Trial Outcomes & Findings for Polyclonal Regulatory T Cells (PolyTregs) for Pemphigus (NCT NCT03239470)

Last Updated: 2024-02-15

Results Overview

Number of significant adverse events, defined as any related National Cancer Institute (NCI) Common Toxicity Criteria for Adverse Events (CTCAE) version 4.03 Grade 3 or higher adverse event or any related serious adverse event (SAE). Related is defined as being possibly related or related to the ex vivo expanded autologous PolyTregs, as determined by the safety review committee. An SAE is any untoward medical occurrence that, at any dose\* results in death, is life-threatening, requires hospitalization or prolongation of existing hospitalization, results in disability/incapacity, or is a congenital anomaly/birth defect or is medically significant. \*Polyclonal Tregs: Ex Vivo Expanded Autologous CD4+CD127lo/-CD25+ Polyclonal Regulatory T Cells.

Recruitment status

TERMINATED

Study phase

PHASE1

Target enrollment

5 participants

Primary outcome timeframe

Up to Week 52

Results posted on

2024-02-15

Participant Flow

Ten participants were screened at four sites in the United States, and 5 of those participants initiated blood donation. Enrollment occurred between October 2017 and May 2020. The first participant signed informed consent on October 10, 2017.

Participant milestones

Participant milestones
Measure	Cohort 1: Polyclonal Treg Infusion (PolyTregs) Low Dose Participants donated peripheral blood to be processed in a lab that expanded regulatory T cells (Tregs). After two weeks, participants received a single infusion of ex vivo expanded autologous CD4+CD127lo/-CD25+ polyclonal Tregs (PolyTregs). Target cell dose was 1 x 10\^8 PolyTregs.	Cohort 2: Polyclonal Treg Infusion (Poly Tregs) High Dose Participants donated peripheral blood to be processed in a lab that expanded regulatory T cells (Tregs). After two weeks, participants received a single infusion of ex vivo expanded autologous CD4+CD127lo/-CD25+ polyclonal Tregs (PolyTregs). Target cell dose was 2.5 x 10\^8 PolyTregs. Study enrollment was closed prior to enrolling participants into Cohort 2.
Blood Donation STARTED	5	0
Blood Donation COMPLETED	5	0
Blood Donation NOT COMPLETED	0	0
Polyclonal Treg Infusion STARTED	5	0
Polyclonal Treg Infusion COMPLETED	4	0
Polyclonal Treg Infusion NOT COMPLETED	1	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Cohort 1: Polyclonal Treg Infusion (PolyTregs) Low Dose Participants donated peripheral blood to be processed in a lab that expanded regulatory T cells (Tregs). After two weeks, participants received a single infusion of ex vivo expanded autologous CD4+CD127lo/-CD25+ polyclonal Tregs (PolyTregs). Target cell dose was 1 x 10\^8 PolyTregs.	Cohort 2: Polyclonal Treg Infusion (Poly Tregs) High Dose Participants donated peripheral blood to be processed in a lab that expanded regulatory T cells (Tregs). After two weeks, participants received a single infusion of ex vivo expanded autologous CD4+CD127lo/-CD25+ polyclonal Tregs (PolyTregs). Target cell dose was 2.5 x 10\^8 PolyTregs. Study enrollment was closed prior to enrolling participants into Cohort 2.
Polyclonal Treg Infusion Baseline Pemphigus Disease Area Index (PDAI) >12, thus ineligible for PolyTregs infusion.	1	0

Baseline Characteristics

Polyclonal Regulatory T Cells (PolyTregs) for Pemphigus

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Cohort 1: Polyclonal Treg Infusion (PolyTregs) Low Dose n=4 Participants Participants donated peripheral blood to be processed in a lab that expanded regulatory T cells (Tregs). After two weeks, participants received a single infusion of ex vivo expanded autologous CD4+CD127lo/-CD25+ polyclonal Tregs (PolyTregs). Target cell dose was 1 x 10\^8 PolyTregs.
Age, Continuous	38.8 years STANDARD_DEVIATION 14.2 • n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	2 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	0 Participants n=5 Participants
Race (NIH/OMB) White	4 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	4 Participants n=5 Participants
Type of Pemphigus Pemphigus Vulgaris	3 Participants n=5 Participants
Type of Pemphigus Pemphigus Foliaceus	1 Participants n=5 Participants
Age at Diagnosis of Pemphigus	33.5 years STANDARD_DEVIATION 16.9 • n=5 Participants

PRIMARY outcome

Timeframe: Up to Week 52

Population: Participants donated peripheral blood to be processed in a lab that expanded regulatory T cells (Tregs). After two weeks, participants received a single infusion of ex vivo expanded autologous CD4+CD127lo/-CD25+ polyclonal Tregs (PolyTregs). Target cell dose was 1 x 10\^8 PolyTregs.

