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

NCT ID: NCT01758042

Last Updated: 2023-03-15

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

NA

Total Enrollment

10 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-11-30

Study Completion Date

2027-07-31

Brief Summary

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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

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Conditions

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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

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Haploidentical Bone Marrow/Kidney

Single Arm Study

Group Type OTHER

Haploidentical Bone Marrow/Kidney

Intervention Type PROCEDURE

Combined bone marrow and kidney transplantation using a haploidentical donor.

Interventions

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Haploidentical Bone Marrow/Kidney

Combined bone marrow and kidney transplantation using a haploidentical donor.

Intervention Type PROCEDURE

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Maximum Eligible Age

70 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Massachusetts General Hospital

OTHER

Sponsor Role lead

Responsible Party

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Yi-Bin A. Chen, MD

Director of Clinical Research

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Yi-Bin A Chen, M.D.

Role: PRINCIPAL_INVESTIGATOR

Director of Clinical Research, Massachusetts General Hospital Bone Marrow Transplant Program

Locations

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Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Yi-Bin A Chen, M.D.

Role: CONTACT

617-724-1124 ext. 2

Candice Del Rio, RN

Role: CONTACT

617-726-6034

Facility Contacts

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Yi-Bin A Chen, M.D.

Role: primary

617-724-1124 ext. 2

Candice Delrio, RN

Role: backup

617-726-6034

References

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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.

Reference Type DERIVED
PMID: 31151984 (View on PubMed)

Other Identifiers

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2012P001355

Identifier Type: -

Identifier Source: org_study_id

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