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| ID | Type | Description | Link |
|---|---|---|---|
| APG01 | Other Identifier | Autoimmunity Centers of Excellence (ACE) | |
| NIAID CRMS ID#: 37534 | Other Identifier | DAIT NIAID |
Not provided
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Lack of recruitment, ongoing and new feasibility issues, and the impact of the coronavirus infectious disease 19 (COVID-19) pandemic
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| Name | Class |
|---|---|
| Autoimmunity Centers of Excellence | OTHER |
| Rho Federal Systems Division, Inc. | INDUSTRY |
Not provided
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T cells, a type of white blood cell called a lymphocyte, play an important role in the immune system. One subtype, the regulatory T cell (Treg) helps to regulate the immune system and may provide protection against the development of autoimmune disease. The hope is that these naturally occurring Treg cells can be utilized for the treatment of autoimmune disease and potentially replace the use of chronic immunosuppressive therapies that are associated with multiple side effects. There has been a small study showing safe administration of Tregs with decreased disease activity in patients with insulin-dependent diabetes. Tregs are being studied in lupus, cancer and organ transplantation.
This phase I trial will be conducted as an open-label, dose-escalation, multicenter trial in adult participants with active pemphigus.The purpose of this study is to test the safety and effect of Treg therapy in participants who have skin (cutaneous) involvement due to pemphigus.
Up to 12 adults between the ages of 18 and 75 years of age who have been diagnosed with pemphigus and meet all other entry criteria will be enrolled to receive one infusion of their own expanded Tregs at one of the following doses:
Safety, disease activity, and mechanism of action will be assessed over a three year period, using biospecimens from blood and skin. Study therapy administration will occur during an overnight stay, followed by 2 weekly visits, then monthly visits from Week 8 to Week 12, then quarterly visits from Week 26 to Week 52, then twice a year visits until Week 156.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1: 1.0 x 10^8 PolyTregs | Experimental | A single intravenous infusion of 1.0 x 10^8 PolyTregs will be administered. |
|
| Cohort 2: 2.5x10^8 PolyTregs | Experimental | A single intravenous infusion of 2.5x10^8 PolyTregs will be administered. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Cohort 1: 1.0 x 10^8 PolyTregs | Biological | Each participant will receive a target cell dose of 1.0 x 10^8 polyclonal Tregs. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Significant Adverse Events Through Week 52 | 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. | Up to Week 52 |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Significant Adverse Events | 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. |
Not provided
Inclusion Criteria:
Ability to provide informed consent;
Diagnosis of Pemphigus Vulgaris (PV) or Pemphigus Foliaceus (PF), defined by H&E staining (e.g., Haemotoxylin and Eosin) and direct immunofluorescence staining of skin biopsy at any time prior to enrollment;
Pemphigus treated with systemic corticosteroids within the 2 years prior to screening (historic or current), or treated with rituximab ≥ 12 months prior to screening;
Presence of:
Active of PV or PF as defined by Pemphigus Disease Area Index (PDAI) overall activity score 3-10 at screening visit, and PDAI overall activity score 1-12 at baseline visit;
Positive test for Epstein-Barr Virus (EBV) antibody;
Adequate venous access to support draw of 400 ml whole blood and infusion of investigational therapy; and
An absolute Treg count of ≥ 42 cells/μL within 6 weeks prior to whole blood collection at Week -2 (i.e., 2 weeks prior to planned PolyTreg Infusion).
