Trial Outcomes & Findings for Efficacy of Low Dose, SubQ Interleukin-2 (IL-2) to Expand Endogenous Regulatory T-Cells in Liver Transplant Recipients (NCT NCT02739412)
NCT ID: NCT02739412
Last Updated: 2026-01-13
Results Overview
Peripheral Blood Mononuclear Cell Flow Cytometry
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
6 participants
Primary outcome timeframe
baseline, week 2, week 4, week8, week12
Results posted on
2026-01-13
Participant Flow
Participant milestones
| Measure |
Active Study Medication Arm
Single arm, prospective, open label efficacy and safety of a 4-weeks course of low dose IL-2 to expand TRegs in liver transplant recipients with stable liver function.
|
|---|---|
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Overall Study
STARTED
|
6
|
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Overall Study
COMPLETED
|
5
|
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Overall Study
NOT COMPLETED
|
1
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
1 patient withdrew from the study after 1 dose due to adverse event and was not included in the efficacy analysis.
Baseline characteristics by cohort
| Measure |
Open Label. Active Study Treatment Arm
n=6 Participants
IL-2 (Interleukin-2; Aldesleukin; Proleukin) administered daily as a single subcutaneous injection 0.30 MIU per meter squared body surface area for a duration of 4 weeks.
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|---|---|
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Age, Categorical
<=18 years
|
0 Participants
n=6 Participants
|
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Age, Categorical
Between 18 and 65 years
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6 Participants
n=6 Participants
|
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Age, Categorical
>=65 years
|
0 Participants
n=6 Participants
|
|
Age, Continuous
|
54 years
n=6 Participants
|
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Sex: Female, Male
Female
|
2 Participants
n=6 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=6 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
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0 Participants
n=6 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=6 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=6 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=6 Participants
|
|
Race (NIH/OMB)
White
|
4 Participants
n=6 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=6 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=6 Participants
|
|
Region of Enrollment
United States
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6 Participants
n=6 Participants
|
|
CD3T cells (%PBMC)
|
53.74 %PBMC
n=5 Participants • 1 patient withdrew from the study after 1 dose due to adverse event and was not included in the efficacy analysis.
|
|
CD4 T cells (% PBMC)
|
25.29 %PBMC
n=5 Participants • 1 patient withdrew from the study after 1 dose due to adverse event and was not included in the efficacy analysis.
|
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CD8 T cells (%PBMC)
|
28.05 %PBMC
n=5 Participants • 1 patient withdrew from the study after 1 dose due to adverse event and was not included in the efficacy analysis.
|
|
CD19 B cells (%PBMC)
|
4.84 %PBMC
n=5 Participants • 1 patient withdrew from the study after 1 dose due to adverse event and was not included in the efficacy analysis.
|
|
CD56 NK (%PBMC)
|
6.37 %PBMC
n=5 Participants • 1 patient withdrew from the study after 1 dose due to adverse event and was not included in the efficacy analysis.
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Monocyte/Dendritic cells (% PBMC)
|
12.82 %PBMC
n=5 Participants • 1 patient withdrew from the study after 1 dose due to adverse event and was not included in the efficacy analysis.
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NKT (%PBMC)
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13.56 %PBMC
n=5 Participants • 1 patient withdrew from the study after 1 dose due to adverse event and was not included in the efficacy analysis.
|
|
CD4Treg (% of PBMC)
|
0.44 %PBMC
n=5 Participants • 1 patient withdrew from the study after 1 dose due to adverse event and was not included in the efficacy analysis.
|
|
CD4 conventional T cells (% of CD4 cells)
|
97.34 %PBMC
n=5 Participants • 1 patient withdrew from the study after 1 dose due to adverse event and was not included in the efficacy analysis.
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CD4 Treg cells (% CD4 T cells)
|
2.13 %PBMC
n=5 Participants • 1 patient withdrew from the study after 1 dose due to adverse event and was not included in the efficacy analysis.
