Leukemia, AcutePhase 1/2NCT01050764
Haploidentical Allogeneic Transplant With Post-transplant Infusion of Regulatory T-cells
A Phase 1/2 study testing Regulatory T-cells, Conventional T-cells and Melphalan in people with Leukemia, Acute and Chronic Myelogenous Leukemia (CML), run by Everett Meyer at 1 site in United States.
Trial summary
Patients with hematologic malignancies will receive myeloablative chemotherapy followed by stem cell rescue with bone marrow or hematopoietic peripheral blood stem cells collected by apheresis from a filgrastim- (G-CSF)-mobilized haploidentical related-donor, ie, hematopoietic peripheral blood stem cell transplant… Read more about this study
- Who is it for?
- Ages Up to 60 years
- Conditions the study is for
- Leukemia, Acute, Chronic Myelogenous Leukemia (CML), Myelodysplastic Syndrome (MDS)
- What is being tested?
- Regulatory T-cells, Conventional T-cells and Melphalan
- Type of treatment
- Injection or infusion, Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2009to Jun 2014
- 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 Up to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- RECIPIENT
- Histopathologically-confirmed:
- Acute leukemia (in first remission with poor risk factors and molecular prognosis)
- Acute myelogenous leukemia (AML) with -5,-7, t (6;9), tri8, -11
- Acute lymphoblastic leukemia (ALL) with Ph+ t (9;22), t (4;22), (q34;q11)
- Acute leukemia with refractory disease or > Complete Remission (CR) 1
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- Chronic myelogenous leukemia (CML) (accelerated, blast or second chronic phase)
- Myelodysplastic syndrome (in high and high intermediate risk categories)
- Non-Hodgkin's lymphoma (NHL) with poor risk features and not suitable for autologous transplantation
- Refractory Chronic lymphocytic leukemia (CLL)
- At least 21 days from the end of most recent prior therapy to start of the transplant conditioning regimen
- Must be < 60 years old at time of registration.
- Karnofsky Performance Status (KPS) > 70%
- Must have related donor who is:
- Genotypically human leukocyte antigen (HLA) -A, B,C and DR beta 1 (DRB1), DQ loci haploidentical to the recipient (but differing for 2 to 3 HLA alleles on the unshared haplotype in the graft-versus-host disease (GvHD) direction)
- No HLA-matched sibling or matched-unrelated donor is identified.
- Adequate cardiac and pulmonary function (left ventricular ejection fraction (LVEF) > 45%, diffusing capacity of the lungs for carbon monoxide (DLCO) >50% corrected for hemoglobin)
- Serum creatinine < 1.5 mg/dL OR Creatinine clearance > 50 mL/min for those above serum creatinine at least 1.5 mg/dL
- Serum bilirubin < 2.0 mg/dL
- Alanine transaminase (ALT) < 2x upper normal limit (ULN) (unless secondary to disease)
- No prior myeloablative therapy or hematopoietic cell transplantation
- DONOR:
- Age ≤ 70 years
- Weight ≥ 25 kg.
- Medical history and physical examination confirm good health status as defined by institutional standards
- Seronegative for HIV Ag within 30 days of apheresis collection for:
- Hepatitis B surface antigen (sAg) or polymerase chain reaction (PCR) +
- Hepatitis C ab or PCR+
- Genotypically haploidentical as determined by HLA typing
- Female donors (child-bearing potential) must have a negative serum or urine beta-human chorionic gonadotropin (HCG) test within 3 weeks of mobilization
- Capable of undergoing leukapheresis
- Has adequate venous access
- Willing to undergo insertion of a central catheter if leukapheresis via peripheral vein is inadequate
- Capable of agreeing to second donation of peripheral blood progenitor cell (PBPC) (or a bone marrow harvest) should the patient fail to demonstrate sustained engraftment following the transplant
- Institutional review board (IRB)-approved consent form signed by donor or legal guardian > 18 years of age
- Donor Selection in the priority order:
- Recipient's biological mother preferred, if available
- Other available haploidentical donors will be selected based upon the presence of natural killer (NK) alloreactivity between donor and recipient by high-resolution HLA typing of the C locus. An NK-alloreactive donor will be preferentially chosen. Recipients lacking a killer immunoglobulin-like receptor (KIR)-ligand present in the donor along with the corresponding KIR defines "NK alloreactivity".
