Trial Outcomes & Findings for Autologous Polyclonal Tregs for Lupus (NCT NCT02428309)
NCT ID: NCT02428309
Last Updated: 2019-11-12
Results Overview
A significant adverse event is any related National Cancer Institute's Common Terminology Criteria for Adverse Events, Version 4.0 Grade 3 or higher AE or any related serious adverse event. Related is defined as being possibly, probably, or definitely related to the ex vivo expanded autologous PolyTregs, as determined by the safety review committee.
TERMINATED
PHASE1
1 participants
From time of signed informed consent to Week 48
2019-11-12
Participant Flow
Participant recruitment occurred at one site in the United States. The site was activated in August 2015.
Participant milestones
| Measure |
Dose 1 (1x10^8)
Participant received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
|
|---|---|
|
Overall Study
STARTED
|
1
|
|
Overall Study
COMPLETED
|
1
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Autologous Polyclonal Tregs for Lupus
Baseline characteristics by cohort
| Measure |
Dose 1 (1x10^8)
n=1 Participants
Participant received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
1 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
46 years
STANDARD_DEVIATION NA • n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
1 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: From time of signed informed consent to Week 48Population: One participant enrolled in the study that received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
A significant adverse event is any related National Cancer Institute's Common Terminology Criteria for Adverse Events, Version 4.0 Grade 3 or higher AE or any related serious adverse event. Related is defined as being possibly, probably, or definitely related to the ex vivo expanded autologous PolyTregs, as determined by the safety review committee.
Outcome measures
| Measure |
Dose 1 (1x10^8)
n=1 Participants
Participant received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
|
|---|---|
|
Number of Significant Adverse Events (AEs) Through Week 48
|
0 Events
|
SECONDARY outcome
Timeframe: From time of signed informed consent to Week 152Population: One participant enrolled in the study that received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
A significant adverse event is any related National Cancer Institute's Common Terminology Criteria for Adverse Events, Version 4.0 Grade 3 or higher AE or any related serious adverse event. Related is defined as being possibly, probably, or definitely related to the ex vivo expanded autologous PolyTregs, as determined by the safety review committee.
Outcome measures
| Measure |
Dose 1 (1x10^8)
n=1 Participants
Participant received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
|
|---|---|
|
Number of Significant Adverse Events (AEs) Through Week 152
|
0 Events
|
SECONDARY outcome
Timeframe: From time of signed informed consent to Week 152Population: One participant enrolled in the study that received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
Adverse events (AEs) Grade 3 or higher were graded according to the National Cancer Institute's Common Terminology Criteria for Adverse Events, Version 4.0.
Outcome measures
| Measure |
Dose 1 (1x10^8)
n=1 Participants
Participant received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
|
|---|---|
|
Number of Grade 3 or Higher Adverse Events (AEs) Through Week 152
|
1 Events
|
SECONDARY outcome
Timeframe: From time of signed informed consent to Week 152Population: One participant enrolled in the study that received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
If the adverse event was believed to be caused by a viral, bacterial, or fungal organism, regardless of whether it was treated with antibiotics or not, then it was classified as infection-related.
Outcome measures
| Measure |
Dose 1 (1x10^8)
n=1 Participants
Participant received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
|
|---|---|
|
Number of Infection-Related Adverse Events (AEs) Through Week 152
|
0 Events
|
SECONDARY outcome
Timeframe: From time of signed informed consent to Week 152Population: One participant enrolled in the study that received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
An activity score increase of ≥4 CLASI points defines a flare. A mild/moderate flare includes at least one of the following SELENA-SLEDAI criteria: Increase in the SLEDAI Score of ≥3 points, new or worse discoid, photosensitive, profundus, cutaneous vasculitis, bullous lupus, nasopharyngeal ulcers, pleuritic, pericarditis, arthritis, fever attributable to SLE; increase in prednisone (\<0.5 mg/kg/day); added NSAID or Plaquenil; increase in PhGA (\<2.5 \[on a 3.0 indexed VAS scale\]). A severe flare includes at least one of the following SELENA-SLEDAI criteria: Increase of \>12 in the SLEDAI Score; new or worse CNS-SLE, vasculitis, nephritis, myositis, platelet count \<60,000/mm\^3, hemolytic anemia with hemoglobin \<7% or decrease in hemoglobin \>3%; prednisone \>0.5 mg/kg/day; new Cyclophosphamide, Azathioprine, Methotrexate, Mycophenolate Mofetil, or hospitalization attributable to SLE; increase in PhGA to \>2.5.