Outcome measures

Outcome measures
Measure	Cohort 1: Polyclonal Treg Infusion (PolyTregs) n=4 Participants Participants donated peripheral blood to be processed in a lab that expanded regulatory T cells (Tregs). After two weeks, participants received a single infusion of ex vivo expanded autologous CD4+CD127lo/-CD25+ polyclonal Tregs (PolyTregs). Target cell dose was 1 x 10\^8 PolyTregs.
Number of Significant Adverse Events Through Week 52	0 Number of Events

SECONDARY outcome

Timeframe: From the start of investigational product infusion through Week 156.

Population: Participants donated peripheral blood to be processed in a lab that expanded the T cells (Tregs) to a target of 1 x 10\^8 PolyTregs/mL. After two weeks, the participant was infused with their own expanded Tregs.

Number of significant adverse events, defined as any related National Cancer Institute's (NCI's) Common Toxicity Criteria for Adverse Events (CTCAE) version 4.03 Grade 3 or higher adverse event or any related serious adverse event. Related is defined as being possibly related or related to the ex vivo expanded autologous PolyTregs, as determined by the safety review committee.

Outcome measures

Outcome measures
Measure	Cohort 1: Polyclonal Treg Infusion (PolyTregs) n=4 Participants Participants donated peripheral blood to be processed in a lab that expanded regulatory T cells (Tregs). After two weeks, participants received a single infusion of ex vivo expanded autologous CD4+CD127lo/-CD25+ polyclonal Tregs (PolyTregs). Target cell dose was 1 x 10\^8 PolyTregs.
Number of Significant Adverse Events	0 Number of Events

SECONDARY outcome

Timeframe: From the start of investigational product infusion through Week 156.

An adverse event is any untoward or unfavorable medical occurrence in a human subject, including any abnormal sign, symptom, or disease, temporally associated with the subject's participation in the research, whether or not considered related to the subject's participation in the research.

Outcome measures

Outcome measures
Measure	Cohort 1: Polyclonal Treg Infusion (PolyTregs) n=4 Participants Participants donated peripheral blood to be processed in a lab that expanded regulatory T cells (Tregs). After two weeks, participants received a single infusion of ex vivo expanded autologous CD4+CD127lo/-CD25+ polyclonal Tregs (PolyTregs). Target cell dose was 1 x 10\^8 PolyTregs.
Number of All Adverse Events	23 Number of Events

SECONDARY outcome

Timeframe: From the start of investigational product infusion through Week 52.

Adverse events Grade 3 or higher were graded according to the National Cancer Institute's Common Terminology Criteria for Adverse Events, Version 4.0.

Outcome measures

Outcome measures
Measure	Cohort 1: Polyclonal Treg Infusion (PolyTregs) n=4 Participants Participants donated peripheral blood to be processed in a lab that expanded regulatory T cells (Tregs). After two weeks, participants received a single infusion of ex vivo expanded autologous CD4+CD127lo/-CD25+ polyclonal Tregs (PolyTregs). Target cell dose was 1 x 10\^8 PolyTregs.
Number of All NCI-CTCAE Grade 3 or Higher Adverse Events	0 Number of Events

SECONDARY outcome

Timeframe: From the start of investigational product infusion through Week 156.

Adverse events Grade 3 or higher were graded according to the National Cancer Institute's Common Terminology Criteria for Adverse Events, Version 4.0.

Outcome measures

Outcome measures
Measure	Cohort 1: Polyclonal Treg Infusion (PolyTregs) n=4 Participants Participants donated peripheral blood to be processed in a lab that expanded regulatory T cells (Tregs). After two weeks, participants received a single infusion of ex vivo expanded autologous CD4+CD127lo/-CD25+ polyclonal Tregs (PolyTregs). Target cell dose was 1 x 10\^8 PolyTregs.
Number of All NCI-CTCAE Grade 3 or Higher Adverse Events	0 Number of Events

SECONDARY outcome

Timeframe: From the start of investigational product infusion through Week 156.