- If more than one NK-alloreactive donor is available, preference is to cytomegalovirus (CMV)-seronegative donor
Who cannot join
- RECIPIENT:
- Suitable candidate for autologous transplantation or allogeneic transplantation with an available matched-related or matched-unrelated donor
- Seropositive for:
- HIV ab
- Hepatitis B sAg or PCR+
- Hepatitis C ab or PCR+
Show all 15Show fewer
- History of invasive Aspergillosis
- Any active, uncontrolled bacterial, viral or fungal infection
- Uncontrolled central nervous system (CNS) disease involvement
- Lactating female
- DONOR:
- Evidence of active infection or viral hepatitis
- Factors of increased risk for complications from leukapheresis or granulocyte-colony stimulating factor (G-CSF) therapy
- Lactating female
- HIV-positive
Trial locations and contacts
1 site in 1 country.
United States1 site
Stanford University School of Medicine
Stanford, California, 94305
About this study
Official title: A Feasibility Trial of Post-Transplant Infusion of Allogeneic Regulatory T Cells and Allogeneic Conventional T Cells in Patients With Hematologic Malignancies Undergoing Allogeneic Myeloablative Hematopoietic Cell Transplantation From Haploidentical-Related Donors
Patients with hematologic malignancies will receive myeloablative chemotherapy followed by stem cell rescue with bone marrow or hematopoietic peripheral blood stem cells collected by apheresis from a filgrastim- (G-CSF)-mobilized haploidentical related-donor, ie, hematopoietic peripheral blood stem cell transplant (HSCT).
Read the detailed description
This is dose-escalation study intended to evaluate the use of classification determinant 15-positive (CD15+), CD4+, CD127dim, and FoxP3+ regulatory T-cells (T-reg cells) supplemented by conventional T-cells (T-con cells), to enhance the efficacy of allogeneic (CliniMACS CD34+ selected) hematopoietic stem cell transplantation (allo-HSCT), in the setting of leukemia, lymphoma, and myelodysplastic syndrome (MDS). This study investigates amelioration of the impaired immune recovery and address the significant relapse incidence in the haploidentical HSCT setting.
Pre-transplant myeloablative conditioning will be melphalan; thiotepa; fludarabine and rabbit antithymocyte globulin (rATG).
Stem cell rescue will be with CD34+ selected cells. The rescue infusion will be supplemented with infusions of regulatory T-cells (T-reg) and conventional T-cells (T-con) from the same donor collection, on Treatment Days 14 and 16 respectively. CD34+ cell infusion day is Treatment Day 0.
T-reg cells are those cells enriched by immunomagnetic selection of CD25+ cells, and further purified by flow cytometric cell sorting for the CD15+, CD4+, CD127dim, FoxP3+ cell population. These cells are an enriched but naturally-occurring T-cell population.
T-con cells are unseparated/unfractionated cells, ie, as collected by the peripheral blood stem cells apheresis procedure.
Post-transplant follow-up is for 5 years.
- Conditions
- Leukemia, Acute, Chronic Myelogenous Leukemia (CML), Myelodysplastic Syndrome (MDS), Non-Hodgkin Lymphoma (NHL), Chronic Lymphocytic Leukemia (CLL), Acute Myelogenous Leukemia (AML)
- Being tested
- Regulatory T-cells, Conventional T-cells, Melphalan, Thiotepa, Fludarabine
- Medical subject headings
- Leukemia, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Myelodysplastic Syndromes, Lymphoma, Non-Hodgkin, Leukemia, Lymphocytic, Chronic, B-Cell, Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Regulatory T-cellsDrug
Also called T-reg cells
To ameliorate the impaired immune recovery and address the significant relapse incidence in the haploidentical setting. Cells will be selected by a tandem selection process and infused on day +14. These are the enriched but naturally-occurring regulatory T cells. Possible dose cohorts and levels are:
Cohort 1
- T-reg: 1 x 10e5/kg
- T-con: 3 x10e5/kg
Cohort 2
- T-reg: 3 x 10e5/kg
- T-con: 1 x 10e6/kg
Cohort 3
- T-reg: 1 x 10e6/kg
- T-con: 3 x 10e6/kg
Cohort 4
- T-reg: 3 x 10e6/kg
- T-con: 1 x 10e7/kg
Conventional T-cellsDrug
Also called T-con cells
These are conventional (unselected) donor T-cells. Cell dosage of the infusion will be based on the CD3+ cell content and infused on day +16.
