Acute Myeloid LeukemiaPhase 3NCT03480360
Haploidentical Allogeneic Peripheral Blood Transplantation: Examining Checkpoint Immune Regulators' Expression
A Phase 3 study testing Cyclophosphamide, Fludarabine and Total Body Irradiation in people with Acute Myeloid Leukemia and Chronic Lymphocytic Leukemia, run by Dartmouth-Hitchcock Medical Center at 1 site in United States.
Trial summary
The standard Johns Hopkins' regimen will be used in study subjects, with the use of donor peripheral blood stem cells, rather than marrow. Clinical outcomes will be defined while focusing efforts on immune reconstitution focusing on immune checkpoint regulators after a related haploidentical stem cell transplant. Read more about this study
- Who is it for?
- Ages 18 to 75 yearsHealthy volunteers can take part
- Conditions the study is for
- Acute Myeloid Leukemia, Chronic Lymphocytic Leukemia, Chronic Myeloid Leukemia
- What is being tested?
- Cyclophosphamide, Fludarabine and Total Body Irradiation
- Type of treatment
- Pill or oral medicine, Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 1 siteUnited States
- Study dates
- 28 Mar 2018to 17 Oct 2025
- Phase
- Phase 3
- 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 75 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Age: less than 75 years
- The patient must be approved for transplant by the treating transplant physician. This includes completion of their pre-transplant workup, as directed by standard Dartmouth-Hitchcock Medical Center (DHMC) Standard Operating Procedure (SOP) (DHMC SOP - Pre-transplant Evaluation of allogeneic recipient (Appendix).
- The patient must have a disease (listed below) with treatment-responsiveness that the treating transplant physician believes will benefit from an allogeneic stem cell transplant. The diseases include:
- Acute leukemia - Acute Myeloid Leukemia, Acute Lymphocytic Leukemia
- Chronic leukemia - Chronic Myeloid Leukemia, Chronic Lymphocytic Leukemia
- Myelodysplasia
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- Myeloproliferative disorder
- Myelofibrosis
- Lymphoma - Non-Hodgkin's Lymphoma or Hodgkin's disease
- Plasma cell disorder, including myeloma, Waldenstrom's Macroglobulinemia
- Donor availability- the patient must have an identified RELATED haplo-identical donor
- No Human Immunodeficiency Virus infection or active hepatitis B or C
- Eastern Cooperative Oncology Group performance status: 0-2
- Diffusing capacity of carbon monoxide (DLCO) greater than or equal to 40 % predicted
- Left ventricular ejection fraction greater than or equal to 40%
- Serum bilirubin < 2x upper limit of normal; transaminases < 3x normal at the time of transplant
- No active or uncontrollable infection
- In female, a negative pregnancy test if experiencing menstrual periods
- No major organ dysfunction precluding transplantation
- No evidence of an active malignancy that would limit the patient's survival to less than 2 years. (If there is any question, the PI can make a decision).
Who cannot join
- Psychiatric disorder or a mental deficiency of the patient that is sufficiently severe to make compliance with the treatment unlikely, and making informed consent impossible.
- Major anticipated illness or organ failure incompatible with survival from bone marrow transplant.
- History of refractory systemic infection
- DONOR ELIGIBILITY
- Human leukocyte antigen (HLA) haplo-identical matched related.
- The donor must be healthy and must be willing to serve as a donor, based on standard National Marrow Donor Program (NMDP) guidelines and DHMC SOP - Donor Evaluation (Appendix)
Show all 15Show fewer
- The donor must have no significant co-morbidities that would put the donor at marked increased risk
- There is no age restriction for the donor
- Informed consent must be signed by donor
- DONOR EXCLUSION CRITERIA
- The NMDP guidelines for exclusion criteria will be used (Appendix). In addition, the following donors are NOT eligible:
- Pregnant or lactating donor
- HIV or active Hep B or C in the donor
- Donor unfit to receive G-CSF and undergo apheresis
- A donor with a psychiatric disorder or mental deficiency that makes compliance with the procedure unlikely and informed consent impossible
Trial locations and contacts
1 site in 1 country.