Number of all serious adverse events, defined as adverse events that result in the following outcomes (21 CFR 312.32(a) and ICH E2A): 1. Death 2. A life-threatening event: An AE or SAR is considered "life-threatening" if, in the view of either the investigator or DAIT, NIAID, its occurrence places the subject at immediate risk of death. It does not include an AE or SAR that, had it occurred in a more severe form, might have caused death. 3. Inpatient hospitalization or prolongation of existing hospitalization 4. Persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions 5. Congenital anomaly or birth defect 6. Important medical event that may not result in death, be life threatening, or require hospitalization may be considered serious when, based upon appropriate medical judgment, it may jeopardize the subject and may require medical or surgical intervention to prevent one of the outcomes listed above.

Outcome measures

Outcome measures
Measure	Cohort 1: Polyclonal Treg Infusion (PolyTregs) n=4 Participants Participants donated peripheral blood to be processed in a lab that expanded regulatory T cells (Tregs). After two weeks, participants received a single infusion of ex vivo expanded autologous CD4+CD127lo/-CD25+ polyclonal Tregs (PolyTregs). Target cell dose was 1 x 10\^8 PolyTregs.
Number of All SAEs	0 Number of Events

SECONDARY outcome

Timeframe: From the start of investigational product infusion through Week 156.

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

Outcome measures
Measure	Cohort 1: Polyclonal Treg Infusion (PolyTregs) n=4 Participants Participants donated peripheral blood to be processed in a lab that expanded regulatory T cells (Tregs). After two weeks, participants received a single infusion of ex vivo expanded autologous CD4+CD127lo/-CD25+ polyclonal Tregs (PolyTregs). Target cell dose was 1 x 10\^8 PolyTregs.
Number of All Infection Related Events	7 Number of Events

SECONDARY outcome

Timeframe: Within 24 hours of infusion

Defined as any adverse reaction of National Cancer Institute - Common Terminology Criteria Grade 1 and higher occurring within 24 hours of infusion. An adverse reaction means any AE caused by a drug.

Outcome measures

Outcome measures
Measure	Cohort 1: Polyclonal Treg Infusion (PolyTregs) n=4 Participants Participants donated peripheral blood to be processed in a lab that expanded regulatory T cells (Tregs). After two weeks, participants received a single infusion of ex vivo expanded autologous CD4+CD127lo/-CD25+ polyclonal Tregs (PolyTregs). Target cell dose was 1 x 10\^8 PolyTregs.
Number of All Infusion Reactions	5 Number of Events

SECONDARY outcome

Timeframe: Weeks 1, 2, 8, 12, 26, 39, 52, 78, 104, 130, and 156

The PDAI consists of a total activity score and a total damage score. The total activity score is a sum of the activity scores for skin, scalp and mucous membrane. The total activity score can range from 0 to 250. The total damage score is a sum of damage scores for skin and scalp. The total damage score can range from 0 to 13. Higher scores represent higher disease activity.

