LeukemiaPhase 1NCT00304018
Donor Umbilical Cord Blood Transplant in Treating Patients With Advanced Hematologic Cancer
A Phase 1 study testing anti-thymocyte globulin, sargramostim and busulfan in people with Leukemia and Lymphoma, run by University of California, San Francisco at 1 site in United States.
Trial summary
RATIONALE: Giving chemotherapy, such as fludarabine, busulfan, and etoposide, before a donor umbilical cord blood stem cell transplant helps stop the growth of cancer cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells… Read more about this study
- Who is it for?
- Ages 18 to 55 years
- Conditions the study is for
- Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- What is being tested?
- anti-thymocyte globulin, sargramostim and busulfan
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- Oct 2002to Mar 2009
- Phase
- Phase 1
- 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 55 years
- All sexes
- No healthy volunteers
- Adult
DISEASE CHARACTERISTICS:
- Diagnosis of 1 of the following advanced hematologic malignancies:
- Acute myeloid leukemia (AML) meeting the following criteria:
- Not expected to be curable with chemotherapy and meets ≥ 1 of the following criteria:
- High-risk cytogenetics (-7, -7q, -5, -5q, t[6,9], t[9,11], complex, Philadelphia chromosome positive [Ph+])
- AML evolved from prior myelodysplasia
- AML secondary to prior chemotherapy
- Failed to achieve remission
- In second or subsequent remission
- Marrow blasts ≤ 10% (may be achieved using chemotherapy)
- Myelodysplastic syndromes (MDS) with high-risk features
- International Prognostic Scoring System (IPSS) score intermediate -2 or high-risk
- Marrow blasts ≤ 20% (may be achieved using chemotherapy)
- Acute lymphoblastic leukemia meeting the following criteria:
- Not expected to be curable with chemotherapy and meets ≥ 1 of the following criteria:
- High-risk cytogenetics (Ph+, t[4,11], 11q23 abnormalities, and monosomy 7)
- Required > 1 induction course to achieve remission
- Failed to achieve remission
- In second or subsequent remission
- Marrow blasts ≤ 10% (may be achieved using chemotherapy)
- Chronic myelogenous leukemia meeting ≥ 1 of the following criteria:
- Accelerated phase
- Chronic phase refractory to imatinib mesylate
- Blastic phase
- Marrow blasts ≤ 10% (may be achieved using chemotherapy)
- Multiple myeloma meeting 1 of the following criteria:
- Stage II or III disease with > first relapse or refractory disease
- Newly diagnosed disease with chromosome 13 abnormalities
- Lymphoma meeting the following criteria:
- One of the following subtypes:
- Diffuse large cell lymphoma
- Mantle cell lymphoma
- Peripheral T-cell lymphoma
- T-natural killer (NK) cell lymphoma
- Hodgkin's lymphoma
- Disease failed to respond to primary therapy, progressed, or recurred after prior therapy
- Patients who have failed autologous stem cell transplantation are eligible provided it has been > 1 year since transplant
- No rapid progression of malignant disease
- Not eligible for autologous stem cell transplantation
- Available umbilical cord blood (1-3 units) donor matching at ≥ 4 of 6 HLA antigens (A, B, and DR)
- Patients with an HLA-identical or 1 antigen-mismatched related donor OR a potential HLA-matched unrelated donor matching at > 6/8 (A, B, C, DR) alleles are not eligible
PATIENT CHARACTERISTICS:
- ECOG performance status 0-2
- Creatinine < 2.0 mg/dL
- Creatinine clearance > 40 mL/min
- Bilirubin < 2.0 mg/dL
- AST and alkaline phosphatase < 3 times upper limit of normal
- Hepatitis C and active hepatitis B allowed if patient has ≤ grade 2 inflammation or fibrosis by liver biopsy
- Ejection fraction > 40% by echocardiogram or MUGA
- DLCO > 40% of predicted
- Not pregnant or nursing
- Negative pregnancy test
- No known HIV infection
- No active infection requiring ongoing antibiotic treatment
PRIOR CONCURRENT THERAPY:
- See Disease Characteristics
Trial locations and contacts
1 site in 1 country.
United States1 site
UCSF Helen Diller Family Comprehensive Cancer Center
San Francisco, California, 94115
About this study
Official title: Pilot Study of Umbilical Cord Blood Transplantation in Adult Patients With Advanced Hematopoietic Malignancies
RATIONALE: Giving chemotherapy, such as fludarabine, busulfan, and etoposide, before a donor umbilical cord blood stem cell transplant helps stop the growth of cancer cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving antithymocyte globulin before transplant and tacrolimus and prednisone after transplant may stop this from happening.
PURPOSE: This phase I trial is studying how well donor umbilical cord blood transplant works in treating patients with advanced hematologic cancer.