United States1 site
Dartmouth Hitchcock Medical Center, Norris Cotton Cancer Center
Lebanon, New Hampshire, 03756
About this study
Official title: Haploidentical Allogeneic Peripheral Blood Transplantation: Clinical Trial and Laboratory Correlates Examining Checkpoint Immune Regulators' Expression
The standard Johns Hopkins' regimen will be used in study subjects, with the use of donor peripheral blood stem cells, rather than marrow. Clinical outcomes will be defined while focusing efforts on immune reconstitution focusing on immune checkpoint regulators after a related haploidentical stem cell transplant.
Read the detailed description
We propose a clinical trial to define clinical endpoints, including engraftment, 100-day survival and one year survival (Objective #1). We will characterize the incidence, prevalence and function of immune checkpoint regulators in patients' blood and bone marrow following transplantation (Objective #2). We will correlate these laboratory results with clinical outcomes and the incidence of GVHD. As an exploratory aim, in those patients experiencing GVHD and requiring treatment, we will define the frequency/expression of checkpoint regulator expression and correlate these results with the patient's response to GVHD therapy.
- Conditions
- Acute Myeloid Leukemia, Chronic Lymphocytic Leukemia, Chronic Myeloid Leukemia, Myelodysplasia, Myeloproliferative Disorder, Myelofibrosis
- Being tested
- Cyclophosphamide, Fludarabine, Total Body Irradiation, Tacrolimus, cellcept
- Keywords
- haploidentical, transplant, peripheral blood, allogeneic
- Medical subject headings
- Leukemia, Myeloid, Acute, Leukemia, Lymphocytic, Chronic, B-Cell, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Anemia, Refractory, with Excess of Blasts, Myeloproliferative Disorders, Primary Myelofibrosis, Lymphoma, Lymphoma, Non-Hodgkin
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CyclophosphamideDrug
14.5 mg/kg for 2 days (days -6, -5) and then 50 mg/kg for two days (days 3, 4)
FludarabineDrug
30 mg/m2 daily for 5 days
Total Body IrradiationRadiation
200 centigray (cGy) for one day (day -1)
TacrolimusDrug
1 mg IV daily, (or the oral equivalent) adjusted to achieve a level between 5 and 15 ng/ml. If there is no evidence of GVHD, discontinue Tacrolimus by Day 180.
cellceptDrug
dose at 15 mg/kg po three times per day (maximum dose of 3 grams/day). Stop Cellcept at Day 35 following transplantation.
g-csfDrug
5 mcg/kg/d starting day 5 and continue until Absolute Neutrophil Count (ANC) > 1000/mcL for 3 days.
Peripheral Blood TransplantProcedure or surgery
cell dose goal: < 5 x 106 Hematopoietic progenitor cell antigen CD34+ cells/kg recipient weight
| Study group | Role | What they receive |
|---|---|---|
| Johns Hopkins' conditioning regimen | Other | Cyclophosphamide, fludarabine, total body irradiation, immune suppression including tacrolimus and cellcept, Granulocyte colony-stimulating factor (G-CSF), and peripheral blood transplant Receives: Drug: Cyclophosphamide; Drug: Fludarabine; Radiation: Total Body Irradiation; Drug: Tacrolimus; Drug: cellcept; Drug: g-csf; Procedure: Peripheral Blood Transplant |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Who Survived to 100-Days Post-transplant
Define 100-day survival of subjects
Measured: 100 days post date of peripheral blood transplant
Number of Participants Who Survived to One Year Post-Transplant.
Define one year survival of subjects
Measured: One year post date of peripheral blood transplant
Number of Participants Who Experienced a Successful Engraftment
Define number of subjects who experience a successful engraftment: Defined as absolute neutrophil count > 500/mm3 and platelets > 20,000/mcl for three consecutive days (count first day as engraftment)
Measured: Post-peripheral blood transplant
Number of Participants Who Achieved a Response to Treatment at 100 Days
Define response to treatment at 100 days post-peripheral blood transplant. The Standard International Criteria for responses for each disease will be used, based on CIBMTR (Center for International Blood and Marrow Transplant Research) criteria.
Measured: 100 days post-peripheral blood transplant
Number of Participants Who Achieved a Response to Treatment at One Year
Define response to treatment at one year post-peripheral blood transplant. The Standard International Criteria for responses for each disease will be used, based on CIBMTR (Center for International Blood and Marrow Transplant Research) criteria.