Outcome measures

Outcome measures
Measure	Cohort 1: Polyclonal Treg Infusion (PolyTregs) n=4 Participants Participants donated peripheral blood to be processed in a lab that expanded regulatory T cells (Tregs). After two weeks, participants received a single infusion of ex vivo expanded autologous CD4+CD127lo/-CD25+ polyclonal Tregs (PolyTregs). Target cell dose was 1 x 10\^8 PolyTregs.
Change in Pemphigus Disease Area Index (PDAI) Score From Baseline PDAI Total Activity Score at Week 1	-1.58 score Standard Deviation 1.179
Change in Pemphigus Disease Area Index (PDAI) Score From Baseline PDAI Total Activity Score at Week 2	-1.15 score Standard Deviation 2.845
Change in Pemphigus Disease Area Index (PDAI) Score From Baseline PDAI Total Activity Score at Week 8	-1.50 score Standard Deviation 3.032
Change in Pemphigus Disease Area Index (PDAI) Score From Baseline PDAI Total Activity Score at Week 12	-0.33 score Standard Deviation 4.392
Change in Pemphigus Disease Area Index (PDAI) Score From Baseline PDAI Total Activity Score at Week 26	-2.87 score Standard Deviation 3.092
Change in Pemphigus Disease Area Index (PDAI) Score From Baseline PDAI Total Activity Score at Week 39	-1.23 score Standard Deviation 6.823
Change in Pemphigus Disease Area Index (PDAI) Score From Baseline PDAI Total Activity Score at Week 52	-3.28 score Standard Deviation 3.323
Change in Pemphigus Disease Area Index (PDAI) Score From Baseline PDAI Total Activity Score at Week 78	-5.08 score Standard Deviation 3.496
Change in Pemphigus Disease Area Index (PDAI) Score From Baseline PDAI Total Activity Score at Week 104	-5.75 score Standard Deviation 4.070
Change in Pemphigus Disease Area Index (PDAI) Score From Baseline PDAI Total Activity Score at Week 130	-3.98 score Standard Deviation 7.617
Change in Pemphigus Disease Area Index (PDAI) Score From Baseline PDAI Total Activity Score at Week 156	-7.23 score Standard Deviation 2.700
Change in Pemphigus Disease Area Index (PDAI) Score From Baseline PDAI Total Damage Score at Week 1	0.000 score Standard Deviation 0.816
Change in Pemphigus Disease Area Index (PDAI) Score From Baseline PDAI Total Damage Score at Week 2	0.25 score Standard Deviation 1.258
Change in Pemphigus Disease Area Index (PDAI) Score From Baseline PDAI Total Damage Score at Week 8	-1.00 score Standard Deviation 1.414
Change in Pemphigus Disease Area Index (PDAI) Score From Baseline PDAI Total Damage Score at Week 12	-1.00 score Standard Deviation 1.414
Change in Pemphigus Disease Area Index (PDAI) Score From Baseline PDAI Total Damage Score at Week 26	-0.67 score Standard Deviation 1.155
Change in Pemphigus Disease Area Index (PDAI) Score From Baseline PDAI Total Damage Score at Week 39	-0.75 score Standard Deviation 0.957
Change in Pemphigus Disease Area Index (PDAI) Score From Baseline PDAI Total Damage Score at Week 52	-0.50 score Standard Deviation 1.291
Change in Pemphigus Disease Area Index (PDAI) Score From Baseline PDAI Total Damage Score at Week 78	-1.25 score Standard Deviation 1.893
Change in Pemphigus Disease Area Index (PDAI) Score From Baseline PDAI Total Damage Score at Week 104	-1.50 score Standard Deviation 2.380
Change in Pemphigus Disease Area Index (PDAI) Score From Baseline PDAI Total Damage Score at Week 130	-1.25 score Standard Deviation 2.630
Change in Pemphigus Disease Area Index (PDAI) Score From Baseline PDAI Total Damage Score at Week 156	-1.50 score Standard Deviation 2.380

SECONDARY outcome

Timeframe: Weeks 1, 2, 8, 12, 26, 39, 52, 78, 104, 130, and 156

Autoantibodies were measured by Enzyme-Linked Immunosorbent Assays (ELISA). Blood samples were taken from participants at baseline and Weeks 1, 2, 8, 12, 26, 39, 52, 78, 104, 130, and 156. If the desmoglein 1 or desmoglein 3 titer was below the limit of detection, the lower limit of detection at the central lab was imputed. The lower limit of detection for both desmoglein 1 and desmoglein 3 was 2.5 U/mL. Change was calculated as the post-baseline value minus the baseline value. A positive difference reflects an increase in the desmoglein titer value over time; a negative difference reflects a decreased desmoglein titer over time.