Outcome measures
| Measure |
Dose 1 (1x10^8)
n=1 Participants
Participant received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
|
|---|---|
|
Number of Lupus Flares Through Week 152 by Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) and Systemic Lupus Erythematosus Disease Activity Index (SELENA-SLEDAI) Criteria
No.of lupus flares (CLASI)
|
0 Flares
|
|
Number of Lupus Flares Through Week 152 by Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) and Systemic Lupus Erythematosus Disease Activity Index (SELENA-SLEDAI) Criteria
No. of lupus flares (SELENA-SLEDAI)
|
1 Flares
|
SECONDARY outcome
Timeframe: From time of infusion to 24 hours post infusionPopulation: One participant enrolled in the study that received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
Any infusion-related adverse events Grade 1 or higher within 24 hours of polyclonal Treg infusion. This study graded the severity of adverse events according to the National Cancer Institute's Common Terminology Criteria for Adverse Events, Version 4.0.
Outcome measures
| Measure |
Dose 1 (1x10^8)
n=1 Participants
Participant received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
|
|---|---|
|
Number Infusion-Related Adverse Events (AEs) Within 24 Hours of Infusion
|
0 Events
|
SECONDARY outcome
Timeframe: Baseline (Visit 0) and Weeks 4, 12, 48, and 152Population: One participant enrolled in the study that received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
Change=Post Baseline value minus Baseline value. A positive difference reflects an increased laboratory parameter value over time; a negative difference reflects a decreased laboratory parameter value over time. Normal laboratory values are based on subject age, gender, and the specific laboratory methods that were used to determine the lab values.
Outcome measures
| Measure |
Dose 1 (1x10^8)
n=1 Participants
Participant received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
|
|---|---|
|
Change From Baseline: Alkaline Phosphatase (ALK), Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST)
Alkaline Phosphatase (ALK) at Week 4
|
-9 U/L
Standard Deviation NA
Not applicable as only one participant was analyzed.
|
|
Change From Baseline: Alkaline Phosphatase (ALK), Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST)
Alkaline Phosphatase (ALK) at Week 12
|
4 U/L
Standard Deviation NA
Not applicable as only one participant was analyzed.
|
|
Change From Baseline: Alkaline Phosphatase (ALK), Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST)
Alkaline Phosphatase (ALK) at Week 48
|
4 U/L
Standard Deviation NA
Not applicable as only one participant was analyzed.
|
|
Change From Baseline: Alkaline Phosphatase (ALK), Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST)
Alkaline Phosphatase (ALK) at Week 152
|
-2 U/L
Standard Deviation NA
Not applicable as only one participant was analyzed.
|
|
Change From Baseline: Alkaline Phosphatase (ALK), Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST)
Alanine Aminotransferase (ALT) at Week 4
|
-1 U/L
Standard Deviation NA
Not applicable as only one participant was analyzed.
|
|
Change From Baseline: Alkaline Phosphatase (ALK), Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST)
Alanine Aminotransferase (ALT) at Week 12
|
-3 U/L
Standard Deviation NA
Not applicable as only one participant was analyzed.
|
|
Change From Baseline: Alkaline Phosphatase (ALK), Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST)
Alanine Aminotransferase (ALT) at Week 48
|
-1 U/L
Standard Deviation NA
Not applicable as only one participant was analyzed.
|
|
Change From Baseline: Alkaline Phosphatase (ALK), Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST)
Alanine Aminotransferase (ALT) at Week 152
|
-1 U/L
Standard Deviation NA
Not applicable as only one participant was analyzed.
