ALLPhase 1/2NCT00814983
Myeloablative Allogeneic Stem Cell Transplantation Using a Naive T-Cell Depleted Peripheral Blood Stem Cell Graft
A Phase 1/2 study testing Naive T-cell Depleted Stem Cell Transplant, Stem Cell Transplant No Manipulation and Isolex device from Baxter in people with ALL and ANLL, run by Duke University.
Trial summary
The primary objectives will be to measure the safety and efficacy of allogeneic stem cell transplantation using a peripheral blood stem cell graft that has been depleted of CD45RA+ Naive T-cells. The secondary objectives will be to measure the pace of immune recovery. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- What is being tested?
- Naive T-cell Depleted Stem Cell Transplant, Stem Cell Transplant No Manipulation and Isolex device from Baxter
- Type of treatment
- Medical device, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- No sites listed yet
- Study dates
- Jul 2007to Sep 2013
- Phase
- Phase 1/2
- 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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age 18 to 65 years.
- 8/8 or 7/8 HLA-identical matched sibling OR Allele level 8/8 (HLA-A, B, C, DRbeta1) matched unrelated donor.
- Patients with high risk ALL in first complete remission, with high risk being defined by the presence of t(4;11), t(9;22) or t(1;19) or patients presenting with extreme hyperleukocytosis (WBC>500,000/ml) or partial remission after initial induction therapy.
- Adult patients with acute non-lymphocytic Leukemia (ANLL) in first complete remission with high-risk cytogenetics (monosomy chromosome 5 or 7, del(5q), abn(3q26), complex karyotypic abnormalities) or failure to achieve complete remission after standard induction therapy.
- All patients with ALL or ANLL in second or subsequent remission or partial remission (<5% blasts in bone marrow as measured by flow cytometry).
- All patients with CML in chronic (failed interferon and/or Gleevec) or accelerated phase.
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- Patients with myelodysplastic syndrome with International Prognostic Scoring System (IPSS) risk category of INT-1 or greater.
- Myelofibrosis with myeloid metaplasia
- Patients with severe aplastic anemia must have failed immunosuppressive therapy such as cyclosporine plus anti-thymocyte globulin.
- Patients with a history of CNS disease must have been treated and have no active CNS disease at the time of protocol treatment.
- ECOG performance status <2
- Patients must have adequate function of other organ systems as measured by:
- Creatinine clearance (by Cockcroft Gault equation [Appendix IV]) > 30ml/min. Hepatic transaminases (ALT/AST) < 4 x normal, bilirubin < 2.0 mg/dl.
- Pulmonary function tests demonstrating FVC and FEV1 of >50% of predicted for age and DLCO > 50% of predicted.
- Ejection fraction of >45% by echocardiogram, radionuclide scan or cardiac MRI.
- Patients must be HIV negative.
- Patients must not be pregnant.
Who cannot join
- Patients with > 5% blasts in bone marrow or peripheral circulation.
- Patients with rapidly progressive ANLL or ALL.
About this study
The primary objectives will be to measure the safety and efficacy of allogeneic stem cell transplantation using a peripheral blood stem cell graft that has been depleted of CD45RA+ Naive T-cells.
The secondary objectives will be to measure the pace of immune recovery.
Read the detailed description
A cohort of patients (Cohort 1) will be enrolled to receive the currently accepted standard approach to myeloablative allogeneic stem cell transplantation. With the exception of volume and/or plasma depletion (in cases of donor/recipient ABO incompatibility), the peripheral blood stem cell graft will be unmodified. The primary purpose of Cohort 1 is to prospectively collect samples for measurement of immune recovery from a relatively homogeneous population of patients treated in a uniform manner. Within the limitations of age-matching, patients accrued to Cohort 1 will be incorporated into a larger retrospective historical control group for purposes of comparison with Cohort 2 of the incidence of grade II-IV acute Graft versus Host disease. The experimental aspects of this trial will be the use of a naïve T-cell depleted peripheral blood stem cell graft (Cohort 2). All other aspects of this stem cell transplantation are in line with the standard of care. Recruitment to this trial will be stratified by donor type as matched sibling or matched unrelated donor. Patients will be conditioned with total body irradiation (1350cGy) and Cyclophosphamide. The donor stem cell grafts will come from mobilized peripheral blood of 6/6 HLA-identical family members or 8/8 (HLA A, B, C, DRB1) allele-level matched unrelated donors.