Read the detailed description
OBJECTIVES:
Primary
- Determine the safety and feasibility of performing donor umbilical cord blood transplantation (UCBT) in patients with advanced hematologic malignancies, in terms of > 80% engraftment rate at day 100 post-transplant and ≤ 50% transplant-related mortality.
Secondary
- Determine the toxicity of a myeloablative preparative regimen comprising busulfan, fludarabine, and etoposide prior to UCBT in these patients.
- Determine the neutrophil and platelet recovery in patients treated with this regimen.
- Determine the event-free and overall survival of patients treated with this regimen.
- Evaluate lineage-specific chimerism after UCBT and assess the contribution of each individual cord blood unit to post-transplantation hematopoiesis in these patients.
- Determine the incidence, severity, and timing of acute and chronic graft-vs-host disease in patients treated with this regimen.
OUTLINE: This is a pilot study.
- Preparative regimen: Patients receive fludarabine IV over 30 minutes on days -7 to -3, busulfan IV over 2 hours 4 times daily on days -7 and -4, etoposide IV over 4 hours on day -3, and anti-thymocyte globulin IV over 6 hours on days -2 and -1.
- Donor umbilical cord blood transplantation (UCBT): Patients undergo donor UCBT on day 0. Beginning on day 7, patients receive sargramostim (GM-CSF) IV or subcutaneously once daily until blood counts recover.
- Graft-vs-host disease prophylaxis: Patients receive tacrolimus IV continuously over 24 hours or orally twice daily beginning on day -2 and continuing until day 180 followed by a taper. Patients also receive oral prednisone twice daily on days 13-50 and then once daily on days 50-60, followed by a rapid taper.
After completion of study treatment, patients are followed periodically for approximately 2 years.
PROJECTED ACCRUAL: A total of 10 patients will be accrued for this study.
- Being tested
- anti-thymocyte globulin, sargramostim, busulfan, etoposide, fludarabine phosphate
- Keywords
- adult acute myeloid leukemia with 11q23 (MLL) abnormalities, recurrent adult acute myeloid leukemia, secondary acute myeloid leukemia, adult acute myeloid leukemia in remission, adult acute erythroid leukemia (M6), adult acute megakaryoblastic leukemia (M7), adult acute monoblastic leukemia (M5a), adult acute monocytic leukemia (M5b), refractory multiple myeloma, adult acute lymphoblastic leukemia in remission, accelerated phase chronic myelogenous leukemia, blastic phase chronic myelogenous leukemia, relapsing chronic myelogenous leukemia, chronic phase chronic myelogenous leukemia, stage II multiple myeloma, stage III multiple myeloma, recurrent adult acute lymphoblastic leukemia, recurrent adult diffuse large cell lymphoma, stage III adult diffuse large cell lymphoma, recurrent mantle cell lymphoma
- Medical subject headings
- Leukemia, Lymphoma, Multiple Myeloma, Neoplasms, Plasma Cell, Myelodysplastic Syndromes, Congenital Abnormalities, Leukemia, Myeloid, Acute, Leukemia, Erythroblastic, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
anti-thymocyte globulinBiological
sargramostimBiological
busulfanDrug
etoposideDrug
prednisoneDrug
tacrolimusDrug
allogeneic hematopoietic stem cell transplantationProcedure or surgery
umbilical cord blood transplantationProcedure or surgery
| Study group | Role | What they receive |
|---|---|---|
| cord blood transplant | Experimental | Not described Receives: Biological: anti-thymocyte globulin; Biological: sargramostim; Drug: busulfan; Drug: etoposide; Drug: fludarabine phosphate; Drug: prednisone; Drug: tacrolimus; Procedure: allogeneic hematopoietic stem cell transplantation; Procedure: umbilical cord blood transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Determine the safety and feasibility of performing donor umbilical cord blood transplantation (UCBT) in patients with advanced hematologic malignancies
Determine the safety and feasibility of performing donor umbilical cord blood transplantation (UCBT) in patients with advanced hematologic malignancies, in terms of > 80% engraftment rate at day 100 post-transplant and ≤ 50% transplant-related mortality.