Measured: One year post-peripheral blood transplant
Number of Participants Who Experienced Toxicities Associated With This Treatment Regimen
Define subjects who experienced toxicities associated with this treatment regimen
Measured: Post-peripheral blood transplant
Number of Participants Who Had Incidence of Acute GVHD
Define subjects who had incidence of acute GVHD
Measured: Post-peripheral blood transplant
Number of Participants Who Had Incidence of Chronic GVHD
Define subjects who had incidence of chronic GVHD
Measured: Post-peripheral blood transplant
Number of Participants Who Experienced Donor-Recipient Chimerism Following Transplant at Days 30, 60, and 90.
Define subjects who experience donor-recipient chimerism following transplant at days 30, 60 and 90. All patients were assessed for donor-recipient chimerism at days 30, 60, and 90, but only one patient experienced chimerism. Day 90 for this patient is reported.
Measured: Days 30, 60, and 90 post-peripheral blood transplant
Number of Participants Who Experienced Treatment-Related Mortality Within the First 100 Days
Define subjects who experienced treatment-related mortality within the first 100 days post-peripheral blood transplant
Measured: 100 days post-peripheral blood transplant
Secondary outcomes (7)
Immune Checkpoint Regulators - Incidence
To characterize the incidence of immune checkpoint regulators (V-domain Ig Suppressor of T-cell Activation, cytotoxic T-lymphocyte-associated protein 4 [CTLA], Programmed cell death protein 1 [PD-1]) during early immune recovery following an allogeneic stem cell transplant.
Measured: Days 30, 60, and 90 post-transplant
Myeloid-derived Suppressor Cells (MDSCs) After Graft vs. Host Disease (GVHD) Diagnosis - Checkpoint Regulator Expression
In those patients experiencing GVHD, the study team will define the checkpoint regulator expression on MDSCs
Measured: Post-transplant through study completion or death, assessed up to 3 years post-transplant
MDSCs After GVHD Diagnosis - Peripheral Blood Mononuclear Cells
In those patients experiencing GVHD, the study team will define the peripheral blood mononuclear cells and myeloid subsets.
Measured: Post-transplant through study completion or death, assessed up to 3 years post-transplant
MDSCs After GVHD Diagnosis - Myeloid Subsets Using Flow Cytometry
In those patients experiencing GVHD, the study team will define the myeloid subsets.
Measured: Post-transplant through study completion or death, assessed up to 3 years post-transplant
MDSCs After GVHD Diagnosis - Frequency
In those patients experiencing GVHD, the study team will define the MDSCs frequency.
Measured: Post-transplant through study completion or death, assessed up to 3 years post-transplant
Immune Checkpoint Regulators - Prevalence
To characterize the prevalence of immune checkpoint regulators (VISTA, CTLA-4, PD-1) during early immune recovery following an allogeneic stem cell transplant.
Measured: Days 30, 60, and 90 post-transplant
Immune Checkpoint Regulators - Function
Flow cytometry will be used to characterize the function of immune checkpoint regulators (VISTA, CTLA-4, PD-1) during early immune recovery following an allogeneic stem cell transplant.
Measured: Days 30, 60, and 90 post-transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Mar 2018
- Study start28 Mar 2018
- Primary completion14 Feb 2025
- Study completion17 Oct 2025
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.
- Publications (23)Papers the sponsor has linked to this study.
- Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- More trials run by Dartmouth-Hitchcock Medical 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- Dartmouth-Hitchcock Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Kenneth Meehan, Dartmouth-Hitchcock Medical Center
- Investigators
- Kenneth Meehan, MD, Principal investigator, Dartmouth-Hitchcock Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03480360
- Sponsor's protocol ID
- D17170
- Other IDs
- NCI-2018-01157
- Status last verified
- Jan 2026
- First submitted
- 8 Jan 2018
- Last updated
- 17 Jul 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Bashey A, Zhang X, Sizemore CA, Manion K, Brown S, Holland HK, Morris LE, Solomon SR. T-cell-replete HLA-haploidentical hematopoietic transplantation for hematologic malignancies using post-transplantation cyclophosphamide results in outcomes equivalent to those of contemporaneous HLA-matched related and unrelated donor transplantation. J Clin Oncol. 2013 Apr 1;31(10):1310-6. doi: 10.1200/JCO.2012.44.3523. Epub 2013 Feb 19.
