Trial Outcomes & Findings for Combined Bone Marrow and Renal Transplantation for Hematologic Disorders With End Stage Renal Disease (NCT NCT02158052)

NCT ID: NCT02158052

Last Updated: 2022-12-13

Results Overview

The Primary Outcome is: the incidence of renal allograft rejection at 6 months post-transplant

• Recruitment status: COMPLETED
• Study phase: NA
• Target enrollment: 2 participants
• Primary outcome timeframe: 6 Months
• Results posted on: 2022-12-13

Participant Flow

Participant milestones

Measure	Bone Marrow and Kidney RECIPIENTS Single arm combined bone marrow and kidney transplantation Tacrolimus: Tacrolimus starting on Day -1 Equine Anti-thymocyte globulin: 20 mg/kg IV on Days -3, -1, +1, +3 Kidney transplant from a related donor: On Day 0 the renal transplant is performed according to standard surgical techniques. Bone marrow transplant from a related donor: Donor bone marrow (\> 2 x 10e8 nucleated cells/kg of recipient body weight) is prepared for infusion according to the standard procedure. The infusion begins in the operating room as soon as the vascular anastomosis of the renal allograft has been completed. Total body irradiation 400 centigray (200 cGy X 2): On transplant day -1	Bone Marrow and Kidney DONORS Donor nephrectomy and bone marrow harvest (\> 2X10e8/kg nucleated cells/kg recipient body weight) on Day 0
Overall Study STARTED	1	1
Overall Study COMPLETED	1	1
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Combined Bone Marrow and Renal Transplantation for Hematologic Disorders With End Stage Renal Disease

Baseline characteristics by cohort

Measure	Bone Marrow and Kidney RECIPIENT n=1 Participants Single arm combined bone marrow and kidney transplantation Tacrolimus: Tacrolimus starting on Day -1 Equine Anti-thymocyte globulin: 20 mg/kg IV on Days -3, -1, +1, +3 Kidney transplant from a related donor: On Day 0 the renal transplant is performed according to standard surgical techniques. Bone marrow transplant from a related donor: Donor bone marrow (\> 2 x 10e8 nucleated cells/kg of recipient body weight) is prepared for infusion according to the standard procedure. The infusion begins in the operating room as soon as the vascular anastomosis of the renal allograft has been completed. Total body irradiation 400 centigray (200 cGy X 2): On transplant day -1	Bone Marrow and Kidney DONOR n=1 Participants Kidney transplant from a related donor: On Day 0 the renal transplant is performed according to standard surgical techniques. Bone marrow transplant from a related donor: Donor bone marrow (\> 2 x 10e8 nucleated cells/kg of recipient body weight) is prepared for infusion according to the standard procedure.	Total n=2 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Continuous	53 years n=5 Participants	46 years n=7 Participants	49.5 years n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment United States	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants

PRIMARY outcome

Timeframe: 6 Months

Population: One bone marrow/kidney transplant recipient was enrolled and treated on this trial.

The Primary Outcome is: the incidence of renal allograft rejection at 6 months post-transplant

Outcome measures

Measure

Bone Marrow and Kidney RECIPIENTS n=1 Participants Single arm combined bone marrow and kidney transplantation Tacrolimus: Tacrolimus starting on Day -1 Equine Anti-thymocyte globulin: 20 mg/kg IV on Days -3, -1, +1, +3 Kidney transplant from a related donor: On Day 0 the renal transplant is performed according to standard surgical techniques. Bone marrow transplant from a related donor: Donor bone marrow (\> 2 x 10e8 nucleated cells/kg of recipient body weight) is prepared for infusion according to the standard procedure. The infusion begins in the operating room as soon as the vascular anastomosis of the renal allograft has been completed. Total body irradiation 400 centigray (200 cGy X 2): On transplant day -1

Number of Participants Without Renal Allograft Rejection at 6 Months Post-transplant

1 Participants

SECONDARY outcome

Timeframe: 3 years

Population: One bone marrow/kidney recipient was enrolled and treated on this trial.

This is measurement is summarized via bone marrow biopsy results and blood assays through 3 years.

Anti-tumor response is assessed according to Center for International Blood and Marrow Transplant Research (CIBMTR.org) criteria.

For multiple myeloma, based on assessment of serum and urine immunofixation, the presence or absence of soft tissue plasmacytomas and analyses of bone marrow aspirate and biopsy samples, response categories include stringent complete remission (sCR), complete remission (CR), near CR (nCR) very good partial response (VGPR), partial response (PR), stable disease (SD), and progressive disease (PD).

For AL amyloidosis, based on assessment of serum and urine immunofixation and serum free light chain ratio, hematologic response categories include complete response, very good partial response, partial response, no response/stable disease and progressive disease. Organ responses are also assessed according to organ specific criteria.

