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Laboratory-Treated Lymphocyte Infusion After Haploidentical Donor Stem Cell Transplant

Phase 1
Completed
Conditions
Leukemia
Myelodysplastic Syndromes
Interventions
Biological: anti-thymocyte globulin
Biological: peripheral blood lymphocyte therapy
Procedure: allogeneic hematopoietic stem cell transplantation
Procedure: in vitro-treated peripheral blood stem cell transplantation
Radiation: total-body irradiation
Registration Number
NCT00376480
Lead Sponsor
Dana-Farber Cancer Institute
Brief Summary

RATIONALE: Giving total-body irradiation and chemotherapy, such as thiotepa and fludarabine, before a donor stem cell transplant helps stop the growth of cancer or abnormal cells. It also helps stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving methylprednisolone and antithymocyte globulin before transplant and peripheral blood cells that have been treated in the laboratory after transplant may stop this from happening.

PURPOSE: This phase I trial is studying the side effects and best dose of laboratory-treated peripheral blood cell infusion after donor stem cell transplant in treating patients with hematologic cancers or other diseases.

Detailed Description

OBJECTIVES:

Primary

* Establish the feasibility of delayed infusion of ex vivo anergized donor peripheral blood mononuclear cells (PBMC) after CD34 (cluster designation 34)-selected megadose haploidentical hematopoietic stem cell transplantation (HSCT) in patients with hematopoietic cancers or other diseases.

* Determine the feasibility of collecting parental allogeneic stimulator cells to induce anergy to the nonshared donor-recipient haplotype in these patients.

* Determine the feasibility of collecting donor PBMC as a source of T cells for ex vivo anergization.

* Determine the number of transplanted individuals who meet the criteria for proceeding to delayed infusion of ex vivo anergized donor PBMC.

* Establish the safety of delayed infusion of ex vivo anergized donor PBMC by establishing the maximum number of donor T cells that can be infused without unacceptable graft-versus-host disease.

Secondary

* Evaluate, in vitro, the induction and specificity of alloantigen hyporesponsiveness in donor PBMC after ex vivo anergization.

* Assess, in vitro, the function of immune cells engrafted in these patients.

* Assess, in vitro, whether alloantigen hyporesponsive donor T cells are present in these patients.

* Develop, preliminarily, in vitro data on the extent of pathogen-specific immunity and its rate of recovery.

* Describe the patterns of opportunistic infections in these patients.

OUTLINE: This is a multicenter, dose-escalation study of ex vivo anergized allogeneic peripheral blood mononuclear cells (PBMC). Patients who are treated on any dose level except dose level 1 are stratified according to age (under 17 \[pediatric\] vs 17 and over \[adult\]).

* Myeloablative conditioning regimen: Patients undergo total-body irradiation twice daily on days -11 to -9. Patients also receive thiotepa IV over 4 hours on days -8 and -7, fludarabine phosphate IV over 30 minutes on days -7 to -3, and anti-thymocyte globulin IV over 8 hours and methylprednisolone IV over 15-30 minutes on days -6 to -3.

* Allogeneic peripheral blood stem cell transplantation (PBSCT): Patients undergo CD34-selected PBSCT on day 0.

* Ex vivo anergized allogeneic PBMC infusion: If cells have engrafted and patients are free of active uncontrolled infection and graft-vs-host disease, patients undergo allogeneic or autologous PBMC infusion on day 35 or 42.

Cohorts of 3-8 patients receive escalating doses of ex vivo anergized allogeneic PBMCs until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose at which 2 of 5 or 3 of 8 patients experience dose-limiting toxicity.

After completion of study, patients are followed periodically for 2 years.

PROJECTED ACCRUAL: A total of 40 patients will be accrued for this study.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
19
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
administration of adoptive donor lymphocyte infusionallogeneic hematopoietic stem cell transplantationadministration of donor lymphocytes made using costimulatory blockade ex vivo
administration of adoptive donor lymphocyte infusionin vitro-treated peripheral blood stem cell transplantationadministration of donor lymphocytes made using costimulatory blockade ex vivo
administration of adoptive donor lymphocyte infusiontotal-body irradiationadministration of donor lymphocytes made using costimulatory blockade ex vivo
administration of adoptive donor lymphocyte infusionanti-thymocyte globulinadministration of donor lymphocytes made using costimulatory blockade ex vivo
administration of adoptive donor lymphocyte infusionperipheral blood lymphocyte therapyadministration of donor lymphocytes made using costimulatory blockade ex vivo
administration of adoptive donor lymphocyte infusionfludarabine phosphateadministration of donor lymphocytes made using costimulatory blockade ex vivo
administration of adoptive donor lymphocyte infusionthiotepaadministration of donor lymphocytes made using costimulatory blockade ex vivo
administration of adoptive donor lymphocyte infusionmethylprednisoloneadministration of donor lymphocytes made using costimulatory blockade ex vivo
Primary Outcome Measures
NameTimeMethod
Feasibility of making and administering the adoptive T cell productfrom conditioning through administration of anergized cells on day 35-42

ability to collect sufficient cells, make anergized product with good viability, without contamination and infuse per study toxicity of the conditioning regimen, the likelihood of engraftment, and the subsequent percentage of individuals who would be eligible to receive aDLI were determined.

Safety of administering the adoptive T cell product on day 35-42 post haploidentical transplantthe period from aDLI infusion through D100

rates of graft failure with CD34 selected product, adverse and severe adverse reactions attributable to infusion of anergized donor cells, including fever, hypotension, acute graft vs host disease, organ dysfunction

Alloreactivity engendered by administering the adoptive T cell productfrom cell infusion through day 100

occurrence and severity of acute GVHD

Secondary Outcome Measures
NameTimeMethod
Efficacy in restoring adaptive immunityfrom aDLI thorough 1 year

incidence of viral infection and type of immune reconstitution by phenotype and function of T cells

Trial Locations

Locations (6)

M. D. Anderson Cancer Center at University of Texas

🇺🇸

Houston, Texas, United States

University of Florida Health Science Center - Gainesville

🇺🇸

Gainesville, Florida, United States

Massachusetts General Hospital

🇺🇸

Boston, Massachusetts, United States

Childrens Hospital Los Angeles

🇺🇸

Los Angeles, California, United States

Children's Hospital Boston

🇺🇸

Boston, Massachusetts, United States

Dana Farber Cancer Institute

🇺🇸

Boston, Massachusetts, United States

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