MelphalanDrug
Also called L-PAM, L-Sarcolysin, Phenylalanine Mustard
Anti-cancer chemotherapy drug administered IV at 140 mg/m² on Day -8 prior to HSCT (a component of the conditioning regiment prior to infusion of cells)
ThiotepaDrug
Also called thiophosphamide, TESPA, TSPA
Anti-cancer chemotherapy drug administered IV at 10 mg/kg on Day -7 prior to HSCT (a component of the conditioning regiment prior to infusion of cells)
FludarabineDevice
Also called Fludarabine phosphate, Fludarabine 5'-monophosphate, FAMP, Fludara
Anti-cancer chemotherapy drug administered IV at 160mg/m² on Days -6; -5; -4; and -3 prior to HSCT (a component of the conditioning regiment prior to infusion of cells
Anti-thymocyte globulin, rabbitDrug
Also called Thymoglobulin, rATG
Rabbit-derived antibodies against human T-cells used as transplant rejection prophylaxis. Administered at 6 mg/kg IV on Days -6; -5; -4; and -3 prior to HSCT
CliniMACS CD34 Reagent SystemDrug
An in vitro medical device system that uses antibodies conjugated to magnetic beads to select and enrich for CD34+ blood stem cells from the allogeneic donor apheresis product prior to HSCT, while removing other cells that can cause GvHD. CD34+ cell dosage will be based on the participant's body weight.
| Study group | Role | What they receive |
|---|---|---|
| T-reg Cell Infusion after Allogeneic Stem Cell Transplant | Experimental | Not described Receives: Drug: Regulatory T-cells; Drug: Conventional T-cells; Drug: Melphalan; Drug: Thiotepa; Device: Fludarabine; Drug: Anti-thymocyte globulin, rabbit; Drug: CliniMACS CD34 Reagent System |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum-tolerated Dose (MTD) of Regulatory and Conventional T-cells
The maximum-tolerated dose (MTD) was to be determined based on the safety and feasibility observed for a pre-determined set of cellular dose level combinations of regulatory T-cells (T-reg) and conventional T-cells (T-con).
Measured: 30 days after HSCT infusion
Secondary outcomes (5)
Acute Graft-versus-Host-Disease (aGvHD)
The primary outcome was incidence of grade 3 or 4 acute graft-vs-host-disease (aGvHD), reported as the number of participants developing grade 3 or 4 aGvHD.
Measured: 1 year
Overall Survival (OS), 1 Year
Assessed as subjects remaining alive 12 months after CD34+ cell infusion (ie, excludes death due to any cause)
Measured: 1 year
Median Overall Survival (OS)
Reported as the median overall survival (OS) in months from infusion of the hematopoietic stem cells (HSCT)
Measured: 25 months
To Measure the Incidence and Severity of Acute and Chronic GvHD
Population of participants that received HSCT and T-reg plus T-con, and developed actue, chronic, or any graft vs host disease (GvHD)
Measured: 1 year
Serious Infections
Serious infections are reported as the number of participants experienced serious infections.