- Being tested
- Naive T-cell Depleted Stem Cell Transplant, Stem Cell Transplant No Manipulation, Isolex device from Baxter
- Keywords
- myeloablative, stem cell, naive T-cell depleted, allogeneic
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Naive T-cell Depleted Stem Cell TransplantProcedure or surgery
The Isolex device from Baxter will be used to perform the cell selection procedure. After the CD34 selected stem cell graft has been collected, the CD34- "flow-through"fraction will be depleted of CD45RA+ naive T-cells. To accomplish this, a second immunomagnetic bead selection process will be performed on the Isolex device, making use of a GMP-grade murine anti-human CE45RA antibody. This depleted fraction will comprise the donor lymphocyte inoculum given to the transplant recipient along with the stem cell component on day 0 of transplant.
Stem Cell Transplant No ManipulationProcedure or surgery
These patients will be transplanted with unmanipulated peripheral blood stem cells.
Isolex device from BaxterDevice
| Study group | Role | What they receive |
|---|---|---|
| Naive T-cell Depleted Stem Cell Transplant | Experimental | Experimental: Cohort 2 will receive a T-cell depleted peripheral blood stem cell graft. All other aspects of this stem cell transplantation are in line with the standard of care. Receives: Procedure: Naive T-cell Depleted Stem Cell Transplant; Device: Isolex device from Baxter |
| Stem Cell Transplant No Manipulation | Active comparator | Control: Cohort 1 Stem Cell Transplant No Manipulation will receive the currently accepted standard approach to myeloablative allogeneic stem cell transplantation Receives: Procedure: Stem Cell Transplant No Manipulation |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Incidence of Grade II-IV Acute Graft Versus Host Disease
Measured: One year from date of transplant
Disease Free Survival
Measured: One year
Secondary outcomes (1)
Rate of Immune Recovery
The time to recovery of T-cell subset, B cell and NK cell recovery will be monitored along with T-Cell proliferative response to mitogens.
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Dec 2008 (estimated)
- Study startJul 2007
- Primary completionSep 2013
- Study completionSep 2013
Why it stopped: IND approval for naive T-cell depletion not obtained
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 ALLStudies of the same condition, open and under way.
- More trials run by Duke 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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Duke University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Mitchell Horwitz, MD, Principal investigator, Duke Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00814983
- Sponsor's protocol ID
- Pro00000993
- Status last verified
- Sep 2013
- First submitted
- 26 Dec 2008
- Last updated
- 25 Nov 2013
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 25 Nov 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Myeloablative Allogeneic Stem Cell Transplantation Using a Naive T-Cell Depleted Peripheral Blood Stem Cell Graft
- ClinicalTrials.gov ID
- NCT00814983
- Acronym / sponsor ID
- Pro00000993
- Keywords
- myeloablative, stem cell, naive T-cell depleted, allogeneic
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: IND approval for naive T-cell depletion not obtained
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Duke UniversityUniversity, hospital or other
- Study start
- Jul 2007
- Primary completion
- Sep 2013 (actual)
- Study completion
- Sep 2013 (actual)
- First posted
- 29 Dec 2008 (estimated)
- Results
- Posted 25 Nov 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objectives will be to measure the safety and efficacy of allogeneic stem cell transplantation using a peripheral blood stem cell graft that has been depleted of CD45RA+ Naive T-cells.
The secondary objectives will be to measure the pace of immune recovery.
