NCT01341301

Donor Stem Cell Transplant in Treating Patients With High-Risk Hematologic Malignancies

A Two Step Approach to Allogeneic Hematopoietic Stem Cell Transplantation for High-Risk Hematologic Malignancies Using One Human Leukocyte Antigen Partially-Matched Related Donor

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 25 (actual)
• Sponsor: Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this research study is to examine the survival of patients undergoing partially matched hematopoietic stem cell transplant (HSCT) on a new type of treatment approach, which has been developed specifically for patients who have evidence of their disease at the time of transplant. In this research study, a way of strengthening the response of the donor cells against the disease has been developed. Patients will undergo one additional day between the two steps of the transplant which may allow their donor's cells to fight the disease more effectively.

Detailed description

This is a phase II study in which patients receive a haploidentical HSCT from a single donor. The period between the donor lymphocyte infusion (DLI) and tolerizing doses of CY has been extended to allow for an increased period of allogeneic response against tumor targets. The outcomes of patients undergoing this extra time period will be compared to historical data to assess efficacy. Primary Objective: 1\) To assess 1 year relapse free survival in patients undergoing hematopoietic stem cell transplant (HSCT) using the Thomas Jefferson University (TJU) 2 step approach with an extra day inserted between the DLI and administration of cyclophosphamide (CY). Secondary Objectives: 1. To assess the consistency and pace of engraftment. 2. To assess the pace of T cell and B cell immune recovery. 3. To assess regimen related toxicity, (GVHD) graft-versus-host disease incidence and severity, and overall survival in patients undergoing treatment on this protocol. . 4. To assess the tolerance of the period of fever, diarrhea, and rash in each arm in an effort to determine whether a longer interval prior to cytoxan changes this side effect qualitatively compared to prior patient groups or concurrent patient groups. N.B. Patients with hematologic malignancies in remission will continue to be transplanted without modification to the original 2-step approach and will serve as a concurrent comparison group. 5. To collect leukemia samples prior to transplant and after relapse whenever possible. To assess the overall degree of HLA-class I and class II expression on these paired samples. To test for loss of one or both HLA haplotypes in the relapsed tumor specimens. 6. To determine the number of cluster of differentiation 4 (CD4+) cluster of differentiation 25 (CD25+) FOXP3+ regulatory cells post HSCT and to assess whether this is correlated with the development of GVHD after transplant.

Conditions

• Hematologic Malignancy

Interventions

Type	Name	Description
RADIATION	Total Body Irradiation	Undergo TBI
BIOLOGICAL	Donor Lymphocyte Infusion (DLI)	Undergo DLI
DRUG	Cyclophosphamide	Given IV
DRUG	Tacrolimus	Given IV or PO
DRUG	Mycophenolate mofetil	Given IV or PO
PROCEDURE	Allogeneic hematopoietic stem cell transplantation	Undergo allogeneic HSCT
OTHER	Laboratory biomarker analysis	Correlative studies

Timeline

• Start date: 2010-05-01
• Primary completion: 2013-11-04
• Completion: 2014-08-07
• First posted: 2011-04-25
• Last updated: 2025-04-30
• Results posted: 2018-03-16

Locations

1 site across 1 country: United States