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Alanine aminotransferase (ALT)
|
19 units of ALT/L
n=6 Participants
|
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White Blood Cell count (WBC)
|
5.85 cells *10^9/L
n=6 Participants
|
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Platelet count
|
162,000 platelets /uL
n=6 Participants
|
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Hematocrit
|
39 %
n=6 Participants
|
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Time from transplant
|
2.85 years
n=6 Participants
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PRIMARY outcome
Timeframe: baseline, week 2, week 4, week8, week12Population: The efficacy analysis was performed on 5 patients. 1 patient withdrew from the study due to an adverse event before any efficacy data was obtained.6 patients were enrolled, 66% male with a median age of 54 years. Median time from transplant was 2.8 years. Five patients were on tacrolimus monotherapy and 1 patients was on tacrolimus and mycophenolate mofetil.
Peripheral Blood Mononuclear Cell Flow Cytometry
Outcome measures
| Measure |
Open Label. Active Study Treatment Arm
n=5 Participants
All patients received IL-2
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|---|---|
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Regulatory T-Cell Count
Baseline CD4Tred (%PBMC)
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0.44 percentage of PBMC
Interval 0.27 to 0.75
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Regulatory T-Cell Count
Wk2 CD4Treg (%PBMC)
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3.71 percentage of PBMC
Interval 2.95 to 4.48
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Regulatory T-Cell Count
Wk4 CD4Treg (%PBMC)
|
3.57 percentage of PBMC
Interval 2.53 to 3.59
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Regulatory T-Cell Count
Wk8 CD4Treg (%PBMC)
|
0.6 percentage of PBMC
Interval 0.42 to 1.37
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Regulatory T-Cell Count
Wk12 CD4Treg (%PBMC)
|
0.64 percentage of PBMC
Interval 0.44 to 1.28
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PRIMARY outcome
Timeframe: baseline, 2 weeks, 4 weeks, 8 weeks, 12 weeks% CD4 T Regs were measured at several time points after IL-2 administration.
Outcome measures
| Measure |
Open Label. Active Study Treatment Arm
n=5 Participants
All patients received IL-2
|
|---|---|
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% Increase in CD4 Tregs
Baseline CD4Treg (%CD4Tcells)
|
2.13 percentage of CD4 T cells
Interval 1.04 to 3.12
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% Increase in CD4 Tregs
Wk2 CD4Treg (%CD4Tcells)
|
13.15 percentage of CD4 T cells
Interval 11.53 to 19.02
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|
% Increase in CD4 Tregs
Wk4 CD4Treg (%CD4Tcells)
|
11.93 percentage of CD4 T cells
Interval 8.88 to 15.94
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|
% Increase in CD4 Tregs
Wk8 CD4Treg(%CD4Tcells)
|
2.79 percentage of CD4 T cells
Interval 1.51 to 5.18
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% Increase in CD4 Tregs
Wk12 CD4Treg (%CD4Tcells)
|
2.63 percentage of CD4 T cells
Interval 1.75 to 4.94
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SECONDARY outcome
Timeframe: baseline, 2 weeks, 4 weeks, 8 weeks, 12 WeeksPeripheral Blood Mononuclear Cell Flow Cytometry
Outcome measures
| Measure |
Open Label. Active Study Treatment Arm
n=5 Participants
All patients received IL-2
|
|---|---|
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Differential Immune Cell Count
Baseline CD3Tcells (%PBMC)
|
53.74 percentage of PMBC
Interval 51.22 to 61.12
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Differential Immune Cell Count
Wk2 CD3Tcells (%PBMC)
|
57.05 percentage of PMBC
Interval 43.29 to 64.8
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Differential Immune Cell Count
