Trial Outcomes & Findings for A Cohort Study of Operationally Tolerant Allograft Recipients (NCT NCT02743793)

NCT ID: NCT02743793

Last Updated: 2021-09-21

Results Overview

A participant is considered to have operational tolerance if they are off all immunosuppression medication while continuing to have stable allograft function and no rejection. For these participants, the date of operational tolerance is considered as 52 weeks from the participant's last documented dose of immunosuppression. A participant will be considered to have lost operational tolerance if they experience rejection, have loss of stable allograft function (in the absence of confounding factors), or restart immunosuppression. The endpoint was analyzed using Kaplan-Meier survival estimate and associated two-sided 95% confidence interval, using a delayed-entry model with left-truncation of survival times by censoring the time from achieving operational tolerance at the time of study enrollment. The survival estimate is at the time of the last of event.

Recruitment status

TERMINATED

Target enrollment

41 participants

Primary outcome timeframe

From operational tolerance to ITN063ST study completion, assessed up to 60 months

Results posted on

2021-09-21

Participant Flow

41 tolerant kidney and liver participants were recruited from June 2016 through February 2020 along three main pathways 1) from current and past Immune Tolerance Network (ITN) trials, 2) by ITN affiliation investigators, academic community transplant physicians, and through outreach to transplant affinity groups, and 3) from the general transplant community through media channels.

Informed consent was obtained from potentially eligible individuals who underwent a screening visit to determine eligibility.

Participant milestones

Participant milestones
Measure
Enrolled, Not Eligible
These participants were consented and enrolled into the study, but did not meet eligibility criteria.
Accrued
These participations met all eligibility criteria and completed at least one mechanistic sample collection.
Overall Study
STARTED
5
36
Overall Study
COMPLETED
0
0
Overall Study
NOT COMPLETED
5
36

Reasons for withdrawal

Reasons for withdrawal
Measure
Enrolled, Not Eligible
These participants were consented and enrolled into the study, but did not meet eligibility criteria.
Accrued
These participations met all eligibility criteria and completed at least one mechanistic sample collection.
Overall Study
Death
1
1
Overall Study
Withdrawal by Subject
0
1
Overall Study
Screen Failure
4
0
Overall Study
No Primary Care Physician
0
1
Overall Study
Travel/remote visits could not be performed at nursing facility
0
1
Overall Study
Study stopped due to COVID-19 pandemic
0
32

Baseline Characteristics

Number of participants analyzed based on number of participants with data available.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Enrolled, Not Eligible
n=5 Participants
These participants were consented and enrolled into the study, but did not meet eligibility criteria.
Accrued
n=36 Participants
These participations met all eligibility criteria and completed at least one mechanistic sample collection.
Total
n=41 Participants
Total of all reporting groups
Age, Categorical
<=18 years
2 Participants
n=5 Participants
9 Participants
n=36 Participants
11 Participants
n=41 Participants
Age, Categorical
Between 18 and 65 years
1 Participants
n=5 Participants
21 Participants
n=36 Participants
22 Participants
n=41 Participants
Age, Categorical
>=65 years
2 Participants
n=5 Participants
6 Participants
n=36 Participants
8 Participants
n=41 Participants
Age, Continuous
39 Years
STANDARD_DEVIATION 29.4 • n=5 Participants
40 Years
STANDARD_DEVIATION 24.3 • n=36 Participants
40 Years
STANDARD_DEVIATION 24.5 • n=41 Participants
Sex: Female, Male
Female
0 Participants
n=5 Participants
20 Participants
n=36 Participants
20 Participants
n=41 Participants
Sex: Female, Male
Male
5 Participants
n=5 Participants
16 Participants
n=36 Participants
21 Participants
n=41 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
0 Participants
n=5 Participants
0 Participants
n=36 Participants
0 Participants
n=41 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
5 Participants
n=5 Participants
36 Participants
n=36 Participants
41 Participants
n=41 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
0 Participants
n=36 Participants
0 Participants
n=41 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
0 Participants
n=36 Participants
0 Participants
n=41 Participants
Race (NIH/OMB)
Asian
0 Participants
n=5 Participants
1 Participants
n=36 Participants
1 Participants
n=41 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=5 Participants
0 Participants
n=36 Participants
0 Participants
n=41 Participants
Race (NIH/OMB)
Black or African American
0 Participants
n=5 Participants
1 Participants
n=36 Participants
1 Participants
n=41 Participants
Race (NIH/OMB)
White
5 Participants
n=5 Participants
33 Participants
n=36 Participants
38 Participants
n=41 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=5 Participants
0 Participants
n=36 Participants
0 Participants
n=41 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
1 Participants
n=36 Participants
1 Participants
n=41 Participants
Region of Enrollment
United States
5 participants
n=5 Participants
36 participants
n=36 Participants
41 participants
n=41 Participants
Duration of operational tolerance at time of enrollment.
7.6 years
STANDARD_DEVIATION 5.07 • n=5 Participants
8.1 years
STANDARD_DEVIATION 7.53 • n=36 Participants
8.1 years
STANDARD_DEVIATION 7.32 • n=41 Participants
Alanine aminotransferase (ALT)
68.0 U/L
STANDARD_DEVIATION NA • n=1 Participants • Number of participants analyzed based on number of participants with data available.
24.5 U/L
STANDARD_DEVIATION 11.98 • n=26 Participants • Number of participants analyzed based on number of participants with data available.
26.1 U/L
STANDARD_DEVIATION 14.42 • n=27 Participants • Number of participants analyzed based on number of participants with data available.
Gamma glutamyltransferase (GGT)
18 U/L
STANDARD_DEVIATION NA • n=1 Participants • Number of participants analyzed based on number of participants with data available.
32.4 U/L
STANDARD_DEVIATION 49.33 • n=25 Participants • Number of participants analyzed based on number of participants with data available.
31.8 U/L
STANDARD_DEVIATION 48.42 • n=26 Participants • Number of participants analyzed based on number of participants with data available.
Creatinine
2.6 mg/dL
STANDARD_DEVIATION NA • n=1 Participants • Number of participants analyzed based on number of participants with data available.
1.3 mg/dL
STANDARD_DEVIATION 0.48 • n=10 Participants • Number of participants analyzed based on number of participants with data available.
1.4 mg/dL
STANDARD_DEVIATION 0.60 • n=11 Participants • Number of participants analyzed based on number of participants with data available.