Detailed description
A cohort of patients (Cohort 1) will be enrolled to receive the currently accepted standard approach to myeloablative allogeneic stem cell transplantation. With the exception of volume and/or plasma depletion (in cases of donor/recipient ABO incompatibility), the peripheral blood stem cell graft will be unmodified. The primary purpose of Cohort 1 is to prospectively collect samples for measurement of immune recovery from a relatively homogeneous population of patients treated in a uniform manner. Within the limitations of age-matching, patients accrued to Cohort 1 will be incorporated into a larger retrospective historical control group for purposes of comparison with Cohort 2 of the incidence of grade II-IV acute Graft versus Host disease. The experimental aspects of this trial will be the use of a naïve T-cell depleted peripheral blood stem cell graft (Cohort 2). All other aspects of this stem cell transplantation are in line with the standard of care. Recruitment to this trial will be stratified by donor type as matched sibling or matched unrelated donor. Patients will be conditioned with total body irradiation (1350cGy) and Cyclophosphamide. The donor stem cell grafts will come from mobilized peripheral blood of 6/6 HLA-identical family members or 8/8 (HLA A, B, C, DRB1) allele-level matched unrelated donors.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Naive T-cell Depleted Stem Cell Transplant | Experimental | Procedure: Naive T-cell Depleted Stem Cell Transplant, Device: Isolex device from BaxterExperimental: Cohort 2 will receive a T-cell depleted peripheral blood stem cell graft. All other aspects of this stem cell transplantation are in line with the standard of care. |
| Stem Cell Transplant No Manipulation | Active comparator | Procedure: Stem Cell Transplant No ManipulationControl: Cohort 1 Stem Cell Transplant No Manipulation will receive the currently accepted standard approach to myeloablative allogeneic stem cell transplantation |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- Age 18 to 65 years.
- 8/8 or 7/8 HLA-identical matched sibling OR Allele level 8/8 (HLA-A, B, C, DRbeta1) matched unrelated donor.
- Patients with high risk ALL in first complete remission, with high risk being defined by the presence of t(4;11), t(9;22) or t(1;19) or patients presenting with extreme hyperleukocytosis (WBC>500,000/ml) or partial remission after initial induction therapy.
- Adult patients with acute non-lymphocytic Leukemia (ANLL) in first complete remission with high-risk cytogenetics (monosomy chromosome 5 or 7, del(5q), abn(3q26), complex karyotypic abnormalities) or failure to achieve complete remission after standard induction therapy.
- All patients with ALL or ANLL in second or subsequent remission or partial remission (<5% blasts in bone marrow as measured by flow cytometry).
Key exclusion criteria (2 listed)
- Patients with > 5% blasts in bone marrow or peripheral circulation.
- Patients with rapidly progressive ANLL or ALL.
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 |
|---|---|---|
| Naive T-cell Depleted Stem Cell TransplantThe Isolex device from Baxter will be used to perform the cell selection procedure. After the CD34 selected stem cell graft has been collected, the CD34- "flow-through"fraction will be depleted of CD45RA+ naive T-cells. To accomplish this, a second immunomagnetic bead selection process will be performed on the Isolex device, making use of a GMP-grade murine anti-human CE45RA antibody. This depleted fraction will comprise the donor lymphocyte inoculum given to the transplant recipient along with the stem cell component on day 0 of transplant. | Procedure or surgery | Naive T-cell Depleted Stem Cell Transplant |
| Stem Cell Transplant No ManipulationThese patients will be transplanted with unmanipulated peripheral blood stem cells. | Procedure or surgery | Stem Cell Transplant No Manipulation |
| Isolex device from Baxter | Device | Naive T-cell Depleted Stem Cell Transplant |
Primary outcome
The Incidence of Grade II-IV Acute Graft Versus Host Disease
Time frame: One year from date of transplant
Disease Free Survival
Time frame: One year
1 secondary outcome
Rate of Immune Recovery
Time frame: 3 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Nov 2013.
- 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.
Mitchell Horwitz, MD principal investigator
Duke Health
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Naive T-cell Depleted Stem Cell Transplant, Stem Cell Transplant No Manipulation and Isolex device from Baxter in people with ALL and ANLL, run by Duke University.
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 17 inclusion and 2 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 25 Nov 2013. Status can change between updates, so confirm with the study contact before making plans.
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 combined 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.
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 25 Nov 2013). 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?
The Incidence of Grade II-IV Acute Graft Versus Host Disease (time frame: One year from date of transplant). The study lists 2 primary outcomes in all. 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 25 Nov 2013; AzTrials last refreshed its copy on 29 Oct 2026.