PubMed 23423745Solomon SR, Sizemore CA, Sanacore M, Zhang X, Brown S, Holland HK, Morris LE, Bashey A. Haploidentical transplantation using T cell replete peripheral blood stem cells and myeloablative conditioning in patients with high-risk hematologic malignancies who lack conventional donors is well tolerated and produces excellent relapse-free survival: results of a prospective phase II trial. Biol Blood Marrow Transplant. 2012 Dec;18(12):1859-66. doi: 10.1016/j.bbmt.2012.06.019. Epub 2012 Aug 1.
PubMed 22863841Ciurea SO, Zhang MJ, Bacigalupo AA, Bashey A, Appelbaum FR, Aljitawi OS, Armand P, Antin JH, Chen J, Devine SM, Fowler DH, Luznik L, Nakamura R, O'Donnell PV, Perales MA, Pingali SR, Porter DL, Riches MR, Ringden OT, Rocha V, Vij R, Weisdorf DJ, Champlin RE, Horowitz MM, Fuchs EJ, Eapen M. Haploidentical transplant with posttransplant cyclophosphamide vs matched unrelated donor transplant for acute myeloid leukemia. Blood. 2015 Aug 20;126(8):1033-40. doi: 10.1182/blood-2015-04-639831. Epub 2015 Jun 30.
PubMed 26130705Luznik L, O'Donnell PV, Symons HJ, Chen AR, Leffell MS, Zahurak M, Gooley TA, Piantadosi S, Kaup M, Ambinder RF, Huff CA, Matsui W, Bolanos-Meade J, Borrello I, Powell JD, Harrington E, Warnock S, Flowers M, Brodsky RA, Sandmaier BM, Storb RF, Jones RJ, Fuchs EJ. HLA-haploidentical bone marrow transplantation for hematologic malignancies using nonmyeloablative conditioning and high-dose, posttransplantation cyclophosphamide. Biol Blood Marrow Transplant. 2008 Jun;14(6):641-50. doi: 10.1016/j.bbmt.2008.03.005.
PubMed 18489989Mielcarek M, Martin PJ, Leisenring W, Flowers ME, Maloney DG, Sandmaier BM, Maris MB, Storb R. Graft-versus-host disease after nonmyeloablative versus conventional hematopoietic stem cell transplantation. Blood. 2003 Jul 15;102(2):756-62. doi: 10.1182/blood-2002-08-2628. Epub 2003 Mar 27.
PubMed 12663454Giralt S, Logan B, Rizzo D, Zhang MJ, Ballen K, Emmanouilides C, Nath R, Parker P, Porter D, Sandmaier B, Waller EK, Barker J, Pavletic S, Weisdorf D. Reduced-intensity conditioning for unrelated donor progenitor cell transplantation: long-term follow-up of the first 285 reported to the national marrow donor program. Biol Blood Marrow Transplant. 2007 Jul;13(7):844-52. doi: 10.1016/j.bbmt.2007.03.011. Epub 2007 May 24.
PubMed 17580263Kekre N, Antin JH. Hematopoietic stem cell transplantation donor sources in the 21st century: choosing the ideal donor when a perfect match does not exist. Blood. 2014 Jul 17;124(3):334-43. doi: 10.1182/blood-2014-02-514760. Epub 2014 Jun 9.
PubMed 24914138Bayraktar UD, Champlin RE, Ciurea SO. Progress in haploidentical stem cell transplantation. Biol Blood Marrow Transplant. 2012 Mar;18(3):372-80. doi: 10.1016/j.bbmt.2011.08.001. Epub 2011 Aug 9.
PubMed 21835146Parmesar K, Raj K. Haploidentical Stem Cell Transplantation in Adult Haematological Malignancies. Adv Hematol. 2016;2016:3905907. doi: 10.1155/2016/3905907. Epub 2016 May 30.
PubMed 27313619Luznik L, Engstrom LW, Iannone R, Fuchs EJ. Posttransplantation cyclophosphamide facilitates engraftment of major histocompatibility complex-identical allogeneic marrow in mice conditioned with low-dose total body irradiation. Biol Blood Marrow Transplant. 2002;8(3):131-8. doi: 10.1053/bbmt.2002.v8.pm11939602.