PRIMARY outcome

Timeframe: From operational tolerance to ITN063ST study completion, assessed up to 60 months

Population: Participants who met all eligibility criteria and completed at least one mechanistic sample collection.

A participant is considered to have operational tolerance if they are off all immunosuppression medication while continuing to have stable allograft function and no rejection. For these participants, the date of operational tolerance is considered as 52 weeks from the participant's last documented dose of immunosuppression. A participant will be considered to have lost operational tolerance if they experience rejection, have loss of stable allograft function (in the absence of confounding factors), or restart immunosuppression. The endpoint was analyzed using Kaplan-Meier survival estimate and associated two-sided 95% confidence interval, using a delayed-entry model with left-truncation of survival times by censoring the time from achieving operational tolerance at the time of study enrollment. The survival estimate is at the time of the last of event.

Outcome measures

Outcome measures
Measure
Accrued
n=36 Participants
These participants met all eligibility criteria and completed at least one mechanistic sample collection.
Time to Loss of Operational Tolerance
6.6 Years to loss of operational tolerance
Standard Deviation 5.38

SECONDARY outcome

Timeframe: Day 0 (Study Enrollment) to Study Completion (Up to 3.7 years)

Time to development of either: * de novo HLA antibodies (anti-HLA Ab) post-transplant. Alloantibody is defined as an antibody produced following the introduction of an alloantigen into the system of an individual lacking that particular antigen; OR * Donor Specific Antibodies (DSA), a newly developed alloantibody that is against the donor organ. Alloantibodies are important mediators of acute and chronic rejection. \*\*These data are not yet available.\*\*

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Day 0 (Study Enrollment) to Study Completion (Up to 3.7 years)

Population: Participants who met all eligibility criteria and completed at least one mechanistic sample collection.

Number of participants with acute rejection, steroid resistant rejection and/or chronic rejection. Criteria employed for acute and chronic rejection: Banff guidelines.

Outcome measures

Outcome measures
Measure
Accrued
n=36 Participants
These participants met all eligibility criteria and completed at least one mechanistic sample collection.
Number of Participants With Biopsy-Proven or Clinical Acute Rejection, Steroid Resistant Rejection and/or Chronic Rejection
0 Participants

SECONDARY outcome

Timeframe: Day 0 (Study Enrollment) to Study Completion (Up to 3.7 years)

Population: Participants who met all eligibility criteria and completed at least one mechanistic sample collection.

A participant is considered to have graft loss when the participant is retransplanted with another donor kidney, relisted for transplantation, or dies with a non-functioning graft.

Outcome measures

Outcome measures
Measure
Accrued
n=36 Participants
These participants met all eligibility criteria and completed at least one mechanistic sample collection.
Number of Participants With Graft Loss, Not Including Death With a Functioning Graft
0 Participants

Adverse Events

Enrolled, Not Eligible

Serious events: 0 serious events
Other events: 0 other events
Deaths: 1 deaths

Accrued

Serious events: 0 serious events
Other events: 0 other events
Deaths: 1 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Director, Clinical Research Operations Program

DAIT/NIAID

Phone: 13015947669

Results disclosure agreements

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