|
|
Change From Baseline: Alkaline Phosphatase (ALK), Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST)
Aspartate Aminotransferase (AST) at Week 4
|
-5 U/L
Standard Deviation NA
Not applicable as only one participant was analyzed.
|
|
Change From Baseline: Alkaline Phosphatase (ALK), Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST)
Aspartate Aminotransferase (AST) at Week 12
|
-5 U/L
Standard Deviation NA
Not applicable as only one participant was analyzed.
|
|
Change From Baseline: Alkaline Phosphatase (ALK), Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST)
Aspartate Aminotransferase (AST) at Week 48
|
-4 U/L
Standard Deviation NA
Not applicable as only one participant was analyzed.
|
|
Change From Baseline: Alkaline Phosphatase (ALK), Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST)
Aspartate Aminotransferase (AST) at Week 152
|
-6 U/L
Standard Deviation NA
Not applicable as only one participant was analyzed.
|
SECONDARY outcome
Timeframe: Baseline (Visit 0) and Weeks 4, 12, 48, and 152Population: One participant enrolled in the study that received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
Change=Post Baseline value minus Baseline value. A positive difference reflects an increased laboratory parameter value over time; a negative difference reflects a decreased laboratory parameter value over time. Normal laboratory values depend on a subject age, gender, and the specific laboratory methods that were used to determine the lab values.
Outcome measures
| Measure |
Dose 1 (1x10^8)
n=1 Participants
Participant received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
|
|---|---|
|
Change From Baseline in g/dL: Albumin, Hemoglobin
Albumin at Week 4
|
NA g/dL
Standard Deviation NA
Not collected/available.
|
|
Change From Baseline in g/dL: Albumin, Hemoglobin
Albumin at Week 12
|
0.4 g/dL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in g/dL: Albumin, Hemoglobin
Albumin at Week 48
|
0.2 g/dL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in g/dL: Albumin, Hemoglobin
Albumin at Week 152
|
0.2 g/dL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in g/dL: Albumin, Hemoglobin
Hemoglobin at Week 4
|
1.6 g/dL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in g/dL: Albumin, Hemoglobin
Hemoglobin at Week 12
|
1.5 g/dL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in g/dL: Albumin, Hemoglobin
Hemoglobin at Week 48
|
1.3 g/dL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in g/dL: Albumin, Hemoglobin
Hemoglobin at Week 152
|
1.0 g/dL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
SECONDARY outcome
Timeframe: Baseline (Visit 0) and Weeks 4, 12, 48, and 152Population: One participant enrolled in the study that received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
Change=Post Baseline value minus Baseline value. A positive difference reflects an increased laboratory parameter value over time; a negative difference reflects a decreased laboratory parameter value over time. Normal laboratory values depend on a subject age, gender, and the specific laboratory methods that were used to determine the lab values.
Outcome measures
| Measure |
Dose 1 (1x10^8)
n=1 Participants
Participant received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
|
|---|---|
|
Change From Baseline in mg/dL: Total Bilirubin, Creatinine
Total Bilirubin at Week 4
|
0.1 mg/dL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in mg/dL: Total Bilirubin, Creatinine
Total Bilirubin at Week 12
|
-0.2 mg/dL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in mg/dL: Total Bilirubin, Creatinine
Total Bilirubin at Week 48
|
0.0 mg/dL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in mg/dL: Total Bilirubin, Creatinine
Total Bilirubin at Week 152
|
-0.2 mg/dL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in mg/dL: Total Bilirubin, Creatinine
Creatinine at Week 4
|
0.1 mg/dL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in mg/dL: Total Bilirubin, Creatinine
Creatinine at Week 12
|
0.0 mg/dL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in mg/dL: Total Bilirubin, Creatinine
Creatinine at Week 48
|
0.1 mg/dL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in mg/dL: Total Bilirubin, Creatinine
Creatinine at Week 152
|
0.1 mg/dL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
SECONDARY outcome
Timeframe: Baseline (Visit 0) and Weeks 4, 12, 48, and 152Population: One participant enrolled in the study that received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
Change=Post Baseline value minus Baseline value. A positive difference reflects an increased laboratory parameter value over time; a negative difference reflects a decreased laboratory parameter value over time. Normal laboratory values depend on a subject age, gender, and the specific laboratory methods that were used to determine the lab values.
