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

NCT ID: NCT03480360

Last Updated: 2024-07-23

Study Results

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Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

21 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-03-28

Study Completion Date

2025-09-13

Brief Summary

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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

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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

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Acute Myeloid Leukemia Chronic Lymphocytic Leukemia Chronic Myeloid Leukemia Myelodysplasia Myeloproliferative Disorder Myelofibrosis Lymphoma Lymphoma, Non-Hodgkin Plasma Cell Disorder

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Johns Hopkins' conditioning regimen

Cyclophosphamide, fludarabine, total body irradiation, immune suppression including tacrolimus and cellcept, Granulocyte colony-stimulating factor (G-CSF), and peripheral blood transplant

Group Type OTHER

Cyclophosphamide

Intervention Type DRUG

14.5 mg/kg for 2 days (days -6, -5) and then 50 mg/kg for two days (days 3, 4)

Fludarabine

Intervention Type DRUG

30 mg/m2 daily for 5 days

Total Body Irradiation

Intervention Type RADIATION

200 centigray (cGy) for one day (day -1)

Tacrolimus

Intervention Type DRUG

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.

cellcept

Intervention Type DRUG

dose at 15 mg/kg po three times per day (maximum dose of 3 grams/day). Stop Cellcept at Day 35 following transplantation.

g-csf

Intervention Type DRUG

5 mcg/kg/d starting day 5 and continue until Absolute Neutrophil Count (ANC) \> 1000/mcL for 3 days.

Peripheral Blood Transplant

Intervention Type PROCEDURE

cell dose goal: \< 5 x 106 Hematopoietic progenitor cell antigen CD34+ cells/kg recipient weight

Interventions

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Cyclophosphamide

14.5 mg/kg for 2 days (days -6, -5) and then 50 mg/kg for two days (days 3, 4)

Intervention Type DRUG

Fludarabine

30 mg/m2 daily for 5 days

Intervention Type DRUG

Total Body Irradiation

200 centigray (cGy) for one day (day -1)

Intervention Type RADIATION

Tacrolimus

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.

Intervention Type DRUG

cellcept

dose at 15 mg/kg po three times per day (maximum dose of 3 grams/day). Stop Cellcept at Day 35 following transplantation.

Intervention Type DRUG

g-csf

5 mcg/kg/d starting day 5 and continue until Absolute Neutrophil Count (ANC) \> 1000/mcL for 3 days.

Intervention Type DRUG

Peripheral Blood Transplant

cell dose goal: \< 5 x 106 Hematopoietic progenitor cell antigen CD34+ cells/kg recipient weight

Intervention Type PROCEDURE

Eligibility Criteria

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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


* 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
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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Dartmouth-Hitchcock Medical Center

OTHER

Sponsor Role lead

Responsible Party

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Kenneth Meehan

Principal Investogator- Kenneth Meehan, MD Staff Physician

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Kenneth Meehan, MD

Role: PRINCIPAL_INVESTIGATOR

Dartmouth-Hitchcock Medical Center

Locations

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Dartmouth Hitchcock Medical Center, Norris Cotton Cancer Center

Lebanon, New Hampshire, United States

Site Status

Countries

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United States

References

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Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

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D17170

Identifier Type: -

Identifier Source: org_study_id

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