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NCT03480360 · REGISTRY CONDITION SEARCH

Haploidentical Allogeneic Peripheral Blood Transplantation: Examining Checkpoint Immune Regulators' Expression

Found through the registry condition index, not selected as a treatment recommendation. This discovery collection was restricted to studies with posted results.

Phase
PHASE3
Status at capture
COMPLETED
Registry last update
2026-07-17

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.

Open the original ClinicalTrials.gov record → · Download preserved record

What did the study report?

Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.

flow

Participant flow

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baseline

Baseline characteristics

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outcome · POSTED

Number of Participants Who Survived to 100-Days Post-transplant

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outcome · POSTED

Number of Participants Who Survived to One Year Post-Transplant.

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outcome · POSTED

Number of Participants Who Experienced a Successful Engraftment

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outcome · POSTED

Number of Participants Who Achieved a Response to Treatment at 100 Days

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outcome · POSTED

Number of Participants Who Achieved a Response to Treatment at One Year

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outcome · POSTED

Number of Participants Who Experienced Toxicities Associated With This Treatment Regimen

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outcome · POSTED

Number of Participants Who Had Incidence of Acute GVHD

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outcome · POSTED

Number of Participants Who Had Incidence of Chronic GVHD

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outcome · POSTED

Number of Participants Who Experienced Donor-Recipient Chimerism Following Transplant at Days 30, 60, and 90.

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outcome · POSTED

Number of Participants Who Experienced Treatment-Related Mortality Within the First 100 Days

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outcome · POSTED

Immune Checkpoint Regulators - Incidence

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outcome · POSTED

Myeloid-derived Suppressor Cells (MDSCs) After Graft vs. Host Disease (GVHD) Diagnosis - Checkpoint Regulator Expression