Outcome measures
| Measure |
Dose 1 (1x10^8)
n=1 Participants
Participant received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
|
|---|---|
|
Change From Baseline in mmol/L: Potassium, Sodium, Chloride
Total Potassium at Week 4
|
-0.2 mmol/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in mmol/L: Potassium, Sodium, Chloride
Total Potassium at Week 12
|
-0.4 mmol/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in mmol/L: Potassium, Sodium, Chloride
Total Potassium at Week 48
|
-0.3 mmol/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in mmol/L: Potassium, Sodium, Chloride
Total Potassium at Week 152
|
-0.7 mmol/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in mmol/L: Potassium, Sodium, Chloride
Sodium at Week 4
|
4 mmol/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in mmol/L: Potassium, Sodium, Chloride
Sodium at Week 12
|
2 mmol/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in mmol/L: Potassium, Sodium, Chloride
Sodium at Week 48
|
3 mmol/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in mmol/L: Potassium, Sodium, Chloride
Sodium at Week 152
|
5 mmol/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in mmol/L: Potassium, Sodium, Chloride
Chloride at Week 4
|
-1 mmol/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in mmol/L: Potassium, Sodium, Chloride
Chloride at Week 12
|
-2 mmol/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in mmol/L: Potassium, Sodium, Chloride
Chloride at Week 48
|
-3 mmol/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in mmol/L: Potassium, Sodium, Chloride
Chloride at Week 152
|
-1 mmol/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
SECONDARY outcome
Timeframe: Baseline (Visit 0) and Weeks 4, 12, 48, and 152Population: One participant enrolled in the study that received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
Change=Post Baseline value minus Baseline value. A positive difference reflects an increased laboratory parameter value over time; a negative difference reflects a decreased laboratory parameter value over time. Normal laboratory values depend on a subject age, gender, and the specific laboratory methods that were used to determine the lab values.
Outcome measures
| Measure |
Dose 1 (1x10^8)
n=1 Participants
Participant received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
|
|---|---|
|
Change From Baseline in Cell Counts: White Blood Cells (WBC), Total Neutrophils, Lymphocytes, Monocytes, Eosinophils, Basophils, Platelets
White Blood Cells (WBC) at Week 4
|
0.8 Cell Count X 10^9/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Cell Counts: White Blood Cells (WBC), Total Neutrophils, Lymphocytes, Monocytes, Eosinophils, Basophils, Platelets
White Blood Cells (WBC) at Week 12
|
1.3 Cell Count X 10^9/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Cell Counts: White Blood Cells (WBC), Total Neutrophils, Lymphocytes, Monocytes, Eosinophils, Basophils, Platelets
White Blood Cells (WBC) at Week 48
|
1.2 Cell Count X 10^9/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Cell Counts: White Blood Cells (WBC), Total Neutrophils, Lymphocytes, Monocytes, Eosinophils, Basophils, Platelets
White Blood Cells (WBC) at Week 152
|
1.1 Cell Count X 10^9/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Cell Counts: White Blood Cells (WBC), Total Neutrophils, Lymphocytes, Monocytes, Eosinophils, Basophils, Platelets
Total Neutrophils at Week 4
|
0.6 Cell Count X 10^9/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Cell Counts: White Blood Cells (WBC), Total Neutrophils, Lymphocytes, Monocytes, Eosinophils, Basophils, Platelets
Total Neutrophils at Week 12
|
0.9 Cell Count X 10^9/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Cell Counts: White Blood Cells (WBC), Total Neutrophils, Lymphocytes, Monocytes, Eosinophils, Basophils, Platelets
Total Neutrophils at Week 48
|
0.9 Cell Count X 10^9/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Cell Counts: White Blood Cells (WBC), Total Neutrophils, Lymphocytes, Monocytes, Eosinophils, Basophils, Platelets
Total Neutrophils at Week 152
|
0.8 Cell Count X 10^9/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Cell Counts: White Blood Cells (WBC), Total Neutrophils, Lymphocytes, Monocytes, Eosinophils, Basophils, Platelets
Lymphocytes at Week 4
|
0.3 Cell Count X 10^9/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Cell Counts: White Blood Cells (WBC), Total Neutrophils, Lymphocytes, Monocytes, Eosinophils, Basophils, Platelets