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 posted15 Jan 2010 (estimated)
- Study startJun 2009
- Primary completionAug 2012
- Study completionJun 2014
Why it stopped: Safety
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 Leukemia, AcuteStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Everett Meyer
- Sponsor type
- University, hospital or other
- Collaborators
- Doris Duke Charitable Foundation
- Responsible party
- Sponsor-investigator, Everett Meyer, Stanford University
- Investigators
- Everett H Meyer, MD, PhD, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01050764
- Sponsor's protocol ID
- IRB-15919
- Other IDs
- BMT204, SU-03312009-2059
- Status last verified
- Jun 2018
- First submitted
- 11 Jan 2010
- Last updated
- 27 Jun 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Jun 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Feasibility Trial of Post-Transplant Infusion of Allogeneic Regulatory T Cells and Allogeneic Conventional T Cells in Patients With Hematologic Malignancies Undergoing Allogeneic Myeloablative Hematopoietic Cell Transplantation From Haploidentical-Related Donors
- ClinicalTrials.gov ID
- NCT01050764
- Acronym / sponsor ID
- IRB-15919
- Conditions
- Leukemia, Acute, Chronic Myelogenous Leukemia (CML), Myelodysplastic Syndrome (MDS), Non-Hodgkin Lymphoma (NHL), Chronic Lymphocytic Leukemia (CLL), Acute Myelogenous Leukemia (AML)
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Safety
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Everett MeyerUniversity, hospital or other
- Collaborators
- Doris Duke Charitable Foundation
- Study start
- Jun 2009
- Primary completion
- Aug 2012 (actual)
- Study completion
- Jun 2014 (actual)
- First posted
- 15 Jan 2010 (estimated)
- Results
- Posted 11 May 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Patients with hematologic malignancies will receive myeloablative chemotherapy followed by stem cell rescue with bone marrow or hematopoietic peripheral blood stem cells collected by apheresis from a filgrastim- (G-CSF)-mobilized haploidentical related-donor, ie, hematopoietic peripheral blood stem cell transplant (HSCT).
Detailed description
This is dose-escalation study intended to evaluate the use of classification determinant 15-positive (CD15+), CD4+, CD127dim, and FoxP3+ regulatory T-cells (T-reg cells) supplemented by conventional T-cells (T-con cells), to enhance the efficacy of allogeneic (CliniMACS CD34+ selected) hematopoietic stem cell transplantation (allo-HSCT), in the setting of leukemia, lymphoma, and myelodysplastic syndrome (MDS). This study investigates amelioration of the impaired immune recovery and address the significant relapse incidence in the haploidentical HSCT setting.
Pre-transplant myeloablative conditioning will be melphalan; thiotepa; fludarabine and rabbit antithymocyte globulin (rATG).
Stem cell rescue will be with CD34+ selected cells. The rescue infusion will be supplemented with infusions of regulatory T-cells (T-reg) and conventional T-cells (T-con) from the same donor collection, on Treatment Days 14 and 16 respectively. CD34+ cell infusion day is Treatment Day 0.
T-reg cells are those cells enriched by immunomagnetic selection of CD25+ cells, and further purified by flow cytometric cell sorting for the CD15+, CD4+, CD127dim, FoxP3+ cell population. These cells are an enriched but naturally-occurring T-cell population.
T-con cells are unseparated/unfractionated cells, ie, as collected by the peripheral blood stem cells apheresis procedure.