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outcome · POSTED

MDSCs After GVHD Diagnosis - Peripheral Blood Mononuclear Cells

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outcome · POSTED

MDSCs After GVHD Diagnosis - Myeloid Subsets Using Flow Cytometry

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outcome · POSTED

MDSCs After GVHD Diagnosis - Frequency

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outcome · POSTED

Immune Checkpoint Regulators - Prevalence

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outcome · POSTED

Immune Checkpoint Regulators - Function

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adverse events

Adverse events

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more info

Limitations, agreements, and source contact

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Who could take part
eligibility Criteria
Inclusion Criteria: * 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 * 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). Exclusion Criteria: * 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) * 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
healthy Volunteers
true
maximum Age
75 Years
minimum Age
18 Years
sex
ALL
std Ages
  1. ADULT
  2. OLDER_ADULT
Treatment arms and interventions
arm Groups
  1. description
    Cyclophosphamide, fludarabine, total body irradiation, immune suppression including tacrolimus and cellcept, Granulocyte colony-stimulating factor (G-CSF), and peripheral blood transplant
    intervention Names
    1. Drug: Cyclophosphamide
    2. Drug: Fludarabine
    3. Radiation: Total Body Irradiation
    4. Drug: Tacrolimus
    5. Drug: cellcept
    6. Drug: g-csf
    7. Procedure: Peripheral Blood Transplant
    label
    Johns Hopkins' conditioning regimen
    type
    OTHER
interventions
  1. arm Group Labels
    1. Johns Hopkins' conditioning regimen
    description
    14.5 mg/kg for 2 days (days -6, -5) and then 50 mg/kg for two days (days 3, 4)
    name
    Cyclophosphamide
    type
    DRUG
  2. arm Group Labels
    1. Johns Hopkins' conditioning regimen
    description
    30 mg/m2 daily for 5 days
    name
    Fludarabine
    type
    DRUG
  3. arm Group Labels
    1. Johns Hopkins' conditioning regimen
    description
    200 centigray (cGy) for one day (day -1)
    name
    Total Body Irradiation
    type
    RADIATION
  4. arm Group Labels
    1. Johns Hopkins' conditioning regimen
    description
    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.
    name
    Tacrolimus
    type
    DRUG
  5. arm Group Labels
    1. Johns Hopkins' conditioning regimen
    description
    dose at 15 mg/kg po three times per day (maximum dose of 3 grams/day). Stop Cellcept at Day 35 following transplantation.
    name
    cellcept
    type
    DRUG
  6. arm Group Labels
    1. Johns Hopkins' conditioning regimen
    description
    5 mcg/kg/d starting day 5 and continue until Absolute Neutrophil Count (ANC) \> 1000/mcL for 3 days.
    name
    g-csf
    type
    DRUG
  7. arm Group Labels
    1. Johns Hopkins' conditioning regimen
    description
    cell dose goal: \< 5 x 106 Hematopoietic progenitor cell antigen CD34+ cells/kg recipient weight
    name
    Peripheral Blood Transplant
    type
    PROCEDURE
Study design
allocation
NA
intervention Model
SINGLE_GROUP
masking Info
masking
NONE
primary Purpose
TREATMENT
Enrollment
count
21
type
ACTUAL
Registered outcome plans (not posted results)
primary Outcomes
  1. description
    Define 100-day survival of subjects
    measure
    Number of Participants Who Survived to 100-Days Post-transplant
    time Frame
    100 days post date of peripheral blood transplant
  2. description
    Define one year survival of subjects
    measure
    Number of Participants Who Survived to One Year Post-Transplant.
    time Frame
    One year post date of peripheral blood transplant
  3. description
    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)
    measure
    Number of Participants Who Experienced a Successful Engraftment
    time Frame
    Post-peripheral blood transplant
  4. description
    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.
    measure
    Number of Participants Who Achieved a Response to Treatment at 100 Days
    time Frame
    100 days post-peripheral blood transplant
  5. description
    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.
    measure
    Number of Participants Who Achieved a Response to Treatment at One Year
    time Frame
    One year post-peripheral blood transplant
  6. description
    Define subjects who experienced toxicities associated with this treatment regimen
    measure
    Number of Participants Who Experienced Toxicities Associated With This Treatment Regimen
    time Frame
    Post-peripheral blood transplant
  7. description
    Define subjects who had incidence of acute GVHD
    measure
    Number of Participants Who Had Incidence of Acute GVHD
    time Frame
    Post-peripheral blood transplant
  8. description
    Define subjects who had incidence of chronic GVHD
    measure
    Number of Participants Who Had Incidence of Chronic GVHD
    time Frame
    Post-peripheral blood transplant
  9. description
    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.
    measure
    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
  10. description
    Define subjects who experienced treatment-related mortality within the first 100 days post-peripheral blood transplant
    measure
    Number of Participants Who Experienced Treatment-Related Mortality Within the First 100 Days
    time Frame
    100 days post-peripheral blood transplant
secondary Outcomes
  1. description
    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.
    measure
    Immune Checkpoint Regulators - Incidence
    time Frame
    Days 30, 60, and 90 post-transplant
  2. description
    In those patients experiencing GVHD, the study team will define the checkpoint regulator expression on MDSCs
    measure
    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
  3. description
    In those patients experiencing GVHD, the study team will define the peripheral blood mononuclear cells and myeloid subsets.
    measure
    MDSCs After GVHD Diagnosis - Peripheral Blood Mononuclear Cells
    time Frame
    Post-transplant through study completion or death, assessed up to 3 years post-transplant
  4. description
    In those patients experiencing GVHD, the study team will define the myeloid subsets.
    measure
    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
  5. description
    In those patients experiencing GVHD, the study team will define the MDSCs frequency.
    measure
    MDSCs After GVHD Diagnosis - Frequency
    time Frame
    Post-transplant through study completion or death, assessed up to 3 years post-transplant
  6. description
    To characterize the prevalence of immune checkpoint regulators (VISTA, CTLA-4, PD-1) during early immune recovery following an allogeneic stem cell transplant.
    measure
    Immune Checkpoint Regulators - Prevalence
    time Frame
    Days 30, 60, and 90 post-transplant
  7. description
    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.
    measure
    Immune Checkpoint Regulators - Function
    time Frame
    Days 30, 60, and 90 post-transplant
Full study description
brief 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.
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.
Source references
references
  1. citation
    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.
    pmid
    23423745
    type
    BACKGROUND
  2. citation
    Solomon 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.
    pmid
    22863841
    type
    BACKGROUND
  3. citation
    Ciurea 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.
    pmid
    26130705
    type
    BACKGROUND
  4. citation
    Luznik 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.
    pmid
    18489989
    type
    BACKGROUND
  5. citation
    Mielcarek 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.
    pmid
    12663454
    type
    BACKGROUND
  6. citation
    Giralt 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.
    pmid
    17580263
    type
    BACKGROUND
  7. citation
    Kekre 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.
    pmid
    24914138
    type
    BACKGROUND
  8. citation
    Bayraktar 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.
    pmid
    21835146
    type
    BACKGROUND
  9. citation
    Parmesar 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.
    pmid
    27313619
    type
    BACKGROUND
  10. citation
    Luznik 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.
    pmid
    11939602
    type
    BACKGROUND
  11. citation
    Ciurea 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.
    pmid
    22796535
    type
    BACKGROUND
  12. citation
    Chang 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.
    pmid
    24315844
    type
    BACKGROUND
Source notices and limitations
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    Preserved source evidence · Not medical advice