Lymphocytes at Week 12
|
0.6 Cell Count X 10^9/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Cell Counts: White Blood Cells (WBC), Total Neutrophils, Lymphocytes, Monocytes, Eosinophils, Basophils, Platelets
Lymphocytes at Week 48
|
0.2 Cell Count X 10^9/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Cell Counts: White Blood Cells (WBC), Total Neutrophils, Lymphocytes, Monocytes, Eosinophils, Basophils, Platelets
Lymphocytes at Week 152
|
0.4 Cell Count X 10^9/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Cell Counts: White Blood Cells (WBC), Total Neutrophils, Lymphocytes, Monocytes, Eosinophils, Basophils, Platelets
Monocytes at Week 4
|
0.1 Cell Count X 10^9/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Cell Counts: White Blood Cells (WBC), Total Neutrophils, Lymphocytes, Monocytes, Eosinophils, Basophils, Platelets
Monocytes at Week 12
|
0.1 Cell Count X 10^9/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Cell Counts: White Blood Cells (WBC), Total Neutrophils, Lymphocytes, Monocytes, Eosinophils, Basophils, Platelets
Monocytes at Week 48
|
0.3 Cell Count X 10^9/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Cell Counts: White Blood Cells (WBC), Total Neutrophils, Lymphocytes, Monocytes, Eosinophils, Basophils, Platelets
Monocytes at Week 152
|
0.2 Cell Count X 10^9/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Cell Counts: White Blood Cells (WBC), Total Neutrophils, Lymphocytes, Monocytes, Eosinophils, Basophils, Platelets
Total Eosinophils at Week 4
|
-0.2 Cell Count X 10^9/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Cell Counts: White Blood Cells (WBC), Total Neutrophils, Lymphocytes, Monocytes, Eosinophils, Basophils, Platelets
Total Eosinophils at Week 12
|
-0.2 Cell Count X 10^9/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Cell Counts: White Blood Cells (WBC), Total Neutrophils, Lymphocytes, Monocytes, Eosinophils, Basophils, Platelets
Total Eosinophils at Week 48
|
-0.2 Cell Count X 10^9/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Cell Counts: White Blood Cells (WBC), Total Neutrophils, Lymphocytes, Monocytes, Eosinophils, Basophils, Platelets
Total Eosinophils at Week 152
|
-0.2 Cell Count X 10^9/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Cell Counts: White Blood Cells (WBC), Total Neutrophils, Lymphocytes, Monocytes, Eosinophils, Basophils, Platelets
Basophils at Week 4
|
0.0 Cell Count X 10^9/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Cell Counts: White Blood Cells (WBC), Total Neutrophils, Lymphocytes, Monocytes, Eosinophils, Basophils, Platelets
Basophils at Week 12
|
0.0 Cell Count X 10^9/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Cell Counts: White Blood Cells (WBC), Total Neutrophils, Lymphocytes, Monocytes, Eosinophils, Basophils, Platelets
Basophils at Week 48
|
0.0 Cell Count X 10^9/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Cell Counts: White Blood Cells (WBC), Total Neutrophils, Lymphocytes, Monocytes, Eosinophils, Basophils, Platelets
Basophils at Week 152
|
0.0 Cell Count X 10^9/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Cell Counts: White Blood Cells (WBC), Total Neutrophils, Lymphocytes, Monocytes, Eosinophils, Basophils, Platelets
Platelets at Week 4
|
66 Cell Count X 10^9/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Cell Counts: White Blood Cells (WBC), Total Neutrophils, Lymphocytes, Monocytes, Eosinophils, Basophils, Platelets
Platelets at Week 12
|
62 Cell Count X 10^9/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Cell Counts: White Blood Cells (WBC), Total Neutrophils, Lymphocytes, Monocytes, Eosinophils, Basophils, Platelets
Platelets at Week 48
|
16 Cell Count X 10^9/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Cell Counts: White Blood Cells (WBC), Total Neutrophils, Lymphocytes, Monocytes, Eosinophils, Basophils, Platelets
Platelets at Week 152
|
44 Cell Count X 10^9/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
SECONDARY outcome
Timeframe: Baseline (Visit 0) and Weeks 4, 12, 48, and 152Population: One participant enrolled in the study that received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
Change=Post Baseline value minus Baseline value. A positive difference reflects an increased laboratory parameter value over time; a negative difference reflects a decreased laboratory parameter value over time. Normal laboratory values depend on a subject age, gender, and the specific laboratory methods that were used to determine the lab values.