PubMed 11939602Ciurea SO, Mulanovich V, Saliba RM, Bayraktar UD, Jiang Y, Bassett R, Wang SA, Konopleva M, Fernandez-Vina M, Montes N, Bosque D, Chen J, Rondon G, Alatrash G, Alousi A, Bashir Q, Korbling M, Qazilbash M, Parmar S, Shpall E, Nieto Y, Hosing C, Kebriaei P, Khouri I, Popat U, de Lima M, Champlin RE. Improved early outcomes using a T cell replete graft compared with T cell depleted haploidentical hematopoietic stem cell transplantation. Biol Blood Marrow Transplant. 2012 Dec;18(12):1835-44. doi: 10.1016/j.bbmt.2012.07.003. Epub 2012 Jul 11.
PubMed 22796535Chang YJ, Zhao XY, Huang XJ. Immune reconstitution after haploidentical hematopoietic stem cell transplantation. Biol Blood Marrow Transplant. 2014 Apr;20(4):440-9. doi: 10.1016/j.bbmt.2013.11.028. Epub 2013 Dec 4.
PubMed 24315844Habicht A, Kewalaramani R, Vu MD, Demirci G, Blazar BR, Sayegh MH, Li XC. Striking dichotomy of PD-L1 and PD-L2 pathways in regulating alloreactive CD4(+) and CD8(+) T cells in vivo. Am J Transplant. 2007 Dec;7(12):2683-92. doi: 10.1111/j.1600-6143.2007.01999.x. Epub 2007 Oct 9.
PubMed 17924994Schilbach K, Schick J, Wehrmann M, Wollny G, Simon P, Schlegel PG, Eyrich M. PD-1-PD-L1 pathway is involved in suppressing alloreactivity of heart infiltrating t cells during murine gvhd across minor histocompatibility antigen barriers. Transplantation. 2007 Jul 27;84(2):214-22. doi: 10.1097/01.tp.0000268074.77929.54.
PubMed 17667813Blazar BR, Taylor PA, Panoskaltsis-Mortari A, Sharpe AH, Vallera DA. Opposing roles of CD28:B7 and CTLA-4:B7 pathways in regulating in vivo alloresponses in murine recipients of MHC disparate T cells. J Immunol. 1999 Jun 1;162(11):6368-77.
PubMed 10352249
Study at a glance
From the registry record, last updated by the sponsor on 17 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Haploidentical Allogeneic Peripheral Blood Transplantation: Clinical Trial and Laboratory Correlates Examining Checkpoint Immune Regulators' Expression
- ClinicalTrials.gov ID
- NCT03480360
- Acronym / sponsor ID
- D17170
- Conditions
- Acute Myeloid Leukemia, Chronic Lymphocytic Leukemia, Chronic Myeloid Leukemia, Myelodysplasia, Myeloproliferative Disorder, Myelofibrosis
- Keywords
- haploidentical, transplant, peripheral blood, allogeneic
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 21 participants (actual)
- Lead sponsor
- Dartmouth-Hitchcock Medical CenterUniversity, hospital or other
- Study start
- 28 Mar 2018 (actual)
- Primary completion
- 14 Feb 2025 (actual)
- Study completion
- 17 Oct 2025 (actual)
- First posted
- 29 Mar 2018 (actual)
- Results
- Posted 23 Jul 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The standard Johns Hopkins' regimen will be used in study subjects, with the use of donor peripheral blood stem cells, rather than marrow. Clinical outcomes will be defined while focusing efforts on immune reconstitution focusing on immune checkpoint regulators after a related haploidentical stem cell transplant.
Detailed description
We propose a clinical trial to define clinical endpoints, including engraftment, 100-day survival and one year survival (Objective #1). We will characterize the incidence, prevalence and function of immune checkpoint regulators in patients' blood and bone marrow following transplantation (Objective #2). We will correlate these laboratory results with clinical outcomes and the incidence of GVHD. As an exploratory aim, in those patients experiencing GVHD and requiring treatment, we will define the frequency/expression of checkpoint regulator expression and correlate these results with the patient's response to GVHD therapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Johns Hopkins' conditioning regimen | Other | Drug: Cyclophosphamide, Drug: Fludarabine, Radiation: Total Body Irradiation, Drug: Tacrolimus, Drug: cellcept, Drug: g-csf, Procedure: Peripheral Blood TransplantCyclophosphamide, fludarabine, total body irradiation, immune suppression including tacrolimus and cellcept, Granulocyte colony-stimulating factor (G-CSF), and peripheral blood transplant |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (20 listed)
- Age: less than 75 years
- The patient must be approved for transplant by the treating transplant physician. This includes completion of their pre-transplant workup, as directed by standard Dartmouth-Hitchcock Medical Center (DHMC) Standard Operating Procedure (SOP) (DHMC SOP - Pre-transplant Evaluation of allogeneic recipient (Appendix).