Post-transplant follow-up is for 5 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| T-reg Cell Infusion after Allogeneic Stem Cell Transplant | Experimental | Drug: Regulatory T-cells, Drug: Conventional T-cells, Drug: Melphalan, Drug: Thiotepa, Device: Fludarabine, Drug: Anti-thymocyte globulin, rabbit, Drug: CliniMACS CD34 Reagent System |
Who may qualify
- Ages
- Up to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (39 listed)
- RECIPIENT
- Histopathologically-confirmed:
- Acute leukemia (in first remission with poor risk factors and molecular prognosis)
- Acute myelogenous leukemia (AML) with -5,-7, t (6;9), tri8, -11
- Acute lymphoblastic leukemia (ALL) with Ph+ t (9;22), t (4;22), (q34;q11)
Key exclusion criteria (15 listed)
- Suitable candidate for autologous transplantation or allogeneic transplantation with an available matched-related or matched-unrelated donor
- Seropositive for:
- HIV ab
- Hepatitis B sAg or PCR+
- Hepatitis C ab or PCR+
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 |
|---|---|---|
| Regulatory T-cellsAlso: T-reg cellsTo ameliorate the impaired immune recovery and address the significant relapse incidence in the haploidentical setting. Cells will be selected by a tandem selection process and infused on day +14. These are the enriched but naturally-occurring regulatory T cells. Possible dose cohorts and levels are: Cohort 1 * T-reg: 1 x 10e5/kg * T-con: 3 x10e5/kg Cohort 2 * T-reg: 3 x 10e5/kg * T-con: 1 x 10e6/kg Cohort 3 * T-reg: 1 x 10e6/kg * T-con: 3 x 10e6/kg Cohort 4 * T-reg: 3 x 10e6/kg * T-con: 1 x 10e7/kg | Drug | T-reg Cell Infusion after Allogeneic Stem Cell Transplant |
| Conventional T-cellsAlso: T-con cellsThese are conventional (unselected) donor T-cells. Cell dosage of the infusion will be based on the CD3+ cell content and infused on day +16. | Drug | T-reg Cell Infusion after Allogeneic Stem Cell Transplant |
| MelphalanAlso: L-PAM, L-Sarcolysin, Phenylalanine MustardAnti-cancer chemotherapy drug administered IV at 140 mg/m² on Day -8 prior to HSCT (a component of the conditioning regiment prior to infusion of cells) | Drug | T-reg Cell Infusion after Allogeneic Stem Cell Transplant |
| ThiotepaAlso: thiophosphamide, TESPA, TSPAAnti-cancer chemotherapy drug administered IV at 10 mg/kg on Day -7 prior to HSCT (a component of the conditioning regiment prior to infusion of cells) | Drug | T-reg Cell Infusion after Allogeneic Stem Cell Transplant |
| FludarabineAlso: Fludarabine phosphate, Fludarabine 5'-monophosphate, FAMP, FludaraAnti-cancer chemotherapy drug administered IV at 160mg/m² on Days -6; -5; -4; and -3 prior to HSCT (a component of the conditioning regiment prior to infusion of cells | Device | T-reg Cell Infusion after Allogeneic Stem Cell Transplant |
| Anti-thymocyte globulin, rabbitAlso: Thymoglobulin, rATGRabbit-derived antibodies against human T-cells used as transplant rejection prophylaxis. Administered at 6 mg/kg IV on Days -6; -5; -4; and -3 prior to HSCT | Drug | T-reg Cell Infusion after Allogeneic Stem Cell Transplant |
| CliniMACS CD34 Reagent SystemAn in vitro medical device system that uses antibodies conjugated to magnetic beads to select and enrich for CD34+ blood stem cells from the allogeneic donor apheresis product prior to HSCT, while removing other cells that can cause GvHD. CD34+ cell dosage will be based on the participant's body weight. | Drug | T-reg Cell Infusion after Allogeneic Stem Cell Transplant |
Primary outcome
Maximum-tolerated Dose (MTD) of Regulatory and Conventional T-cells
Time frame: 30 days after HSCT infusion
The maximum-tolerated dose (MTD) was to be determined based on the safety and feasibility observed for a pre-determined set of cellular dose level combinations of regulatory T-cells (T-reg) and conventional T-cells (T-con).
5 secondary outcomes
Acute Graft-versus-Host-Disease (aGvHD)
Time frame: 1 year
Overall Survival (OS), 1 Year
Time frame: 1 year
Median Overall Survival (OS)
Time frame: 25 months
To Measure the Incidence and Severity of Acute and Chronic GvHD
Time frame: 1 year
Serious Infections
Time frame: 1 year
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jun 2018.
- 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.
Everett H Meyer, MD, PhD principal investigator
Stanford University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Regulatory T-cells, Conventional T-cells and Melphalan in people with Leukemia, Acute and Chronic Myelogenous Leukemia (CML), run by Everett Meyer 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 39 inclusion and 15 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 27 Jun 2018. 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 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 11 May 2017). 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?
Maximum-tolerated Dose (MTD) of Regulatory and Conventional T-cells (time frame: 30 days after HSCT infusion). 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 27 Jun 2018.