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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01758042
Recruitment Status : Recruiting
First Posted : December 31, 2012
Last Update Posted : March 15, 2023
Sponsor:
Information provided by (Responsible Party):
Yi-Bin A. Chen, MD, Massachusetts General Hospital

Tracking Information
First Submitted Date  ICMJE December 17, 2012
First Posted Date  ICMJE December 31, 2012
Last Update Posted Date March 15, 2023
Study Start Date  ICMJE November 2012
Estimated Primary Completion Date July 2025   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 23, 2012)		 Number of patients who die of treatment-related complications. [ Time Frame: 100 days and 1 year post transplant ]
Assess safety of haploidentical combined bone marrow and kidney transplantation as measured treatment related mortality.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 23, 2012)		 Number of patients with acute and delayed renal allograft rejection [ Time Frame: 2 years post-transplant ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures
 (submitted: October 3, 2016)
  • Number of patients who are able to discontinue immunosuppressive therapy by one year post transplant [ Time Frame: one year post transplant ]
  • Number of patients who develop acute and chronic graft versus host disease (GVHD). [ Time Frame: post transplant ]
  • Number of patients who relapse from their underlying hematological disease [ Time Frame: 6 months, 1 year, and 2 years post transplant. ]
Original Other Pre-specified Outcome Measures
 (submitted: December 23, 2012)
  • Number of patients who are able to discontinue immunosuppressive therapy by one year post transplant [ Time Frame: one year post transplant ]
  • Number of patients who develp acute and chronic graft versus host disease (GVHD). [ Time Frame: post transplant ]
  • Number of patients who relapse from their underlying hematological disease [ Time Frame: 6 months, 1 year, and 2 years post transplant. ]
 
Descriptive Information
Brief Title  ICMJE Bone Marrow and Kidney Transplant for Patients With Chronic Kidney Disease and Blood Disorders
Official Title  ICMJE Combined Haploidentical Reduced Intensity Bone Marrow and Kidney Transplantation for Patients With Chronic Kidney Disease and Advanced Hematological Disorders
Brief Summary

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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • 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
Intervention  ICMJE Procedure: Haploidentical Bone Marrow/Kidney
Combined bone marrow and kidney transplantation using a haploidentical donor.
Study Arms  ICMJE Haploidentical Bone Marrow/Kidney
Single Arm Study
Intervention: Procedure: Haploidentical Bone Marrow/Kidney
Publications * Chen YB, Elias N, Heher E, McCune JS, Collier K, Li S, Del Rio C, El-Jawahri A, Williams W, Tolkoff-Rubin N, Fishman JA, McAfee S, Dey BR, DeFilipp Z, O'Donnell PV, Cosimi AB, Sachs D, Kawai T, Spitzer TR. Haploidentical hematopoietic cell and kidney transplantation for hematological malignancies and end-stage renal failure. Blood. 2019 Jul 11;134(2):211-215. doi: 10.1182/blood.2019000775. Epub 2019 May 31.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: December 23, 2012)		 10
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE July 2027
Estimated Primary Completion Date July 2025   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 70 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
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
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01758042
Other Study ID Numbers  ICMJE 2012P001355
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Current Responsible Party Yi-Bin A. Chen, MD, Massachusetts General Hospital
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Massachusetts General Hospital
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Yi-Bin A Chen, M.D. Director of Clinical Research, Massachusetts General Hospital Bone Marrow Transplant Program
PRS Account Massachusetts General Hospital
Verification Date March 2023

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP