Bone Marrow and Kidney Transplant for Patients With Chronic Kidney Disease and Blood Disorders (BMT)
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Purpose
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.
| Condition | Intervention |
|---|---|
|
Chronic Kidney Disease Acute Myeloid Leukemia (AML) Acute Lymphoblastic Leukemia (ALL) Chronic Myelogenous Leukemia (CML) Chronic Lymphocytic Leukemia (CLL) Non-Hodgkin's Lymphoma (NHL) Hodgkin Disease Multiple Myeloma Myelodysplastic Syndrome (MDS) Aplastic Anemia AL Amyloidosis Diamond Blackfan Anemia Myelofibrosis Myeloproliferative Disease Sickle Cell Anemia Autoimmune Diseases Thalassemia |
Procedure: Haploidentical Bone Marrow/Kidney |
| Study Type: | Interventional |
| 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 ]
| Estimated Enrollment: | 10 |
| Study Start Date: | November 2012 |
| Estimated Study Completion Date: | January 2015 |
| Estimated Primary Completion Date: | January 2015 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Haploidentical Bone Marrow/Kidney
Single Arm Study
|
Procedure: Haploidentical Bone Marrow/Kidney
Combined bone marrow and kidney transplantation using a haploidentical donor.
|
Eligibility| Ages Eligible for Study: | 18 Years to 70 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Patients ages 18-70
- Underlying hematological disorder which is potentially curable with allogeneic bone marrow transplantation. This includes, but is not limited to: acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), chronic myelogenous leukemia (CML), chronic lymphocytic leukemia (CLL), non-Hodgkin lymphoma (NHL), Hodgkin lymphoma, multiple myeloma (MM), myelodysplastic syndrome (MDS), AL amyloidosis, diamond blackfan anemia, myelofibrosis or other myeloproliferative disease, sickle cell anemia, and thalassemia.
- Existence of haploidentical first degree relative who passes standard donor evaluations for bone marrow and kidney donation
- LVEF > 40% as measured by echocardiography or MUGA
- FEV1, FVC, and DLCO > 50% of predicted as measured by standard PFTs
- Total bilirubin < 2.0 (unless diagnosis of Gilbert's or hemolysis is made) and AST, ALT, alkaline phosphatase all < 5x institutions upper limit of normal
- ABO compatibility in the host vs. graft direction
- Men and women of reproductive potential must agree to use a reliable method of birth control during the treatment, and women should do so for a period of 1 year following the transplant.
- Participants should be on dialysis or have an estimated or measured CrCl < 35 ml/min
- Life expectancy greater than six months.
- Recipient ability to understand and provide informed consent
Exclusion Criteria:
- Active serious infection
- Participation in other investigational drug use at the time of enrollment
- Contraindication to therapy with any one of the proposed agents (e.g., history of allergy to rabbit serum in ATG)
- Serologic positivity for HIV, HCV, or HbsAg positivity
- ABO blood group incompatibility in the host-vs-graft direction
- Active serious infection
Contacts and Locations| Contact: Yi-Bin A Chen, M.D. | 617-724-1124 ext 2 | ychen6@partners.org |
| Contact: Candice J Del Rio, R.N. | 617-726-6034 | cdelrio@partners.org |
| United States, Massachusetts | |
| Massachusetts General Hospital | Recruiting |
| Boston, Massachusetts, United States, 02114 | |
| Contact: Yi-Bin A Chen, M.D. 617-724-1124 ext 2 ychen6@partners.org | |
| Contact: Candice J Del Rio, R.N. 617-726-6034 cdelrio@partners.org | |
| Principal Investigator: Yi-Bin Chen, MD | |
| Principal Investigator: | Yi-Bin A Chen, M.D. | Director of Clinical Research, Massachusetts General Hospital Bone Marrow Transplant Program |
More Information
No publications provided
| Responsible Party: | Yi-Bin A. Chen, MD, Director of Clinical Research, Massachusetts General Hospital |
| ClinicalTrials.gov Identifier: | NCT01758042 History of Changes |
| Other Study ID Numbers: | 2012P001355 |
| Study First Received: | December 17, 2012 |
| Last Updated: | December 23, 2012 |
| Health Authority: | United States: Data and Safety Monitoring Board United States: Institutional Review Board |
Keywords provided by Massachusetts General Hospital:
|
Kidney Chronic Kidney Disease CKD Bone Marrow Bone Marrow Transplant BMT Leukemia |
AML ALL CML CLL MM NHL MDS |
Additional relevant MeSH terms:
|
Primary Myelofibrosis Amyloidosis Anemia Anemia, Aplastic Anemia, Sickle Cell Autoimmune Diseases Hematologic Diseases Hodgkin Disease Kidney Diseases Leukemia Leukemia, Lymphocytic, Chronic, B-Cell Leukemia, Lymphoid Precursor Cell Lymphoblastic Leukemia-Lymphoma Leukemia, Myeloid, Acute Leukemia, Myeloid |
Leukemia, Myelogenous, Chronic, BCR-ABL Positive Lymphoma Lymphoma, Non-Hodgkin Multiple Myeloma Neoplasms, Plasma Cell Myelodysplastic Syndromes Preleukemia Myeloproliferative Disorders Thalassemia Anemia, Diamond-Blackfan Renal Insufficiency, Chronic Kidney Failure, Chronic Bone Marrow Diseases Proteostasis Deficiencies Metabolic Diseases |
ClinicalTrials.gov processed this record on May 19, 2013