-risk International Prognosis Scoring System (IPSS) score, including any of the following:
- Refractory anemia
- Refractory anemia with excess blasts
- Chronic myelomonocytic leukemia
- Myeloproliferative disorders with poor prognosis, including any of the following:
- Myelofibrosis with myeloid metaplasia
- No ≥ grade 3 myelofibrosis
- Atypical CML
- Juvenile myelomonocytic leukemia
- Other clonal hemopathies with an accepted poor prognosis
- Multiple myeloma with chromosome 13 abnormalities and/or progression after prior autologous bone marrow transplantation (BMT)
- Chronic lymphocytic leukemia, meeting 1 of the following criteria:
- Primary refractory OR relapsed and refractory disease (less than partial remission)
- Relapsed twice on or after prior chemotherapy
- Lymphoma, meeting both of the following criteria:
- Hodgkin's or non-Hodgkin's lymphoma in > CR-1 OR failed primary induction
- Chemosensitive disease, defined as > 50% reduction in mass size after the most recent chemotherapy
- Must meet ≥ 1 of the following criteria:
- Over 45 years of age
- Has undergone prior autologous or allogeneic BMT
- Charlson^ comorbidity score ≥ 2
- Must have a high degree of tumor control (salvage therapy allowed)
- At high risk for treatment-related mortality with a myeloablative conditioning regimen
- No massive splenomegaly
- Patients may become eligible after splenectomy or radiotherapy to the spleen
- No 5/6 or 6/6 HLA-matched related donor available
- No well-matched (i.e., ≥ 9/10 HLA match by high-resolution typing) unrelated donor available
PATIENT CHARACTERISTICS:
Performance status
- Not specified
Life expectancy
- Not specified
Hematopoietic
- See Disease Characteristics
Hepatic
- Bilirubin ≤ 2 times upper limit of normal (ULN)
- Transaminases ≤ 4 times ULN (unless due to underlying disease)
Renal
- Creatinine clearance ≥ 50 mL/min
Cardiovascular
- Ejection fraction ≥ 30%
Pulmonary
- DCLO ≥ 35%
Other
- Negative pregnancy test
- No uncontrolled viral, bacterial, or fungal infection
- HIV negative
PRIOR CONCURRENT THERAPY:
Biologic therapy
- See Disease Characteristics
Chemotherapy
- See Disease Characteristics
Radiotherapy
- See Disease Characteristics
Other
- At least 3 months since prior immunosuppressive therapy
- At least 10 days since prior salvage therapy for patients not in at least morphologic or radiologic complete remission
Trial locations and contacts
1 site in 1 country.
United States1 site
James P. Wilmot Cancer Center at University of Rochester Medical Center
Rochester, New York, 14642
About this study
Official title: Non-Myeloablative Conditioning and Unrelated Umbilical Cord Blood Transplantation for Children and Adults With Serious Oncohematologic Diseases
RATIONALE: Giving low doses of chemotherapy, such as fludarabine and cyclophosphamide, and radiation therapy before a donor umbilical cord blood stem cell transplant helps stop the growth of cancer cells. It also stops the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune system and help destroy any remaining cancer cells (graft-versus-tumor effect). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving cyclosporine and mycophenolate mofetil after transplant may stop this from happening.
PURPOSE: This clinical trial is studying how well giving fludarabine and cyclophosphamide together with total-body irradiation followed by cyclosporine and mycophenolate mofetil works in treating patients who are undergoing a donor umbilical cord blood transplant for hematologic cancer.
Read the detailed description
OBJECTIVES:
- Determine the frequency, extent, and rate of donor (myeloid and lymphoid) engraftment in patients with serious hematologic malignancies treated with nonmyeloablative conditioning regimen comprising fludarabine, cyclophosphamide, and low-dose total-body irradiation followed by unrelated allogeneic umbilical cord blood transplantation and post-transplant immunosuppression comprising cyclosporine and mycophenolate mofetil.
- Correlate clinical and umbilical cord blood-related factors with engraftment in patients treated with this regimen.
- Determine transplant-related complications, in terms of toxicity, myelosuppression, infections, and acute and chronic graft-versus-host disease, in patients treated with this regimen.
- Determine disease-free and overall survival of patients treated with this regimen.
- Determine treatment-related mortality of patients treated with this regimen.
OUTLINE: This is a uncontrolled, pilot study.