Wk4 CD3Tcells (%PBMC)
|
53.69 percentage of PMBC
Interval 47.02 to 58.79
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|
Differential Immune Cell Count
Wk8 CD3Tcells (%PBMC)
|
58.00 percentage of PMBC
Interval 49.32 to 61.33
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Differential Immune Cell Count
Wk12 CD3 Tcells (%PBMC)
|
53.35 percentage of PMBC
Interval 47.08 to 63.17
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Differential Immune Cell Count
Baseline CD4Tcells (%PBMC)
|
25.59 percentage of PMBC
Interval 17.68 to 28.44
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Differential Immune Cell Count
Wk2 CD4Tcells (%PBMC)
|
23.7 percentage of PMBC
Interval 16.06 to 29.94
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Differential Immune Cell Count
Wk4 CD4Tcells (%PBMC)
|
26.11 percentage of PMBC
Interval 17.61 to 30.02
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Differential Immune Cell Count
Wk8 CD4Tcells (%PBMC)
|
26.91 percentage of PMBC
Interval 17.86 to 31.73
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|
Differential Immune Cell Count
Wk12 CD4Tcells (%PBMC)
|
26.08 percentage of PMBC
Interval 17.43 to 33.03
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|
Differential Immune Cell Count
Wk12 CD8Tcells (%PBMC)
|
23.23 percentage of PMBC
Interval 20.34 to 29.0
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|
Differential Immune Cell Count
Baseline CD19Bcells (%PBMC)
|
4.84 percentage of PMBC
Interval 3.93 to 9.175
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Differential Immune Cell Count
Wk2 CD19Bcells (%PBMC)
|
4.84 percentage of PMBC
Interval 4.35 to 7.7
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Differential Immune Cell Count
Wk4 CD19Bcells (%PBMC)
|
3.63 percentage of PMBC
Interval 3.54 to 9.0
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|
Differential Immune Cell Count
Wk8 CD19Bcells (%PBMC)
|
6.16 percentage of PMBC
Interval 4.73 to 11.65
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|
Differential Immune Cell Count
Wk12 CD19Bcells (%PBMC)
|
6.63 percentage of PMBC
Interval 4.41 to 12.4
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|
Differential Immune Cell Count
Baseline CD56NKcells (%PBMC)
|
6.37 percentage of PMBC
Interval 5.26 to 16.91
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|
Differential Immune Cell Count
Wk2 CD56NKcells (%PBMC)
|
12.53 percentage of PMBC
Interval 8.58 to 19.51
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Differential Immune Cell Count
Wk4 CD56NKcells (%PBMC)
|
11.72 percentage of PMBC
Interval 9.71 to 18.91
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|
Differential Immune Cell Count
Wk8 CD56NKcells (%PBMC)
|
6.95 percentage of PMBC
Interval 4.68 to 16.62
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|
Differential Immune Cell Count
Wk12 CD56NKcells (%PBMC)
|
6.09 percentage of PMBC
Interval 4.86 to 18.72
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Differential Immune Cell Count
Baseline NKTcells (%PBMC)
|
13.56 percentage of PMBC
Interval 3.41 to 17.01
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Differential Immune Cell Count
Wk2 NKTcells (%PBMC)
|
11.75 percentage of PMBC
Interval 2.94 to 12.88
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Differential Immune Cell Count
Wk4 NKTcells (%PBMC)
|
9.33 percentage of PMBC
Interval 3.4 to 12.42
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Differential Immune Cell Count
Wk8 NKTcells (%PBMC)
|
10.69 percentage of PMBC
Interval 3.12 to 12.21
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|
Differential Immune Cell Count
Wk12 NKTcells (%PBMC)
|
11.58 percentage of PMBC
Interval 2.95 to 12.04
|
|
Differential Immune Cell Count
Baseline Monocytes/Dendritic
|
12.82 percentage of PMBC
Interval 10.78 to 18.19