- The patient must have a disease (listed below) with treatment-responsiveness that the treating transplant physician believes will benefit from an allogeneic stem cell transplant. The diseases include:
- Acute leukemia - Acute Myeloid Leukemia, Acute Lymphocytic Leukemia
- Chronic leukemia - Chronic Myeloid Leukemia, Chronic Lymphocytic Leukemia
Key exclusion criteria (15 listed)
- Psychiatric disorder or a mental deficiency of the patient that is sufficiently severe to make compliance with the treatment unlikely, and making informed consent impossible.
- Major anticipated illness or organ failure incompatible with survival from bone marrow transplant.
- History of refractory systemic infection
- DONOR ELIGIBILITY
- Human leukocyte antigen (HLA) haplo-identical matched related.
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 |
|---|---|---|
| Cyclophosphamide14.5 mg/kg for 2 days (days -6, -5) and then 50 mg/kg for two days (days 3, 4) | Drug | Johns Hopkins' conditioning regimen |
| Fludarabine30 mg/m2 daily for 5 days | Drug | Johns Hopkins' conditioning regimen |
| Total Body Irradiation200 centigray (cGy) for one day (day -1) | Radiation | Johns Hopkins' conditioning regimen |
| Tacrolimus1 mg IV daily, (or the oral equivalent) adjusted to achieve a level between 5 and 15 ng/ml. If there is no evidence of GVHD, discontinue Tacrolimus by Day 180. | Drug | Johns Hopkins' conditioning regimen |
| cellceptdose at 15 mg/kg po three times per day (maximum dose of 3 grams/day). Stop Cellcept at Day 35 following transplantation. | Drug | Johns Hopkins' conditioning regimen |
| g-csf5 mcg/kg/d starting day 5 and continue until Absolute Neutrophil Count (ANC) > 1000/mcL for 3 days. | Drug | Johns Hopkins' conditioning regimen |
| Peripheral Blood Transplantcell dose goal: < 5 x 106 Hematopoietic progenitor cell antigen CD34+ cells/kg recipient weight | Procedure or surgery | Johns Hopkins' conditioning regimen |
Primary outcome
Number of Participants Who Survived to 100-Days Post-transplant
Time frame: 100 days post date of peripheral blood transplant
Define 100-day survival of subjects
Number of Participants Who Survived to One Year Post-Transplant.
Time frame: One year post date of peripheral blood transplant
Define one year survival of subjects
Number of Participants Who Experienced a Successful Engraftment
Time frame: Post-peripheral blood transplant
Define number of subjects who experience a successful engraftment: Defined as absolute neutrophil count > 500/mm3 and platelets > 20,000/mcl for three consecutive days (count first day as engraftment)
Number of Participants Who Achieved a Response to Treatment at 100 Days
Time frame: 100 days post-peripheral blood transplant
Define response to treatment at 100 days post-peripheral blood transplant. The Standard International Criteria for responses for each disease will be used, based on CIBMTR (Center for International Blood and Marrow Transplant Research) criteria.
Number of Participants Who Achieved a Response to Treatment at One Year
Time frame: One year post-peripheral blood transplant
Define response to treatment at one year post-peripheral blood transplant. The Standard International Criteria for responses for each disease will be used, based on CIBMTR (Center for International Blood and Marrow Transplant Research) criteria.
Number of Participants Who Experienced Toxicities Associated With This Treatment Regimen
Time frame: Post-peripheral blood transplant
Define subjects who experienced toxicities associated with this treatment regimen
Number of Participants Who Had Incidence of Acute GVHD
Time frame: Post-peripheral blood transplant
Define subjects who had incidence of acute GVHD
Number of Participants Who Had Incidence of Chronic GVHD
Time frame: Post-peripheral blood transplant
Define subjects who had incidence of chronic GVHD
Number of Participants Who Experienced Donor-Recipient Chimerism Following Transplant at Days 30, 60, and 90.
