Trial Outcomes & Findings for Safety and Efficacy of Desensitization Therapy in Sensitized Participants Awaiting Heart Transplantation (NCT NCT01769443)
NCT ID: NCT01769443
Last Updated: 2015-11-11
Results Overview
* Death, * Removal from the transplant waiting list for any reason except improvement of cardiac function, * Initiation of any mechanical circulatory support device, * Severe infection requiring intravenous antibiotics, * Cerebral vascular accident, * Acute renal failure requiring dialysis.
TERMINATED
PHASE2
2 participants
At transplant, or 90 days post-randomization, whichever occurs first
2015-11-11
Participant Flow
The study planned to enroll 80 participants; however, the decision to terminate the study was made due to the very slow rate of participant accrual and the inability to meet the recruitment goal within the funding period. Only 2 participants were enrolled at one site before study recruitment status changed to "Active, not recruiting."
Participant milestones
| Measure |
No Desensitization
No desensitization therapy pre-transplantation
|
Desensitization
Plasmapheresis with concomitant bortezomib.
* Plasmapheresis will be given for 3 consecutive days (treatment days 0, 1 and 2) within 2 weeks of Status I listing for heart transplantation. Plasmapheresis is a procedure that involves the extracorporeal separation of plasma from cellular blood components by centrifugation or membrane filtration, which removes the alloantibody. The plasma depleted blood is reconstituted with exogenous fresh-frozen plasma or albumin solution which is then infused back into the patient.
* Four doses of bortezomib (1.3 mg/m\^2) were administered intravenously on treatment days 0, 3, 7 and 10. The first dose was given between 4-8 hours after the first plasmapheresis session was completed and there was at least 96 hours between the second and third dose of bortezomib
|
|---|---|---|
|
Overall Study
STARTED
|
1
|
1
|
|
Overall Study
COMPLETED
|
0
|
1
|
|
Overall Study
NOT COMPLETED
|
1
|
0
|
Reasons for withdrawal
| Measure |
No Desensitization
No desensitization therapy pre-transplantation
|
Desensitization
Plasmapheresis with concomitant bortezomib.
* Plasmapheresis will be given for 3 consecutive days (treatment days 0, 1 and 2) within 2 weeks of Status I listing for heart transplantation. Plasmapheresis is a procedure that involves the extracorporeal separation of plasma from cellular blood components by centrifugation or membrane filtration, which removes the alloantibody. The plasma depleted blood is reconstituted with exogenous fresh-frozen plasma or albumin solution which is then infused back into the patient.
* Four doses of bortezomib (1.3 mg/m\^2) were administered intravenously on treatment days 0, 3, 7 and 10. The first dose was given between 4-8 hours after the first plasmapheresis session was completed and there was at least 96 hours between the second and third dose of bortezomib
|
|---|---|---|
|
Overall Study
Study closure
|
1
|
0
|
Baseline Characteristics
Safety and Efficacy of Desensitization Therapy in Sensitized Participants Awaiting Heart Transplantation
Baseline characteristics by cohort
| Measure |
No Desensitization
n=1 Participants
No desensitization therapy pre-transplantation
|
Desensitization
n=1 Participants
Plasmapheresis with concomitant bortezomib.
* Plasmapheresis will be given for 3 consecutive days (treatment days 0, 1 and 2) within 2 weeks of Status I listing for heart transplantation. Plasmapheresis is a procedure that involves the extracorporeal separation of plasma from cellular blood components by centrifugation or membrane filtration, which removes the alloantibody. The plasma depleted blood is reconstituted with exogenous fresh-frozen plasma or albumin solution which is then infused back into the patient.
* Four doses of bortezomib (1.3 mg/m\^2) were administered intravenously on treatment days 0, 3, 7 and 10. The first dose was given between 4-8 hours after the first plasmapheresis session was completed and there was at least 96 hours between the second and third dose of bortezomib
|
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
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
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
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 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
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 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
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 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: At transplant, or 90 days post-randomization, whichever occurs firstPopulation: No analyses were performed due to slow enrollment and early study closure.