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|
Differential Immune Cell Count
Wk2 Monocytes/Dendritic cells (%PBMC)
|
9.00 percentage of PMBC
Interval 6.17 to 23.54
|
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Differential Immune Cell Count
Wk4 Monocytes/Dendritic (%PBMC)
|
12.87 percentage of PMBC
Interval 9.25 to 21.35
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|
Differential Immune Cell Count
Wk8 Monocytes/Dendritic (%PBMC)
|
11.77 percentage of PMBC
Interval 10.6 to 21.05
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Differential Immune Cell Count
Wk12 Monocytes/Dendritic (%PBMC)
|
12.35 percentage of PMBC
Interval 10.09 to 22.83
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Differential Immune Cell Count
Baseline CD8Tcells (%PBMC)
|
28.05 percentage of PMBC
Interval 21.94 to 32.78
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Differential Immune Cell Count
Wk2 CD8Tcells (%PBMC)
|
22.9 percentage of PMBC
Interval 18.46 to 29.94
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Differential Immune Cell Count
Wk4 CD8Tcells (%PBMC)
|
22.9 percentage of PMBC
Interval 18.26 to 24.57
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Differential Immune Cell Count
Wk8 CD8Tcells (%PBMC)
|
24.88 percentage of PMBC
Interval 22.23 to 29.51
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OTHER_PRE_SPECIFIED outcome
Timeframe: 2 weeks, 4 weeks, 8 weeks, 12 weeks, 36 weeksNumber of participants with a serum creatinine \> 1.5 x upper limit of normal through week 36
Outcome measures
| Measure |
Open Label. Active Study Treatment Arm
n=6 Participants
All patients received IL-2
|
|---|---|
|
Kidney Function Serum Panel (> 1.5 x Upper Limit Normal)
Week 2
|
1 Participants
|
|
Kidney Function Serum Panel (> 1.5 x Upper Limit Normal)
week 4
|
0 Participants
|
|
Kidney Function Serum Panel (> 1.5 x Upper Limit Normal)
week 8
|
0 Participants
|
|
Kidney Function Serum Panel (> 1.5 x Upper Limit Normal)
week 12
|
0 Participants
|
|
Kidney Function Serum Panel (> 1.5 x Upper Limit Normal)
week 36
|
0 Participants
|
OTHER_PRE_SPECIFIED outcome
Timeframe: week 2, 4 week, week 8, week12, week36Number of patients with a serum amino alaninetransferase \> 2 x upper limit of normal through week 36
Outcome measures
| Measure |
Open Label. Active Study Treatment Arm
n=6 Participants
All patients received IL-2
|
|---|---|
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Liver Function Serum Panel (> 2 x Upper Limit Normal)
week 2
|
0 Participants
|
|
Liver Function Serum Panel (> 2 x Upper Limit Normal)
week 4
|
0 Participants
|
|
Liver Function Serum Panel (> 2 x Upper Limit Normal)
week 8
|
0 Participants
|
|
Liver Function Serum Panel (> 2 x Upper Limit Normal)
weeks 12
|
0 Participants
|
|
Liver Function Serum Panel (> 2 x Upper Limit Normal)
week 36
|
0 Participants
|
Adverse Events
Active Study Medication Arm
Serious events: 1 serious events
Other events: 6 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Active Study Medication Arm
n=6 participants at risk
Single arm, prospective, open label efficacy and safety of a 4-weeks course of low dose IL-2 to expand TRegs in liver transplant recipients with stable liver function.
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Legionella disease
|
16.7%
1/6 • Number of events 1 • 36 weeks
Adverse events and serious adverse events were collected at day 1, 3, week 1, 2, 3, 4, 8, 12 and 36
|
|
Respiratory, thoracic and mediastinal disorders
Lung nodule
|
16.7%
1/6 • Number of events 1 • 36 weeks
Adverse events and serious adverse events were collected at day 1, 3, week 1, 2, 3, 4, 8, 12 and 36
|
Other adverse events
| Measure |
Active Study Medication Arm
n=6 participants at risk
Single arm, prospective, open label efficacy and safety of a 4-weeks course of low dose IL-2 to expand TRegs in liver transplant recipients with stable liver function.
