Bone Marrow and Kidney Transplant for Patients With Chronic Kidney Disease and Blood Disorders (BMT)
The main purpose of this study is to examine the outcome of a combined bone marrow and kidney transplant from a partially matched related (haploidentical or "haplo") donor. This is a pilot study, you are being asked to participate because you have a blood disorder and kidney disease. The aim of the combined transplant is to treat both your underlying blood disorder and kidney disease. We expect to have about 10 people participate in this study.
Additionally, because the same person who is donating the kidney will also be donating the bone marrow, there may be a smaller chance of kidney rejection and less need for long-term use of anti-rejection drugs.
Traditionally, very strong cancer treatment drugs (chemotherapy) and radiation are used to prepare a subject's body for bone marrow transplant. This is associated with a high risk for serious complications, even in subjects without kidney disease. This therapy can be toxic to the liver, lungs, mucous membranes, and intestines. Additionally, it is believed that standard therapy may be associated with a higher risk of a complication called graft versus host disease (GVHD) where the new donor cells attack the recipient's normal body. Recently, less intense chemotherapy and radiation regimens have been employed (these are called reduced intensity regimens) which cause less injury and GVHD to patients, and thus, have allowed older and less healthy patients to undergo bone marrow transplant. In this study, a reduced intensity regimen of chemotherapy and radiation will be used with the intent of producing fewer toxicities than standard therapy.
Typical therapy following a standard kidney transplant includes multiple lifelong medications that aim to prevent the recipient's body from attacking or rejecting the donated kidney. These are called immunosuppressant drugs and they work by "quieting" the recipient's immune system to allow the donated kidney to function properly. One goal in our study is to decrease the duration you will need to be on immunosuppressant drugs following your kidney transplant as the bone marrow transplant will provide you with the donor's immune system which should not attack the donor kidney.
Chronic Kidney Disease
Acute Myeloid Leukemia (AML)
Acute Lymphoblastic Leukemia (ALL)
Chronic Myelogenous Leukemia (CML)
Chronic Lymphocytic Leukemia (CLL)
Non-Hodgkin's Lymphoma (NHL)
Myelodysplastic Syndrome (MDS)
Diamond Blackfan Anemia
Sickle Cell Anemia
Procedure: Haploidentical Bone Marrow/Kidney
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Combined Haploidentical Reduced Intensity Bone Marrow and Kidney Transplantation for Patients With Chronic Kidney Disease and Advanced Hematological Disorders|
- Number of patients who die of treatment-related complications. [ Time Frame: 100 days and 1 year post transplant ] [ Designated as safety issue: Yes ]Assess safety of haploidentical combined bone marrow and kidney transplantation as measured treatment related mortality.
- Number of patients with acute and delayed renal allograft rejection [ Time Frame: 2 years post-transplant ] [ Designated as safety issue: Yes ]
- Number of patients who are able to discontinue immunosuppressive therapy by one year post transplant [ Time Frame: one year post transplant ] [ Designated as safety issue: No ]
- Number of patients who develp acute and chronic graft versus host disease (GVHD). [ Time Frame: post transplant ] [ Designated as safety issue: No ]
- Number of patients who relapse from their underlying hematological disease [ Time Frame: 6 months, 1 year, and 2 years post transplant. ] [ Designated as safety issue: No ]
|Study Start Date:||November 2012|
|Estimated Study Completion Date:||July 2016|
|Estimated Primary Completion Date:||July 2016 (Final data collection date for primary outcome measure)|
Haploidentical Bone Marrow/Kidney
Single Arm Study
Procedure: Haploidentical Bone Marrow/Kidney
Combined bone marrow and kidney transplantation using a haploidentical donor.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01758042
|United States, Massachusetts|
|Massachusetts General Hospital|
|Boston, Massachusetts, United States, 02114|
|Principal Investigator:||Yi-Bin A Chen, M.D.||Director of Clinical Research, Massachusetts General Hospital Bone Marrow Transplant Program|