- Nonmyeloablative conditioning regimen: Patients receive fludarabine IV over 30 minutes daily on days -6 to -2 and cyclophosphamide IV over 2 hours on day -6 and undergo low-dose total-body irradiation (TBI) on day 0.
- Unrelated allogeneic umbilical cord blood transplantation (UCBT): After completion of TBI, patients undergo 1 or 2 unrelated allogeneic UCBTs on day 0.
- Post-transplant immunosuppression: Patients receive oral or IV cyclosporine daily beginning on day -3 and continuing until day 180 and oral or IV mycophenolate mofetil twice daily on days 0-30.
Patients are followed periodically for 1 year after transplantation.
PROJECTED ACCRUAL: A total of 20 patients will be accrued for this study.
- Conditions
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma, Multiple Myeloma, Myelodysplastic Syndromes
- Being tested
- graft-versus-tumor induction therapy, cyclophosphamide, cyclosporine, fludarabine phosphate, mycophenolate mofetil
- Keywords
- graft versus host disease, adult acute myeloid leukemia in remission, adult acute myeloid leukemia with 11q23 (MLL) abnormalities, adult acute myeloid leukemia with t(16;16)(p13;q22), adult acute myeloid leukemia with t(8;21)(q22;q22), recurrent adult acute myeloid leukemia, childhood acute myeloid leukemia in remission, recurrent childhood acute myeloid leukemia, adult acute lymphoblastic leukemia in remission, recurrent adult acute lymphoblastic leukemia, childhood acute lymphoblastic leukemia in remission, recurrent childhood acute lymphoblastic leukemia, acute undifferentiated leukemia, accelerated phase chronic myelogenous leukemia, chronic phase chronic myelogenous leukemia, childhood chronic myelogenous leukemia, refractory anemia with excess blasts, refractory anemia, chronic myelomonocytic leukemia, atypical chronic myeloid leukemia, BCR-ABL1 negative
- Medical subject headings
- Myeloproliferative Disorders, Graft vs Host Disease, Leukemia, Lymphoma, Multiple Myeloma, Myelodysplastic Syndromes, Myelodysplastic-Myeloproliferative Diseases, Congenital Abnormalities
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
graft-versus-tumor induction therapyBiological
cyclophosphamideDrug
cyclosporineDrug
umbilical cord blood transplantationProcedure or surgery
radiation therapyRadiation
| Study group | Role | What they receive |
|---|---|---|
| Conditioning therapy followed by TBI | Experimental | Fludarabine, Cyclophosphamide; Total-Body Irradiation Followed by Cyclosporine and Mycophenolate Mofetil Receives: Biological: graft-versus-tumor induction therapy; Drug: cyclophosphamide; Drug: cyclosporine; Drug: fludarabine phosphate; Drug: mycophenolate mofetil; Procedure: umbilical cord blood transplantation; Radiation: radiation therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Who Survived 100 Days or Longer
Measured: 100 days
Secondary outcomes (1)
Number of Participants Who Developed Acute Graft Versus Host Disease
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Nov 2005 (estimated)
- Study startDec 2003
- Primary completionMar 2013
- Study completionJun 2016
Why it stopped: low 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 Chronic Myeloproliferative DisordersStudies of the same condition, open and under way.
- More trials run by University of RochesterThe 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
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Rochester
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Gordon L. Phillips, MD, Principal investigator, James P. Wilmot Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00255684
- Sponsor's protocol ID
- CDR0000448637
- Other IDs
- URCC-U19403, URCC-RSRB-10063
- Status last verified
- Sep 2016
- First submitted
- 18 Nov 2005
- Last updated
- 3 Nov 2016
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 3 Nov 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Non-Myeloablative Conditioning and Unrelated Umbilical Cord Blood Transplantation for Children and Adults With Serious Oncohematologic Diseases
- ClinicalTrials.gov ID
- NCT00255684
- Acronym / sponsor ID
- CDR0000448637
- Conditions
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma, Multiple Myeloma, Myelodysplastic Syndromes
- Keywords
- graft versus host disease, adult acute myeloid leukemia in remission, adult acute myeloid leukemia with 11q23 (MLL) abnormalities, adult acute myeloid leukemia with t(16;16)(p13;q22), adult acute myeloid leukemia with t(8;21)(q22;q22), recurrent adult acute myeloid leukemia, childhood acute myeloid leukemia in remission, recurrent childhood acute myeloid leukemia
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: low accrual
- Enrolment
- 16 participants (actual)
- Lead sponsor
- University of RochesterUniversity, hospital or other
- Study start
- Dec 2003
- Primary completion
- Mar 2013 (actual)
- Study completion
- Jun 2016 (actual)
- First posted
- 21 Nov 2005 (estimated)
- Results
- Posted 27 Jun 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
RATIONALE: Giving low doses of chemotherapy, such as fludarabine and cyclophosphamide, and radiation therapy before a donor umbilical cord blood stem cell transplant helps stop the growth of cancer cells. It also stops the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune system and help destroy any remaining cancer cells (graft-versus-tumor effect). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving cyclosporine and mycophenolate mofetil after transplant may stop this from happening.