|
|---|---|
|
General disorders
Edema
|
16.7%
1/6 • Number of events 1 • 36 weeks
Adverse events and serious adverse events were collected at day 1, 3, week 1, 2, 3, 4, 8, 12 and 36
|
|
Musculoskeletal and connective tissue disorders
Ankle Injury
|
16.7%
1/6 • Number of events 1 • 36 weeks
Adverse events and serious adverse events were collected at day 1, 3, week 1, 2, 3, 4, 8, 12 and 36
|
|
Musculoskeletal and connective tissue disorders
arthralgia
|
33.3%
2/6 • Number of events 2 • 36 weeks
Adverse events and serious adverse events were collected at day 1, 3, week 1, 2, 3, 4, 8, 12 and 36
|
|
Skin and subcutaneous tissue disorders
Injection site reaction
|
100.0%
6/6 • Number of events 6 • 36 weeks
Adverse events and serious adverse events were collected at day 1, 3, week 1, 2, 3, 4, 8, 12 and 36
|
|
General disorders
Fatigue
|
33.3%
2/6 • Number of events 2 • 36 weeks
Adverse events and serious adverse events were collected at day 1, 3, week 1, 2, 3, 4, 8, 12 and 36
|
|
Blood and lymphatic system disorders
eosinophilia
|
83.3%
5/6 • Number of events 5 • 36 weeks
Adverse events and serious adverse events were collected at day 1, 3, week 1, 2, 3, 4, 8, 12 and 36
|
|
Gastrointestinal disorders
Diarrhea
|
33.3%
2/6 • Number of events 2 • 36 weeks
Adverse events and serious adverse events were collected at day 1, 3, week 1, 2, 3, 4, 8, 12 and 36
|
|
Gastrointestinal disorders
Mouth Ulcer
|
16.7%
1/6 • Number of events 1 • 36 weeks
Adverse events and serious adverse events were collected at day 1, 3, week 1, 2, 3, 4, 8, 12 and 36
|
|
Respiratory, thoracic and mediastinal disorders
Pharyngitis
|
16.7%
1/6 • Number of events 1 • 36 weeks
Adverse events and serious adverse events were collected at day 1, 3, week 1, 2, 3, 4, 8, 12 and 36
|
|
Endocrine disorders
Hyperglycemia
|
16.7%
1/6 • Number of events 1 • 36 weeks
Adverse events and serious adverse events were collected at day 1, 3, week 1, 2, 3, 4, 8, 12 and 36
|
|
Respiratory, thoracic and mediastinal disorders
upper respiratory infection
|
50.0%
3/6 • Number of events 4 • 36 weeks
Adverse events and serious adverse events were collected at day 1, 3, week 1, 2, 3, 4, 8, 12 and 36
|
|
Renal and urinary disorders
Acute kidney injury
|
33.3%
2/6 • Number of events 2 • 36 weeks
Adverse events and serious adverse events were collected at day 1, 3, week 1, 2, 3, 4, 8, 12 and 36
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
33.3%
2/6 • Number of events 2 • 36 weeks
Adverse events and serious adverse events were collected at day 1, 3, week 1, 2, 3, 4, 8, 12 and 36
|
|
Musculoskeletal and connective tissue disorders
Chest pain
|
16.7%
1/6 • Number of events 1 • 36 weeks
Adverse events and serious adverse events were collected at day 1, 3, week 1, 2, 3, 4, 8, 12 and 36
|
|
Respiratory, thoracic and mediastinal disorders
Hiccough
|
16.7%
1/6 • Number of events 1 • 36 weeks
Adverse events and serious adverse events were collected at day 1, 3, week 1, 2, 3, 4, 8, 12 and 36
|
|
Blood and lymphatic system disorders
anemia
|
16.7%
1/6 • Number of events 1 • 36 weeks
Adverse events and serious adverse events were collected at day 1, 3, week 1, 2, 3, 4, 8, 12 and 36
|
|
Musculoskeletal and connective tissue disorders
Fracture radius
|
16.7%
1/6 • Number of events 1 • 36 weeks
Adverse events and serious adverse events were collected at day 1, 3, week 1, 2, 3, 4, 8, 12 and 36
|
|
Cardiac disorders
abnormal EKG
|
16.7%
1/6 • Number of events 1 • 36 weeks
Adverse events and serious adverse events were collected at day 1, 3, week 1, 2, 3, 4, 8, 12 and 36
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place