Outcome measures
| Measure |
Dose 1 (1x10^8)
n=1 Participants
Participant received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
|
|---|---|
|
Change From Baseline Red Blood Cell Count
Red Blood Cell Count at Week 4
|
0.6 Cell Count x10^12/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline Red Blood Cell Count
Red Blood Cell Count at Week 12
|
0.7 Cell Count x10^12/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline Red Blood Cell Count
Red Blood Cell Count at Week 48
|
0.6 Cell Count x10^12/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline Red Blood Cell Count
Red Blood Cell Count at Week 152
|
0.9 Cell Count x10^12/L
Standard Deviation NA
Not applicable as only one participant was analyzed
|
SECONDARY outcome
Timeframe: Baseline (Visit 0) and Weeks 4, 12, 48, and 152Population: One participant enrolled in the study that received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
Change=Post Baseline value minus Baseline value. A positive difference reflects an increased laboratory parameter value over time; a negative difference reflects a decreased laboratory parameter value over time. Normal laboratory values depend on a subject age, gender, and the specific laboratory methods that were used to determine the lab values.
Outcome measures
| Measure |
Dose 1 (1x10^8)
n=1 Participants
Participant received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
|
|---|---|
|
Change From Baseline in mm/hr: Sedimentation Rate (ESR)
Sedimentation Rate (ESR) at Week 4
|
0 mm/hr
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in mm/hr: Sedimentation Rate (ESR)
Sedimentation Rate (ESR) at Week 12
|
4 mm/hr
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in mm/hr: Sedimentation Rate (ESR)
Sedimentation Rate (ESR) at Week 48
|
20 mm/hr
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in mm/hr: Sedimentation Rate (ESR)
Sedimentation Rate (ESR) at Week 152
|
2 mm/hr
Standard Deviation NA
Not applicable as only one participant was analyzed
|
SECONDARY outcome
Timeframe: Baseline (Visit 0) and Weeks 12, 24, 36, 48, 100, 126, and 152Population: One participant enrolled in the study that received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
The Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) is a validated, physician-based assessment tool used to measure cutaneous lupus severity. Severity is calculated based on disease activity (erythema and scale) and damage (dyspigmentation and scarring) for the cumulative areas of involved skin. Severity categories based on the CLASI activity score are as follows: mild (0-9), moderate (10-20), and severe (21-70). A 4-point or 20% change in the CLASI activity score identifies a clinically meaningful change. A 4-point increase in the CLASI activity score indicates a flare.
Outcome measures
| Measure |
Dose 1 (1x10^8)
n=1 Participants
Participant received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
|
|---|---|
|
Change From Baseline in Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) Activity Score
Week 12
|
-2 units on a scale
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) Activity Score
Week 24
|
-1 units on a scale
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) Activity Score
Week 36
|
-1 units on a scale
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) Activity Score
Week 48
|
-1 units on a scale
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) Activity Score
Week 100
|
-15 units on a scale
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) Activity Score
Week 126
|
-10 units on a scale
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) Activity Score
Week 152
|
-12 units on a scale
Standard Deviation NA
Not applicable as only one participant was analyzed
|
SECONDARY outcome
Timeframe: Baseline (Visit 0) and Weeks 12, 24, 36, 48, 100, 126, and 152Population: One participant enrolled in the study that received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
The Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus (SLE) Disease Activity Index (SELENA-SLEDAI) score is a weighted scale score ranging from 0 to 105 based on the presence or absence of 24 manifestations of SLE. The SELENA-SLEDAI assesses disease activity for 10 days prior to and including the day of assessment. Positive change in the SELENA-SLEDAI score indicates increased disease activity.