Outcome measures

Outcome measures
Measure	Cohort 1: Polyclonal Treg Infusion (PolyTregs) n=4 Participants Participants donated peripheral blood to be processed in a lab that expanded regulatory T cells (Tregs). After two weeks, participants received a single infusion of ex vivo expanded autologous CD4+CD127lo/-CD25+ polyclonal Tregs (PolyTregs). Target cell dose was 1 x 10\^8 PolyTregs.
Change in Desmoglein 1 and 3 Titers by ELISA From Baseline Desmoglein 1 at Week 1	1.00 U/mL Standard Deviation 1.414
Change in Desmoglein 1 and 3 Titers by ELISA From Baseline Desmoglein 1 at Week 2	1.50 U/mL Standard Deviation 2.082
Change in Desmoglein 1 and 3 Titers by ELISA From Baseline Desmoglein 1 at Week 8	-0.50 U/mL Standard Deviation 3.873
Change in Desmoglein 1 and 3 Titers by ELISA From Baseline Desmoglein 1 at Week 12	3.50 U/mL Standard Deviation 4.123
Change in Desmoglein 1 and 3 Titers by ELISA From Baseline Desmoglein 1 at Week 26	-7.67 U/mL Standard Deviation 17.786
Change in Desmoglein 1 and 3 Titers by ELISA From Baseline Desmoglein 1 at Week 39	-8.67 U/mL Standard Deviation 15.144
Change in Desmoglein 1 and 3 Titers by ELISA From Baseline Desmoglein 1 at Week 52	-17.00 U/mL Standard Deviation 33.237
Change in Desmoglein 1 and 3 Titers by ELISA From Baseline Desmoglein 1 at Week 78	-20.75 U/mL Standard Deviation 57.968
Change in Desmoglein 1 and 3 Titers by ELISA From Baseline Desmoglein 1 at Week 104	-33.88 U/mL Standard Deviation 68.940
Change in Desmoglein 1 and 3 Titers by ELISA From Baseline Desmoglein 1 at Week 130	-28.75 U/mL Standard Deviation 75.168
Change in Desmoglein 1 and 3 Titers by ELISA From Baseline Desmoglein 1 at Week 156	-28.25 U/mL Standard Deviation 79.943
Change in Desmoglein 1 and 3 Titers by ELISA From Baseline Desmoglein 3 at Week 1	-1.75 U/mL Standard Deviation 1.708
Change in Desmoglein 1 and 3 Titers by ELISA From Baseline Desmoglein 3 at Week 2	-0.50 U/mL Standard Deviation 4.123
Change in Desmoglein 1 and 3 Titers by ELISA From Baseline Desmoglein 3 at Week 8	3.00 U/mL Standard Deviation 9.309
Change in Desmoglein 1 and 3 Titers by ELISA From Baseline Desmoglein 3 at Week 12	1.25 U/mL Standard Deviation 15.457
Change in Desmoglein 1 and 3 Titers by ELISA From Baseline Desmoglein 3 at Week 26	13.33 U/mL Standard Deviation 27.538
Change in Desmoglein 1 and 3 Titers by ELISA From Baseline Desmoglein 3 at Week 39	-33.50 U/mL Standard Deviation 36.919
Change in Desmoglein 1 and 3 Titers by ELISA From Baseline Desmoglein 3 at Week 52	-30.75 U/mL Standard Deviation 45.966
Change in Desmoglein 1 and 3 Titers by ELISA From Baseline Desmoglein 3 at Week 78	-33.25 U/mL Standard Deviation 49.789
Change in Desmoglein 1 and 3 Titers by ELISA From Baseline Desmoglein 3 at Week 104	-38.88 U/mL Standard Deviation 50.150
Change in Desmoglein 1 and 3 Titers by ELISA From Baseline Desmoglein 3 at Week 130	-34.38 U/mL Standard Deviation 51.406
Change in Desmoglein 1 and 3 Titers by ELISA From Baseline Desmoglein 3 at Week 156	-42.25 U/mL Standard Deviation 49.291

SECONDARY outcome

Timeframe: From the start of investigational product infusion through Week 156.

Pemphigus relapses/flares were assessed using the consensus definition of 3 or more new lesions a month that do not heal spontaneously within 1 week or by the extension of established lesions in a patient who has achieved disease control.

Outcome measures

Outcome measures
Measure	Cohort 1: Polyclonal Treg Infusion (PolyTregs) n=4 Participants Participants donated peripheral blood to be processed in a lab that expanded regulatory T cells (Tregs). After two weeks, participants received a single infusion of ex vivo expanded autologous CD4+CD127lo/-CD25+ polyclonal Tregs (PolyTregs). Target cell dose was 1 x 10\^8 PolyTregs.
Number of Participants Experiencing a Relapse/Flare	2 participants

SECONDARY outcome

Timeframe: From the start of investigational product infusion through Week 156.

Population: Participants who initiated the investigational product infusion and experienced a flare between investigational product infusion and Week 156.

Pemphigus relapses/flares will be assessed using the consensus definition of 3 or more new lesions a month that do not heal spontaneously within 1 week or by the extension of established lesions in a patient who has achieved disease control. Time to flare is defined as the number of days between the date of the flare and the date of the investigational product infusion. Only participants who experienced a flare are summarized.

Outcome measures

Outcome measures
Measure	Cohort 1: Polyclonal Treg Infusion (PolyTregs) n=2 Participants Participants donated peripheral blood to be processed in a lab that expanded regulatory T cells (Tregs). After two weeks, participants received a single infusion of ex vivo expanded autologous CD4+CD127lo/-CD25+ polyclonal Tregs (PolyTregs). Target cell dose was 1 x 10\^8 PolyTregs.
Time to Relapse (Flare)	149.0 Days Standard Deviation 181.02

SECONDARY outcome

Timeframe: Weeks 12, 26, 39, 52, 78, 104, 130, and 156

Defined as the number of participants who were taking 0 mg/day, \>0 and \<=10 mg/day, or \>10 mg/day of prednisone at the time of the associated study visit.