Time frame: Days 30, 60, and 90 post-peripheral blood transplant
Define subjects who experience donor-recipient chimerism following transplant at days 30, 60 and 90. All patients were assessed for donor-recipient chimerism at days 30, 60, and 90, but only one patient experienced chimerism. Day 90 for this patient is reported.
Number of Participants Who Experienced Treatment-Related Mortality Within the First 100 Days
Time frame: 100 days post-peripheral blood transplant
Define subjects who experienced treatment-related mortality within the first 100 days post-peripheral blood transplant
7 secondary outcomes
Immune Checkpoint Regulators - Incidence
Time frame: Days 30, 60, and 90 post-transplant
Myeloid-derived Suppressor Cells (MDSCs) After Graft vs. Host Disease (GVHD) Diagnosis - Checkpoint Regulator Expression
Time frame: Post-transplant through study completion or death, assessed up to 3 years post-transplant
MDSCs After GVHD Diagnosis - Peripheral Blood Mononuclear Cells
Time frame: Post-transplant through study completion or death, assessed up to 3 years post-transplant
MDSCs After GVHD Diagnosis - Myeloid Subsets Using Flow Cytometry
Time frame: Post-transplant through study completion or death, assessed up to 3 years post-transplant
MDSCs After GVHD Diagnosis - Frequency
Time frame: Post-transplant through study completion or death, assessed up to 3 years post-transplant
Immune Checkpoint Regulators - Prevalence
Time frame: Days 30, 60, and 90 post-transplant
Immune Checkpoint Regulators - Function
Time frame: Days 30, 60, and 90 post-transplant
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Jul 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 23423745Bashey A, Zhang X, Sizemore CA, Manion K, Brown S, Holland HK, Morris LE, Solomon SR. T-cell-replete HLA-haploidentical hematopoietic transplantation for hematologic malignancies using post-transplantation cyclophosphamide results in outcomes equivalent to those of contemporaneous HLA-matched related and unrelated donor transplantation. J Clin Oncol. 2013 Apr 1;31(10):1310-6. doi: 10.1200/JCO.2012.44.3523. Epub 2013 Feb 19.
- Background: PubMed 22863841Solomon SR, Sizemore CA, Sanacore M, Zhang X, Brown S, Holland HK, Morris LE, Bashey A. Haploidentical transplantation using T cell replete peripheral blood stem cells and myeloablative conditioning in patients with high-risk hematologic malignancies who lack conventional donors is well tolerated and produces excellent relapse-free survival: results of a prospective phase II trial. Biol Blood Marrow Transplant. 2012 Dec;18(12):1859-66. doi: 10.1016/j.bbmt.2012.06.019. Epub 2012 Aug 1.
- Background: PubMed 26130705Ciurea SO, Zhang MJ, Bacigalupo AA, Bashey A, Appelbaum FR, Aljitawi OS, Armand P, Antin JH, Chen J, Devine SM, Fowler DH, Luznik L, Nakamura R, O'Donnell PV, Perales MA, Pingali SR, Porter DL, Riches MR, Ringden OT, Rocha V, Vij R, Weisdorf DJ, Champlin RE, Horowitz MM, Fuchs EJ, Eapen M. Haploidentical transplant with posttransplant cyclophosphamide vs matched unrelated donor transplant for acute myeloid leukemia. Blood. 2015 Aug 20;126(8):1033-40. doi: 10.1182/blood-2015-04-639831. Epub 2015 Jun 30.
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- 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.
Kenneth Meehan, MD principal investigator
Dartmouth-Hitchcock Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Cyclophosphamide, Fludarabine and Total Body Irradiation in people with Acute Myeloid Leukemia and Chronic Lymphocytic Leukemia, run by Dartmouth-Hitchcock Medical 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 20 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: finished, as of the sponsor's last update on 17 Jul 2026. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 23 Jul 2024). 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 to 100-Days Post-transplant (time frame: 100 days post date of peripheral blood transplant). The study lists 10 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03480360, which also lists 23 linked publications.
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 17 Jul 2026.