Outcome measures

Measure

Bone Marrow and Kidney RECIPIENTS n=1 Participants Single arm combined bone marrow and kidney transplantation Tacrolimus: Tacrolimus starting on Day -1 Equine Anti-thymocyte globulin: 20 mg/kg IV on Days -3, -1, +1, +3 Kidney transplant from a related donor: On Day 0 the renal transplant is performed according to standard surgical techniques. Bone marrow transplant from a related donor: Donor bone marrow (\> 2 x 10e8 nucleated cells/kg of recipient body weight) is prepared for infusion according to the standard procedure. The infusion begins in the operating room as soon as the vascular anastomosis of the renal allograft has been completed. Total body irradiation 400 centigray (200 cGy X 2): On transplant day -1

Anti-Tumor Response Rate

1 Participants

Adverse Events

Bone Marrow and Kidney RECIPIENT

Serious events: 1 serious events

Other events: 1 other events

Deaths: 0 deaths

Bone Marrow and Kidney DONOR

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Measure	Bone Marrow and Kidney RECIPIENT n=1 participants at risk Single arm combined bone marrow and kidney transplantation Tacrolimus: Tacrolimus starting on Day -1 Equine Anti-thymocyte globulin: 20 mg/kg IV on Days -3, -1, +1, +3 Kidney transplant from a related donor: On Day 0 the renal transplant is performed according to standard surgical techniques. Bone marrow transplant from a related donor: Donor bone marrow (\> 2 x 10e8 nucleated cells/kg of recipient body weight) is prepared for infusion according to the standard procedure. The infusion begins in the operating room as soon as the vascular anastomosis of the renal allograft has been completed. Total body irradiation 400 centigray (200 cGy X 2): On transplant day -1	Bone Marrow and Kidney DONOR n=1 participants at risk Kidney transplant from a related donor: On Day 0 the renal transplant is performed according to standard surgical techniques. Bone marrow transplant from a related donor: Donor bone marrow (\> 2 x 10e8 nucleated cells/kg of recipient body weight) is prepared for infusion according to the standard procedure.
Renal and urinary disorders Acute kidney injury	100.0% 1/1 • Number of events 1 • 6 years Adverse event information was assessed by study investigators and entered into an internal Bone Marrow Transplant database.	0.00% 0/1 • 6 years Adverse event information was assessed by study investigators and entered into an internal Bone Marrow Transplant database.
Blood and lymphatic system disorders Hematopoietic graft rejection	100.0% 1/1 • Number of events 1 • 6 years Adverse event information was assessed by study investigators and entered into an internal Bone Marrow Transplant database.	0.00% 0/1 • 6 years Adverse event information was assessed by study investigators and entered into an internal Bone Marrow Transplant database.
Respiratory, thoracic and mediastinal disorders Aspergillosis	100.0% 1/1 • Number of events 1 • 6 years Adverse event information was assessed by study investigators and entered into an internal Bone Marrow Transplant database.	0.00% 0/1 • 6 years Adverse event information was assessed by study investigators and entered into an internal Bone Marrow Transplant database.

Other adverse events

Measure	Bone Marrow and Kidney RECIPIENT n=1 participants at risk Single arm combined bone marrow and kidney transplantation Tacrolimus: Tacrolimus starting on Day -1 Equine Anti-thymocyte globulin: 20 mg/kg IV on Days -3, -1, +1, +3 Kidney transplant from a related donor: On Day 0 the renal transplant is performed according to standard surgical techniques. Bone marrow transplant from a related donor: Donor bone marrow (\> 2 x 10e8 nucleated cells/kg of recipient body weight) is prepared for infusion according to the standard procedure. The infusion begins in the operating room as soon as the vascular anastomosis of the renal allograft has been completed. Total body irradiation 400 centigray (200 cGy X 2): On transplant day -1	Bone Marrow and Kidney DONOR n=1 participants at risk Kidney transplant from a related donor: On Day 0 the renal transplant is performed according to standard surgical techniques. Bone marrow transplant from a related donor: Donor bone marrow (\> 2 x 10e8 nucleated cells/kg of recipient body weight) is prepared for infusion according to the standard procedure.
Renal and urinary disorders Perinephric hematoma	100.0% 1/1 • Number of events 1 • 6 years Adverse event information was assessed by study investigators and entered into an internal Bone Marrow Transplant database.	0.00% 0/1 • 6 years Adverse event information was assessed by study investigators and entered into an internal Bone Marrow Transplant database.
Gastrointestinal disorders Abdominal pain	100.0% 1/1 • Number of events 1 • 6 years Adverse event information was assessed by study investigators and entered into an internal Bone Marrow Transplant database.	0.00% 0/1 • 6 years Adverse event information was assessed by study investigators and entered into an internal Bone Marrow Transplant database.

Additional Information

Thomas Spitzer, MD

Massachusetts General Hospital

Phone: 6177241124

Email: tspitzer@mgh.harvard.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place