Outcome measures

Outcome measures
Measure	Cohort 1: Polyclonal Treg Infusion (PolyTregs) n=4 Participants Participants donated peripheral blood to be processed in a lab that expanded regulatory T cells (Tregs). After two weeks, participants received a single infusion of ex vivo expanded autologous CD4+CD127lo/-CD25+ polyclonal Tregs (PolyTregs). Target cell dose was 1 x 10\^8 PolyTregs.
Number of Participants on Prednisone Dose ≤10 mg/Day Number taking 0 mg/day at Week 12	2 participants
Number of Participants on Prednisone Dose ≤10 mg/Day Number taking >0 mg/day and <= 10 mg/day at Week 12	2 participants
Number of Participants on Prednisone Dose ≤10 mg/Day Number taking >10 mg/day at Week 12	0 participants
Number of Participants on Prednisone Dose ≤10 mg/Day Number taking 0 mg/day at Week 26	2 participants
Number of Participants on Prednisone Dose ≤10 mg/Day Number taking >0 mg/day and <= 10 mg/day at Week 26	2 participants
Number of Participants on Prednisone Dose ≤10 mg/Day Number taking >10 mg/day at Week 26	0 participants
Number of Participants on Prednisone Dose ≤10 mg/Day Number taking 0 mg/day at Week 39	2 participants
Number of Participants on Prednisone Dose ≤10 mg/Day Number taking >0 mg/day and <= 10 mg/day at Week 39	2 participants
Number of Participants on Prednisone Dose ≤10 mg/Day Number taking >10 mg/day at Week 39	0 participants
Number of Participants on Prednisone Dose ≤10 mg/Day Number taking 0 mg/day at Week 52	2 participants
Number of Participants on Prednisone Dose ≤10 mg/Day Number taking >0 mg/day and <= 10 mg/day at Week 52	2 participants
Number of Participants on Prednisone Dose ≤10 mg/Day Number taking >10 mg/day at Week 52	0 participants
Number of Participants on Prednisone Dose ≤10 mg/Day Number taking 0 mg/day at Week 78	2 participants
Number of Participants on Prednisone Dose ≤10 mg/Day Number taking >0 mg/day and <= 10 mg/day at Week 78	2 participants
Number of Participants on Prednisone Dose ≤10 mg/Day Number taking >10 mg/day at Week 78	0 participants
Number of Participants on Prednisone Dose ≤10 mg/Day Number taking 0 mg/day at Week 104	2 participants
Number of Participants on Prednisone Dose ≤10 mg/Day Number taking >0 mg/day and <= 10 mg/day at Week 104	2 participants
Number of Participants on Prednisone Dose ≤10 mg/Day Number taking >10 mg/day at Week 104	0 participants
Number of Participants on Prednisone Dose ≤10 mg/Day Number taking 0 mg/day at Week 130	2 participants
Number of Participants on Prednisone Dose ≤10 mg/Day Number taking >0 mg/day and <= 10 mg/day at Week 130	2 participants
Number of Participants on Prednisone Dose ≤10 mg/Day Number taking >10 mg/day at Week 130	0 participants
Number of Participants on Prednisone Dose ≤10 mg/Day Number taking 0 mg/day at Week 156	2 participants
Number of Participants on Prednisone Dose ≤10 mg/Day Number taking >0 mg/day and <= 10 mg/day at Week 156	2 participants
Number of Participants on Prednisone Dose ≤10 mg/Day Number taking >10 mg/day at Week 156	0 participants