Measured: up to 24 months post-transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Mar 2006 (estimated)
- Study startOct 2002
- Primary completionMar 2009
- Study completionMar 2009
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for 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
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of California, San Francisco
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Thomas G. Martin, MD, Study chair, University of California, San Francisco
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00304018
- Sponsor's protocol ID
- CDR0000463370
- Other IDs
- UCSF-02253, UCSF-H24045-21269-04, UCSF-2207
- Status last verified
- Aug 2013
- First submitted
- 15 Mar 2006
- Last updated
- 14 Aug 2013
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Aug 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study of Umbilical Cord Blood Transplantation in Adult Patients With Advanced Hematopoietic Malignancies
- ClinicalTrials.gov ID
- NCT00304018
- Acronym / sponsor ID
- CDR0000463370
- Keywords
- adult acute myeloid leukemia with 11q23 (MLL) abnormalities, recurrent adult acute myeloid leukemia, secondary acute myeloid leukemia, adult acute myeloid leukemia in remission, adult acute erythroid leukemia (M6), adult acute megakaryoblastic leukemia (M7), adult acute monoblastic leukemia (M5a), adult acute monocytic leukemia (M5b)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- University of California, San FranciscoUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- Oct 2002
- Primary completion
- Mar 2009 (actual)
- Study completion
- Mar 2009 (actual)
- First posted
- 17 Mar 2006 (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.
RATIONALE: Giving chemotherapy, such as fludarabine, busulfan, and etoposide, before a donor umbilical cord blood stem cell transplant helps stop the growth of cancer cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving antithymocyte globulin before transplant and tacrolimus and prednisone after transplant may stop this from happening.
PURPOSE: This phase I trial is studying how well donor umbilical cord blood transplant works in treating patients with advanced hematologic cancer.
Detailed description
OBJECTIVES:
Primary
- Determine the safety and feasibility of performing donor umbilical cord blood transplantation (UCBT) in patients with advanced hematologic malignancies, in terms of > 80% engraftment rate at day 100 post-transplant and ≤ 50% transplant-related mortality.
Secondary
- Determine the toxicity of a myeloablative preparative regimen comprising busulfan, fludarabine, and etoposide prior to UCBT in these patients.
- Determine the neutrophil and platelet recovery in patients treated with this regimen.
- Determine the event-free and overall survival of patients treated with this regimen.
- Evaluate lineage-specific chimerism after UCBT and assess the contribution of each individual cord blood unit to post-transplantation hematopoiesis in these patients.
- Determine the incidence, severity, and timing of acute and chronic graft-vs-host disease in patients treated with this regimen.
OUTLINE: This is a pilot study.
- Preparative regimen: Patients receive fludarabine IV over 30 minutes on days -7 to -3, busulfan IV over 2 hours 4 times daily on days -7 and -4, etoposide IV over 4 hours on day -3, and anti-thymocyte globulin IV over 6 hours on days -2 and -1.
- Donor umbilical cord blood transplantation (UCBT): Patients undergo donor UCBT on day 0. Beginning on day 7, patients receive sargramostim (GM-CSF) IV or subcutaneously once daily until blood counts recover.
- Graft-vs-host disease prophylaxis: Patients receive tacrolimus IV continuously over 24 hours or orally twice daily beginning on day -2 and continuing until day 180 followed by a taper. Patients also receive oral prednisone twice daily on days 13-50 and then once daily on days 50-60, followed by a rapid taper.
After completion of study treatment, patients are followed periodically for approximately 2 years.
PROJECTED ACCRUAL: A total of 10 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 |
|---|---|---|
| cord blood transplant | Experimental | Biological: anti-thymocyte globulin, Biological: sargramostim, Drug: busulfan, Drug: etoposide, Drug: fludarabine phosphate, Drug: prednisone, Drug: tacrolimus, Procedure: allogeneic hematopoietic stem cell transplantation, Procedure: umbilical cord blood transplantation |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- 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 |
|---|---|---|
| anti-thymocyte globulin | Biological | cord blood transplant |
| sargramostim | Biological | cord blood transplant |
| busulfan | Drug | cord blood transplant |
| etoposide | Drug | cord blood transplant |
| fludarabine phosphate | Drug | cord blood transplant |
| prednisone | Drug | cord blood transplant |
| tacrolimus | Drug | cord blood transplant |
| allogeneic hematopoietic stem cell transplantation | Procedure or surgery | cord blood transplant |
| umbilical cord blood transplantation | Procedure or surgery | cord blood transplant |
Primary outcome
Determine the safety and feasibility of performing donor umbilical cord blood transplantation (UCBT) in patients with advanced hematologic malignancies
Time frame: up to 24 months post-transplant
Determine the safety and feasibility of performing donor umbilical cord blood transplantation (UCBT) in patients with advanced hematologic malignancies, in terms of > 80% engraftment rate at day 100 post-transplant and ≤ 50% transplant-related mortality.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 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, MD study chair
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?
A Phase 1 study testing anti-thymocyte globulin, sargramostim and busulfan in people with Leukemia and Lymphoma, 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?
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 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.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Determine the safety and feasibility of performing donor umbilical cord blood transplantation (UCBT) in patients with advanced hematologic malignancies (time frame: up to 24 months post-transplant). 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 Aug 2013.