* Death, * Removal from the transplant waiting list for any reason except improvement of cardiac function, * Initiation of any mechanical circulatory support device, * Severe infection requiring intravenous antibiotics, * Cerebral vascular accident, * Acute renal failure requiring dialysis.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: At transplant, or 1 year post-randomization, whichever occurs firstPopulation: No analyses were performed due to slow enrollment and early study closure.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: At transplant, or 1 year post-randomization, whichever occurs firstPopulation: No analyses were performed due to slow enrollment and early study closure.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: At transplant, or 1 year post-randomization, whichever occurs firstPopulation: No analyses were performed due to slow enrollment and early study closure.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: At transplant, or 1 year post-randomization, whichever occurs firstPopulation: No analyses were performed due to slow enrollment and early study closure.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: At transplant, or 1 year post-randomization, whichever occurs firstPopulation: No analyses were performed due to slow enrollment and early study closure.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: At transplant, or 1 year post-randomization, whichever occurs firstPopulation: No analyses were performed due to slow enrollment and early study closure.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: At transplant, or 1 year post-randomization, whichever occurs firstPopulation: No analyses were performed due to slow enrollment and early study closure.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: At transplant, or 1 year post-randomization, whichever occurs firstPopulation: No analyses were performed due to slow enrollment and early study closure.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: At transplant, or 1 year post-randomization, whichever occurs firstPopulation: No analyses were performed due to slow enrollment and early study closure.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 24 and 52 weeks post-transplantationPopulation: No analyses were performed due to slow enrollment and early study closure.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 24 and 52 weeks post-transplantationPopulation: No analyses were performed due to slow enrollment and early study closure.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 24 and 52 weeks post-transplantationPopulation: No analyses were performed due to slow enrollment and early study closure.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 24 and 52 weeks:Population: No analyses were performed due to slow enrollment and early study closure.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 24 and 52 weeks post-transplantationPopulation: No analyses were performed due to slow enrollment and early study closure.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 24 and 52 weeks post-transplantationPopulation: No analyses were performed due to slow enrollment and early study closure.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 24 and 52 weeks post-transplantationPopulation: No analyses were performed due to slow enrollment and early study closure.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 24 and 52 weeks post-transplantationPopulation: No analyses were performed due to slow enrollment and early study closure.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 24 and 52 weeks post-transplantationPopulation: No analyses were performed due to slow enrollment and early study closure.
Rejection is defined as follows: * Biopsy proven acute rejection (BPAR) of any grade (cellular rejection per 2004 ISHLT \[International Society of Heart and Lung Transplantation\] grading scale), * BPAR (individual grades), * BPAR (Biopsy Proven Acute Rejection) \> 2R * antibody mediated rejection (AMR), * Any treated rejection, * Rejection associated with hemodynamic compromise (HDC).
Outcome measures
Outcome data not reported
Adverse Events
No Desensitization
Desensitization
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
No Desensitization
n=1 participants at risk
No desensitization therapy pre-transplantation
|
Desensitization
n=1 participants at risk
Plasmapheresis with concomitant bortezomib.
* Plasmapheresis will be given for 3 consecutive days (treatment days 0, 1 and 2) within 2 weeks of Status I listing for heart transplantation. Plasmapheresis is a procedure that involves the extracorporeal separation of plasma from cellular blood components by centrifugation or membrane filtration, which removes the alloantibody. The plasma depleted blood is reconstituted with exogenous fresh-frozen plasma or albumin solution which is then infused back into the patient.
* Four doses of bortezomib (1.3 mg/m\^2) were administered intravenously on treatment days 0, 3, 7 and 10. The first dose was given between 4-8 hours after the first plasmapheresis session was completed and there was at least 96 hours between the second and third dose of bortezomib
|
|---|---|---|
|
Injury, poisoning and procedural complications
compression fracture
|
100.0%
1/1 • Number of events 1 • Adverse events were collected from the time of the first protocol mandated procedure until the study completion, or until 30 days after the subject prematurely withdraws from the study.
The participant randomized to the control arm ("No Desensitization Therapy") completed week 24 (6 months) of post-transplantation evaluations. The participant assigned to the Investigational Arm ("Desensitization Therapy") completed week 52 (12 months) of post-transplantation follow-up.
|
0.00%
0/1 • Adverse events were collected from the time of the first protocol mandated procedure until the study completion, or until 30 days after the subject prematurely withdraws from the study.
The participant randomized to the control arm ("No Desensitization Therapy") completed week 24 (6 months) of post-transplantation evaluations. The participant assigned to the Investigational Arm ("Desensitization Therapy") completed week 52 (12 months) of post-transplantation follow-up.
|
Additional Information
Director, Clinical Research Operations Program
DAIT/NIAID
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place