PURPOSE: This clinical trial is studying how well giving fludarabine and cyclophosphamide together with total-body irradiation followed by cyclosporine and mycophenolate mofetil works in treating patients who are undergoing a donor umbilical cord blood transplant for hematologic cancer.
Detailed description
OBJECTIVES:
- Determine the frequency, extent, and rate of donor (myeloid and lymphoid) engraftment in patients with serious hematologic malignancies treated with nonmyeloablative conditioning regimen comprising fludarabine, cyclophosphamide, and low-dose total-body irradiation followed by unrelated allogeneic umbilical cord blood transplantation and post-transplant immunosuppression comprising cyclosporine and mycophenolate mofetil.
- Correlate clinical and umbilical cord blood-related factors with engraftment in patients treated with this regimen.
- Determine transplant-related complications, in terms of toxicity, myelosuppression, infections, and acute and chronic graft-versus-host disease, in patients treated with this regimen.
- Determine disease-free and overall survival of patients treated with this regimen.
- Determine treatment-related mortality of patients treated with this regimen.
OUTLINE: This is a uncontrolled, pilot study.
- Nonmyeloablative conditioning regimen: Patients receive fludarabine IV over 30 minutes daily on days -6 to -2 and cyclophosphamide IV over 2 hours on day -6 and undergo low-dose total-body irradiation (TBI) on day 0.
- Unrelated allogeneic umbilical cord blood transplantation (UCBT): After completion of TBI, patients undergo 1 or 2 unrelated allogeneic UCBTs on day 0.
- Post-transplant immunosuppression: Patients receive oral or IV cyclosporine daily beginning on day -3 and continuing until day 180 and oral or IV mycophenolate mofetil twice daily on days 0-30.
Patients are followed periodically for 1 year after transplantation.
PROJECTED ACCRUAL: A total of 20 patients will be accrued for this study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Conditioning therapy followed by TBI | Experimental | Biological: graft-versus-tumor induction therapy, Drug: cyclophosphamide, Drug: cyclosporine, Drug: fludarabine phosphate, Drug: mycophenolate mofetil, Procedure: umbilical cord blood transplantation, Radiation: radiation therapyFludarabine, Cyclophosphamide; Total-Body Irradiation Followed by Cyclosporine and Mycophenolate Mofetil |
Who may qualify
- Ages
- Up to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
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 |
|---|---|---|
| graft-versus-tumor induction therapy | Biological | Conditioning therapy followed by TBI |
| cyclophosphamide | Drug | Conditioning therapy followed by TBI |
| cyclosporine | Drug | Conditioning therapy followed by TBI |
| fludarabine phosphate | Drug | Conditioning therapy followed by TBI |
| mycophenolate mofetil | Drug | Conditioning therapy followed by TBI |
| umbilical cord blood transplantation | Procedure or surgery | Conditioning therapy followed by TBI |
| radiation therapy | Radiation | Conditioning therapy followed by TBI |
Primary outcome
Number of Participants Who Survived 100 Days or Longer
Time frame: 100 days
1 secondary outcome
Number of Participants Who Developed Acute Graft Versus Host Disease
Time frame: 3 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Nov 2016.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Gordon L. Phillips, MD principal investigator
James P. Wilmot Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing graft-versus-tumor induction therapy, cyclophosphamide and cyclosporine in people with Chronic Myeloproliferative Disorders and Graft Versus Host Disease, run by University of Rochester 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?
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 3 Nov 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.
Are results available for this study?
Yes. Results are posted in the registry (first posted 27 Jun 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 Who Survived 100 Days or Longer (time frame: 100 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 3 Nov 2016.