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

March 13, 2023 updated by: Yi-Bin A. Chen, MD, Massachusetts General Hospital

Combined Haploidentical Reduced Intensity Bone Marrow and Kidney Transplantation for Patients With Chronic Kidney Disease and Advanced Hematological Disorders

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Anticipated)

10

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • United States
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Recruiting
        • Massachusetts General Hospital
        • Contact:
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 70 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Other: Haploidentical Bone Marrow/Kidney
Single Arm Study
Procedure: Haploidentical Bone Marrow/Kidney
Combined bone marrow and kidney transplantation using a haploidentical donor.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
100 days and 1 year post transplant

Secondary Outcome Measures

Outcome Measure
Time Frame
Number of patients with acute and delayed renal allograft rejection
Time Frame: 2 years post-transplant
2 years post-transplant

Other Outcome Measures

Outcome Measure
Time Frame
Number of patients who are able to discontinue immunosuppressive therapy by one year post transplant
Time Frame: one year post transplant
one year post transplant
Number of patients who develop acute and chronic graft versus host disease (GVHD).
Time Frame: post transplant
post transplant
Number of patients who relapse from their underlying hematological disease
Time Frame: 6 months, 1 year, and 2 years post transplant.
6 months, 1 year, and 2 years post transplant.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Massachusetts General Hospital

Investigators

  • Principal Investigator: Yi-Bin A Chen, M.D., Director of Clinical Research, Massachusetts General Hospital Bone Marrow Transplant Program

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

November 1, 2012

Primary Completion (Anticipated)

July 1, 2025

Study Completion (Anticipated)

July 1, 2027

Study Registration Dates

First Submitted

December 17, 2012

First Submitted That Met QC Criteria

December 23, 2012

First Posted (Estimate)

December 31, 2012

Study Record Updates

Last Update Posted (Actual)

March 15, 2023

Last Update Submitted That Met QC Criteria

March 13, 2023

Last Verified

March 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2012P001355

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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