Adverse Events

Polyclonal Treg Infusion

Serious events: 0 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Polyclonal Treg Infusion n=4 participants at risk Participants donated peripheral blood to be processed in a lab that expanded the T cells (Tregs) to 1 x 10\^8 PolyTregs/mL. After two weeks, the participant was infused with their own expanded Tregs.
Blood and lymphatic system disorders Leukopenia	25.0% 1/4 • Number of events 1 • Adverse Event data were collected during the following time frames: • From time of signing of informed consent until start of investigational product infusion: all SAEs • From start of investigational product infusion until 24 hours post infusion: all NCI-CTCAE Grade 1 and higher AEs • From 24 hours post-infusion until Week 52: all NCI-CTCAE Grade 2 and higher AEs • From Week 52 until Week 156: all SAEs and all NCI-CTCAE Grade 3 and higher AEs
Blood and lymphatic system disorders Neutropenia	25.0% 1/4 • Number of events 1 • Adverse Event data were collected during the following time frames: • From time of signing of informed consent until start of investigational product infusion: all SAEs • From start of investigational product infusion until 24 hours post infusion: all NCI-CTCAE Grade 1 and higher AEs • From 24 hours post-infusion until Week 52: all NCI-CTCAE Grade 2 and higher AEs • From Week 52 until Week 156: all SAEs and all NCI-CTCAE Grade 3 and higher AEs
Gastrointestinal disorders Nausea	25.0% 1/4 • Number of events 1 • Adverse Event data were collected during the following time frames: • From time of signing of informed consent until start of investigational product infusion: all SAEs • From start of investigational product infusion until 24 hours post infusion: all NCI-CTCAE Grade 1 and higher AEs • From 24 hours post-infusion until Week 52: all NCI-CTCAE Grade 2 and higher AEs • From Week 52 until Week 156: all SAEs and all NCI-CTCAE Grade 3 and higher AEs
Infections and infestations COVID-19	50.0% 2/4 • Number of events 3 • Adverse Event data were collected during the following time frames: • From time of signing of informed consent until start of investigational product infusion: all SAEs • From start of investigational product infusion until 24 hours post infusion: all NCI-CTCAE Grade 1 and higher AEs • From 24 hours post-infusion until Week 52: all NCI-CTCAE Grade 2 and higher AEs • From Week 52 until Week 156: all SAEs and all NCI-CTCAE Grade 3 and higher AEs
Infections and infestations Eye infection	25.0% 1/4 • Number of events 1 • Adverse Event data were collected during the following time frames: • From time of signing of informed consent until start of investigational product infusion: all SAEs • From start of investigational product infusion until 24 hours post infusion: all NCI-CTCAE Grade 1 and higher AEs • From 24 hours post-infusion until Week 52: all NCI-CTCAE Grade 2 and higher AEs • From Week 52 until Week 156: all SAEs and all NCI-CTCAE Grade 3 and higher AEs
Infections and infestations Herpes simplex	25.0% 1/4 • Number of events 1 • Adverse Event data were collected during the following time frames: • From time of signing of informed consent until start of investigational product infusion: all SAEs • From start of investigational product infusion until 24 hours post infusion: all NCI-CTCAE Grade 1 and higher AEs • From 24 hours post-infusion until Week 52: all NCI-CTCAE Grade 2 and higher AEs • From Week 52 until Week 156: all SAEs and all NCI-CTCAE Grade 3 and higher AEs
Infections and infestations Viral upper respiratory tract infection	50.0% 2/4 • Number of events 2 • Adverse Event data were collected during the following time frames: • From time of signing of informed consent until start of investigational product infusion: all SAEs • From start of investigational product infusion until 24 hours post infusion: all NCI-CTCAE Grade 1 and higher AEs • From 24 hours post-infusion until Week 52: all NCI-CTCAE Grade 2 and higher AEs • From Week 52 until Week 156: all SAEs and all NCI-CTCAE Grade 3 and higher AEs
Injury, poisoning and procedural complications Fascial rupture	25.0% 1/4 • Number of events 1 • Adverse Event data were collected during the following time frames: • From time of signing of informed consent until start of investigational product infusion: all SAEs • From start of investigational product infusion until 24 hours post infusion: all NCI-CTCAE Grade 1 and higher AEs • From 24 hours post-infusion until Week 52: all NCI-CTCAE Grade 2 and higher AEs • From Week 52 until Week 156: all SAEs and all NCI-CTCAE Grade 3 and higher AEs
Injury, poisoning and procedural complications Infusion related reaction	50.0% 2/4 • Number of events 2 • Adverse Event data were collected during the following time frames: • From time of signing of informed consent until start of investigational product infusion: all SAEs • From start of investigational product infusion until 24 hours post infusion: all NCI-CTCAE Grade 1 and higher AEs • From 24 hours post-infusion until Week 52: all NCI-CTCAE Grade 2 and higher AEs • From Week 52 until Week 156: all SAEs and all NCI-CTCAE Grade 3 and higher AEs