Outcome measures
| Measure |
Dose 1 (1x10^8)
n=1 Participants
Participant received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
|
|---|---|
|
Change From Baseline in SELENA-SLEDAI Total Score
Week 12
|
0 units on a scale
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in SELENA-SLEDAI Total Score
Week 24
|
0 units on a scale
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in SELENA-SLEDAI Total Score
Week 36
|
-2 units on a scale
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in SELENA-SLEDAI Total Score
Week 48
|
-2 units on a scale
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in SELENA-SLEDAI Total Score
Week 100
|
-2 units on a scale
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in SELENA-SLEDAI Total Score
Week 126
|
0 units on a scale
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in SELENA-SLEDAI Total Score
Week 152
|
0 units on a scale
Standard Deviation NA
Not applicable as only one participant was analyzed
|
SECONDARY outcome
Timeframe: Baseline (Visit 0) and Weeks 12, 24, 36, 48, 100, 126, and 152Population: One participant enrolled in the study that received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
The global assessment (PGA) is a visual 3-inch analog scale from 0 to 3 in which the participant marks the scale according to perceived disease activity. A score of 0 corresponds to no lupus disease activity and a score of 3 corresponds to severe disease activity. A positive change from baseline indicates more disease activity.
Outcome measures
| Measure |
Dose 1 (1x10^8)
n=1 Participants
Participant received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
|
|---|---|
|
Change From Baseline in Patient's Global Assessment (PGA)
Week 12
|
-0.625 inches
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Patient's Global Assessment (PGA)
Week 24
|
-0.875 inches
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Patient's Global Assessment (PGA)
Week 36
|
-0.625 inches
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Patient's Global Assessment (PGA)
Week 48
|
1.000 inches
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Patient's Global Assessment (PGA)
Week 100
|
-1.000 inches
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Patient's Global Assessment (PGA)
Week 126
|
0.500 inches
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Patient's Global Assessment (PGA)
Week 152
|
0.000 inches
Standard Deviation NA
Not applicable as only one participant was analyzed
|
SECONDARY outcome
Timeframe: Baseline (Visit 0) and Weeks 12, 24, 36, 48, 100, 126, and 152Population: One participant enrolled in the study that received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
The physician's global assessment (PhGA) is a visual analog 3-inch scale in the SELENA-SLEDAI that is scored from 0 to 3 by the physician. A score of 0 corresponds to no lupus disease activity and a score of 3 corresponds to severe disease activity. A positive change from baseline indicates more disease activity.
Outcome measures
| Measure |
Dose 1 (1x10^8)
n=1 Participants
Participant received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
|
|---|---|
|
Change From Baseline in Physician's Global Assessment (PhGA)
Week 12
|
-0.125 inches
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Physician's Global Assessment (PhGA)
Week 24
|
-0.125 inches
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Physician's Global Assessment (PhGA)
Week 36
|
-0.125 inches
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Physician's Global Assessment (PhGA)
Week 48
|
-0.125 inches
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Physician's Global Assessment (PhGA)
Week 100
|
-0.938 inches
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Physician's Global Assessment (PhGA)
Week 126
|
0.875 inches
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Physician's Global Assessment (PhGA)
Week 152
|
0.563 inches
Standard Deviation NA
Not applicable as only one participant was analyzed
|
SECONDARY outcome
Timeframe: Baseline ( Visit 0) and Weeks 12, 24, 36, 48, 100, 126, and 152Population: One participant enrolled in the study that received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
Double-stranded DNA is one of multiple diagnostic tests for SLE and high levels may be associated with disease activity. The positive range is based on the normal range from the local laboratory. A positive change from baseline value indicates the detection of autoantibodies to double-stranded DNA.
