Bone Marrow and Kidney Transplant for Patients With Chronic Kidney Disease and Blood Disorders (BMT)

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2014 by Massachusetts General Hospital
Sponsor:
Information provided by (Responsible Party):
Yi-Bin A. Chen, MD, Massachusetts General Hospital
ClinicalTrials.gov Identifier:
NCT01758042
First received: December 17, 2012
Last updated: February 11, 2014
Last verified: February 2014
  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

Resource links provided by NLM:


Further study details as provided by Massachusetts General Hospital:

Primary Outcome Measures:
  • 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.


Secondary Outcome Measures:
  • Number of patients with acute and delayed renal allograft rejection [ Time Frame: 2 years post-transplant ] [ Designated as safety issue: Yes ]

Other Outcome Measures:
  • 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: July 2016
Estimated Primary Completion Date: July 2016 (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
Criteria

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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01758042

Contacts
Contact: Yi-Bin A Chen, M.D. 617-724-1124 ext 2 ychen6@partners.org
Contact: Candice Del Rio, RN 617-726-6034 cdelrio@partners.org

Locations
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 Delrio, RN    617-726-6034    cdelrio@partners.org   
Principal Investigator: Yi-Bin Chen, MD         
Sponsors and Collaborators
Massachusetts General Hospital
Investigators
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: February 11, 2014
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
Renal Insufficiency, Chronic
Anemia, Diamond-Blackfan
Bone Marrow Diseases
Proteostasis Deficiencies
Metabolic Diseases
Anemia, Hemolytic, Congenital

ClinicalTrials.gov processed this record on August 18, 2014