NCT00429143

A Two-Step Approach to Bone Marrow Transplant Using Cells From A Partially-Matched Relative

A Two Step Approach To Allogeneic Hematopoietic Stem Cell Transplantation for Hematologic Malignancies From HLA Partially-Matched Related Donors

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 27 (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 study is to develop a way of treating patients who do not have a completely matched family donor or a readily available unrelated donor with bone marrow transplant by using a partially-matched family donor. Patients receiving this type of transplant will receive chemotherapy and/or radiation to treat their disease. They will also receive their donor's cells in 2 parts. During the first part, the donor's lymphocytes will be exposed to one of the chemotherapy agents to help the patient become tolerant to the lymphocytes. In the second part of the transplant, the patient will receive their donor's stem cells to help recover their peripheral blood counts and establish long-term engraftment. The hypothesis of this study is that in partially-matched allogeneic transplant, there is a defined number of donor T-cells that can be treated and given to the recipient to avoid post-transplant infection without causing severe graft-versus-host disease.

Detailed description

Haploidentical hematopoietic stem cell transplant is a life-saving therapy for patients who are without well matched donors. This type of therapy has been associated with poor outcomes in the past due to complications such as infection. The Jefferson 2 Step approach was designed to allow the infusion of an exact dose of tolerized lymphocytes in haploidentical transplant in order to allow for immune reconstitution post transplant to avoid infectious complications while still having acceptable rates of GVHD. In this approach, patients with high-risk hematological malignancies undergo 8 fractions of TBI (12 Gy) followed by an exact dose of donor lymphocytes. The phase I portion of the study determined the optimal dose of lymphocytes. Two days after receiving the donor lymphocytes, the patients receive 2 daily doses of cyclophosphamide. One day after receiving cyclophosphamide, the patients receive stem cell from their donor. Tacrolimus and mycophenylate mofetil are used as GVHD prophylaxis.

Conditions

• Hematologic Malignancies

Interventions

Type	Name	Description
RADIATION	Total Body Irradiation (TBI)	TBI twice daily days 6-9 prior to transplant (HSCT)
BIOLOGICAL	Donor Lymphocyte Infusion (DLI)	DLI given 6 days prior to transplant (HSCT).
DRUG	Cyclophosphamide (CY)	Cyclophosphamide given once daily at 60 mg/kg on days 2 and 3 prior to transplant (HSCT).
DRUG	Tacrolimus	Tacrolimus given one day prior to transplant (HSCT).
DRUG	Mycophenolate Mofetil (MMF)	MMF given one day prior to transplant (HSCT).
BIOLOGICAL	Hematopoietic Stem Cell Transplant (HSCT)	CD34+ selected Hematopoietic Stem Cell Transplant (HSCT) is performed. This is the day of transplantation.

Timeline

• Start date: 2006-01-01
• Primary completion: 2009-08-01
• Completion: 2010-06-01
• First posted: 2007-01-31
• Last updated: 2025-05-04
• Results posted: 2013-05-31

Locations

1 site across 1 country: United States