Outcome measures
| Measure |
Dose 1 (1x10^8)
n=1 Participants
Participant received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
|
|---|---|
|
Change From Baseline in Anti-dsDNA Antibody Titers
Week 12
|
7 IU/mL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Anti-dsDNA Antibody Titers
Week 24
|
35 IU/mL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Anti-dsDNA Antibody Titers
Week 36
|
136 IU/mL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Anti-dsDNA Antibody Titers
Week 48
|
25 IU/mL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Anti-dsDNA Antibody Titers
Week 100
|
138 IU/mL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Anti-dsDNA Antibody Titers
Week 126
|
283 IU/mL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Anti-dsDNA Antibody Titers
Week 152
|
-37 IU/mL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
SECONDARY outcome
Timeframe: Baseline (Visit 0) and Weeks 12, 24, 36, 48, 100, 126, and 152Population: One participant enrolled in the study that received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
C3 is a blood test that measures the activity of the complement component 3 (C3) protein. The normal C3 range is 71 to 159 mg/dL. Those with active systemic lupus erythematosus (SLE) may have a lower-than-normal level of C3. A decrease in C3 level over time may indicate SLE disease activity.
Outcome measures
| Measure |
Dose 1 (1x10^8)
n=1 Participants
Participant received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
|
|---|---|
|
Change From Baseline in Serum C3 Complement Levels
Week 36
|
11 mg/dL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Serum C3 Complement Levels
Week 12
|
-2 mg/dL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Serum C3 Complement Levels
Week 24
|
11 mg/dL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Serum C3 Complement Levels
Week 48
|
-4 mg/dL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Serum C3 Complement Levels
Week 100
|
8 mg/dL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Serum C3 Complement Levels
Week 126
|
-6 mg/dL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Serum C3 Complement Levels
Week 152
|
0 mg/dL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
SECONDARY outcome
Timeframe: Baseline (Visit 0) and Weeks 12, 24, 36, 48, 100, 126, and 152Population: One participant enrolled in the study that received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
C4 is a blood test that measures the activity of the complement component 4 (C4) protein. The normal range is 13 to 30 mg/dL. Individuals with active systemic lupus erythematosus (SLE) may have a lower-than-normal level of C4. A decrease in C4 level over time may indicate disease activity.
Outcome measures
| Measure |
Dose 1 (1x10^8)
n=1 Participants
Participant received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
|
|---|---|
|
Change From Baseline in Serum C4 Complement Levels
Week 12
|
-1 mg/dL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Serum C4 Complement Levels
Week 24
|
-1 mg/dL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Serum C4 Complement Levels
Week 36
|
-3 mg/dL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Serum C4 Complement Levels
Week 48
|
-5 mg/dL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Serum C4 Complement Levels
Week 100
|
1 mg/dL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Serum C4 Complement Levels
Week 126
|
-1 mg/dL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
|
Change From Baseline in Serum C4 Complement Levels
Week 152
|
3 mg/dL
Standard Deviation NA
Not applicable as only one participant was analyzed
|
Adverse Events
Dose 1 (1x10^8)
Serious adverse events
| Measure |
Dose 1 (1x10^8)
n=1 participants at risk
Participant received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
|
|---|---|
|
Nervous system disorders
Neuropathy peripheral
|
100.0%
1/1 • Number of events 1 • From time of signed informed consent to Week 48.
|
Other adverse events
| Measure |
Dose 1 (1x10^8)
n=1 participants at risk
Participant received a single infusion of 1 x 10\^8 autologous polyclonal Tregs (ex vivo selected and expanded)
|
|---|---|
|
Cardiac disorders
Pericarditis
|
100.0%
1/1 • Number of events 1 • From time of signed informed consent to Week 48.
|
|
General disorders
Oedema peripheral
|
100.0%
1/1 • Number of events 1 • From time of signed informed consent to Week 48.
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
100.0%
1/1 • Number of events 1 • From time of signed informed consent to Week 48.
|
|
Skin and subcutaneous tissue disorders
Cutaneous lupus erythematosus
|
100.0%
1/1 • Number of events 1 • From time of signed informed consent to Week 48.
|
Additional Information
Director, Clinical Research Operations Program
DAIT/NIAID
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place