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

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

27 participants

Study Classification

INTERVENTIONAL

Study Start Date

2006-01-31

Study Completion Date

2010-06-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Bone Marrow Transplantation of Patients in Remission Using Partially Matched Relative Donor

NCT01350245

Acute Myeloid Leukemia Myelodysplastic Syndromes Biphenotypic Leukemia +7 more

COMPLETED PHASE2

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

NCT01341301

Hematologic Malignancy

COMPLETED PHASE2

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

NCT01532635

Hematologic Malignancy Leukemia Acute Lymphoblastic Leukemia +9 more

TERMINATED PHASE2

A Two-Step Approach to Reduced Intensity Bone Marrow Transplant for Patients With Hematological Malignancies

NCT01384513

Adult Acute Lymphoblastic Leukemia in Remission Adult Acute Myeloid Leukemia in Remission Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities +44 more

COMPLETED PHASE2

Haploidentical Donor Hematopoietic Stem Cell Transplant in Treating Patients With Hematologic Malignancies

NCT01871441

Malignant Neoplasm

TERMINATED PHASE2

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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

See the medical conditions and disease areas that this research is targeting or investigating.

Hematologic Malignancies

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Haploidentical Allogeneic Transplantation

Patients undergoing hematopoietic stem cell transplant from a partially matched related donor

Group Type EXPERIMENTAL

Total Body Irradiation (TBI)

Intervention Type RADIATION

TBI twice daily days 6-9 prior to transplant (HSCT)

Donor Lymphocyte Infusion (DLI)

Intervention Type BIOLOGICAL

DLI given 6 days prior to transplant (HSCT).

Cyclophosphamide (CY)

Intervention Type DRUG

Cyclophosphamide given once daily at 60 mg/kg on days 2 and 3 prior to transplant (HSCT).

Tacrolimus

Intervention Type DRUG

Tacrolimus given one day prior to transplant (HSCT).

Mycophenolate Mofetil (MMF)

Intervention Type DRUG

MMF given one day prior to transplant (HSCT).

Hematopoietic Stem Cell Transplant (HSCT)

Intervention Type BIOLOGICAL

CD34+ selected Hematopoietic Stem Cell Transplant (HSCT) is performed. This is the day of transplantation.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Total Body Irradiation (TBI)

TBI twice daily days 6-9 prior to transplant (HSCT)

Intervention Type RADIATION

Donor Lymphocyte Infusion (DLI)

DLI given 6 days prior to transplant (HSCT).

Intervention Type BIOLOGICAL

Cyclophosphamide (CY)

Cyclophosphamide given once daily at 60 mg/kg on days 2 and 3 prior to transplant (HSCT).

Intervention Type DRUG

Tacrolimus

Tacrolimus given one day prior to transplant (HSCT).

Intervention Type DRUG

Mycophenolate Mofetil (MMF)

MMF given one day prior to transplant (HSCT).

Intervention Type DRUG

Hematopoietic Stem Cell Transplant (HSCT)

CD34+ selected Hematopoietic Stem Cell Transplant (HSCT) is performed. This is the day of transplantation.

Intervention Type BIOLOGICAL

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

TBI radiotherapy DLI T cell infusion CY Endoxan Cytoxan Neosar Procytox Revimmune cytophosphane FK-506 fujimycin Prograf Advagraf Protopic MMF CellCept Myfortic HSCT stem cell transplant

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

1. Any patient with a hematologic or oncologic diagnosis in which allogeneic HSCT is thought to be beneficial, and in whom front-line therapy has already been applied.
2. Patients must have a related donor who is either a one, two or three out of six antigen mismatch at the HLA-A;B;DR loci.
3. Patients without a well-matched unrelated donor or those who have a disease status that precludes a wait for an identified unrelated donor.
4. Patients must adequate organ function:

* LVEF of \>45%
* FVC or FEV1 \>45% of predicted
* Adequate liver function as defined by a serum bilirubin \<1.8, AST or ALT \< 2.5X upper limit of normal
* Serum creatinine \< 2.0 mg/dl or creatinine clearance of \> 40 ml/min
5. Performance status \> 60% (Karnofsky)
6. Patients must be willing to use contraception if they have childbearing potential
7. Able to give informed consent

Exclusion Criteria

1. An eligible HLA-identical sibling donor.
2. Performance status \< 60% (Karnosfsky)
3. HIV positive
4. Active involvement of the central nervous system with malignancy
5. Psychiatric disorder that would preclude patients from signing an informed consent
6. Pregnancy
7. Patients with life expectancy of \< 6 months for reasons other than their underlying hematologic/oncologic disorder.

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No