Injury, poisoning and procedural complications Procedural pain	25.0% 1/4 • Number of events 1 • Adverse Event data were collected during the following time frames: • From time of signing of informed consent until start of investigational product infusion: all SAEs • From start of investigational product infusion until 24 hours post infusion: all NCI-CTCAE Grade 1 and higher AEs • From 24 hours post-infusion until Week 52: all NCI-CTCAE Grade 2 and higher AEs • From Week 52 until Week 156: all SAEs and all NCI-CTCAE Grade 3 and higher AEs
Musculoskeletal and connective tissue disorders Exostosis	25.0% 1/4 • Number of events 1 • Adverse Event data were collected during the following time frames: • From time of signing of informed consent until start of investigational product infusion: all SAEs • From start of investigational product infusion until 24 hours post infusion: all NCI-CTCAE Grade 1 and higher AEs • From 24 hours post-infusion until Week 52: all NCI-CTCAE Grade 2 and higher AEs • From Week 52 until Week 156: all SAEs and all NCI-CTCAE Grade 3 and higher AEs
Nervous system disorders Paraesthesia	25.0% 1/4 • Number of events 1 • Adverse Event data were collected during the following time frames: • From time of signing of informed consent until start of investigational product infusion: all SAEs • From start of investigational product infusion until 24 hours post infusion: all NCI-CTCAE Grade 1 and higher AEs • From 24 hours post-infusion until Week 52: all NCI-CTCAE Grade 2 and higher AEs • From Week 52 until Week 156: all SAEs and all NCI-CTCAE Grade 3 and higher AEs
Respiratory, thoracic and mediastinal disorders Rhinitis allergic	25.0% 1/4 • Number of events 1 • Adverse Event data were collected during the following time frames: • From time of signing of informed consent until start of investigational product infusion: all SAEs • From start of investigational product infusion until 24 hours post infusion: all NCI-CTCAE Grade 1 and higher AEs • From 24 hours post-infusion until Week 52: all NCI-CTCAE Grade 2 and higher AEs • From Week 52 until Week 156: all SAEs and all NCI-CTCAE Grade 3 and higher AEs
Respiratory, thoracic and mediastinal disorders Upper-airway cough syndrome	25.0% 1/4 • Number of events 1 • Adverse Event data were collected during the following time frames: • From time of signing of informed consent until start of investigational product infusion: all SAEs • From start of investigational product infusion until 24 hours post infusion: all NCI-CTCAE Grade 1 and higher AEs • From 24 hours post-infusion until Week 52: all NCI-CTCAE Grade 2 and higher AEs • From Week 52 until Week 156: all SAEs and all NCI-CTCAE Grade 3 and higher AEs
Skin and subcutaneous tissue disorders Pemphigus	25.0% 1/4 • Number of events 1 • Adverse Event data were collected during the following time frames: • From time of signing of informed consent until start of investigational product infusion: all SAEs • From start of investigational product infusion until 24 hours post infusion: all NCI-CTCAE Grade 1 and higher AEs • From 24 hours post-infusion until Week 52: all NCI-CTCAE Grade 2 and higher AEs • From Week 52 until Week 156: all SAEs and all NCI-CTCAE Grade 3 and higher AEs
Vascular disorders Flushing	25.0% 1/4 • Number of events 1 • Adverse Event data were collected during the following time frames: • From time of signing of informed consent until start of investigational product infusion: all SAEs • From start of investigational product infusion until 24 hours post infusion: all NCI-CTCAE Grade 1 and higher AEs • From 24 hours post-infusion until Week 52: all NCI-CTCAE Grade 2 and higher AEs • From Week 52 until Week 156: all SAEs and all NCI-CTCAE Grade 3 and higher AEs
Vascular disorders Hypertension	25.0% 1/4 • Number of events 1 • Adverse Event data were collected during the following time frames: • From time of signing of informed consent until start of investigational product infusion: all SAEs • From start of investigational product infusion until 24 hours post infusion: all NCI-CTCAE Grade 1 and higher AEs • From 24 hours post-infusion until Week 52: all NCI-CTCAE Grade 2 and higher AEs • From Week 52 until Week 156: all SAEs and all NCI-CTCAE Grade 3 and higher AEs
Vascular disorders Hypotension	25.0% 1/4 • Number of events 1 • Adverse Event data were collected during the following time frames: • From time of signing of informed consent until start of investigational product infusion: all SAEs • From start of investigational product infusion until 24 hours post infusion: all NCI-CTCAE Grade 1 and higher AEs • From 24 hours post-infusion until Week 52: all NCI-CTCAE Grade 2 and higher AEs • From Week 52 until Week 156: all SAEs and all NCI-CTCAE Grade 3 and higher AEs
Vascular disorders Orthostatic hypotension	25.0% 1/4 • Number of events 1 • Adverse Event data were collected during the following time frames: • From time of signing of informed consent until start of investigational product infusion: all SAEs • From start of investigational product infusion until 24 hours post infusion: all NCI-CTCAE Grade 1 and higher AEs • From 24 hours post-infusion until Week 52: all NCI-CTCAE Grade 2 and higher AEs • From Week 52 until Week 156: all SAEs and all NCI-CTCAE Grade 3 and higher AEs

Additional Information

Director, Clinical Research Operations Program

DAIT/NIAID

Phone: 240-669-5064

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place