Exclusion Criteria:
Initiation of systemic corticosteroid therapy, prednisone dose > 25 mg/d (or equivalent) or change in prednisone dose within 4 weeks prior to screening;
Addition of a new medication, or change in the dose of any background medication used to treat any aspect of pemphigus within the timeframes listed below. Specifically:
Doses of background medications at screening:
Use of rituximab within the 12 months prior to screening;
Change in dosing frequency, concentration, or applied surface area of topical steroids and/or topical calcineurin inhibitors within 2 weeks prior to screening;
Paraneoplastic pemphigus;
Pemphigus erythematosus;
Pemphigus vegetans;
Immunoglobulin A (IgA) pemphigus;
Drug-induced pemphigus;
Blood donation within 10 weeks prior to baseline visit (Day 0);
Hemoglobin < 10 g/dL;
White blood cell (WBC) count < 3,000/ mm^3 (equivalent to < 3 x10^9/L);
Lymphocyte count < 800/mm^3 (equivalent to < 0.8 x10^9/L);
Absolute neutrophil count < 1,500/mm^3 (equivalent to < 1.5 x10^9/L);
Platelets < 100,000/mm^3 (equivalent to < 100 x 10^9/L);
Liver function test [aspartate aminotransferase (AST)], alanine aminotransferase (ALT), or alkaline phosphatase (ALK)] results that are ≥ 2 times the upper limit of normal (ULN);
Direct bilirubin > ULN;
End stage renal disease [estimated glomerular filtration rate (eGFR) < 20 ml/min/1.73m^2 using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation];
At or within three months of screening:
Recent or ongoing active bacterial, viral, fungal, or opportunistic infections requiring systemic anti-infective therapy;
Evidence of current or prior infection with human immunodeficiency virus (HIV), hepatitis B [as assessed by HBsAg and anti-hepatitis B core antigen (HBc) Ab] or hepatitis C [as assessed by anti-Hepatitis C Virus (anti-HCV) Ab];
Detectable circulating EBV or Cytomegalovirus (CMV) genomes or active infection;
Chronic infection that is currently being treated with suppressive anti-infective therapy, including but not limited to tuberculosis, pneumocystis, CMV, herpes zoster, and atypical mycobacteria, with the exception of historical orolabial or localized cutaneous herpes simplex infections treated with suppressive anti- viral therapy;
Receipt of a live-attenuated vaccine within 12 months prior to screening;
Concomitant malignancies or a history of malignancy, with the exception of completely treated basal cell carcinoma of the skin;
Pregnancy;
Lactating or breastfeeding;
Unwilling or unable to use reliable method(s) of contraception:
For females of child-bearing potential, from four weeks prior to Day 0 through
1 year after Treg dosing;
For males, from the day of Treg infusion (baseline visit) to three months after Treg infusion.
Use of an investigational therapeutic medication, or other biologic medications except rituximab, within the past 90 days, or 5 half-lives prior to screening, whichever is greater;
Concomitant medical condition that places the subject at risk by participating in this study, including but not limited to:
Comorbidities requiring glucocorticoid therapy, including those which have required three or more courses of systemic glucocorticoids within the previous 12 months;
Current or history within the past year of substance abuse; or
Inability to comply with study and follow-up procedures.
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| Name | Affiliation | Role |
|---|---|---|
| Haley Naik, MD,MHSc | University of California San Francisco School of Medicine: Department of Dermatology | Study Chair |
| Anna Haemel, MD | University of California San Francisco School of Medicine: Department of Dermatology | Study Chair |
| Michael Rosenblum, MD, Ph.D. | University of California San Francisco School of Medicine: Department of Dermatology | Study Chair |
| Jeffrey Bluestone, Ph.D. | UCSF School of Medicine: UCSF Diabetes Clinic | Study Chair |
| David Wofsy, M.D. | University of California San Francisco School of Medicine: Lupus Clinic and Rheumatology Clinical Research Center | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of California San Francisco School of Medicine: Department of Dermatology | San Francisco | California | 94115 | United States | ||
Not provided
| Label | URL |
|---|---|
| National Institute of Allergy and Infectious Diseases (NIAID) website | View source |
| Division of Allergy, Immunology, and Transplantation (DAIT) website | View source |
| Autoimmunity Centers of Excellence (ACE) website |
Not provided
The plan is to share data upon completion of the study in: Immunology Database and Analysis Portal (ImmPort), a long-term archive of clinical and mechanistic data from DAIT-funded grants and contracts.
Not provided
On average, within 24 months after database lock for the trial.
Open access.
Not provided
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.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | 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. |
| FG001 | 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. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Blood Donation |
| |||||||||||||
| Polyclonal Treg Infusion |
|
Participants who donated peripheral blood to be processed in a lab that expanded regulatory T cells (Tregs) that, after two weeks, received a single infusion of ex vivo expanded autologous CD4+CD127lo/-CD25+ polyclonal Tregs (PolyTregs). Target cell dose was 1 x 10^8 PolyTregs.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | 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. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Significant Adverse Events Through Week 52 | 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. | 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. | Posted | Number | Number of Events | Up to Week 52 |
|
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
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Polyclonal Treg Infusion | 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. |
Not provided
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Leukopenia | Blood and lymphatic system disorders | 23.0 | Systematic Assessment |
Enrollment was stopped early, on May 1, 2020, due to:
No participants were enrolled in Cohort 2.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Director, Clinical Research Operations Program | DAIT/NIAID | 240-669-5064 | DAITClinicalTrialsGov@niaid.nih.gov |
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Dec 17, 2018 | Oct 6, 2021 | Prot_SAP_000.pdf |
Not provided
| ID | Term |
|---|---|
| D010392 | Pemphigus |
| ID | Term |
|---|---|
| D012872 | Skin Diseases, Vesiculobullous |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D001327 | Autoimmune Diseases |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Cohort 2: 2.5x10^8 PolyTregs | Biological | Each participant will receive a target cell dose of 2.5x10^8 polyclonal Tregs. |
|
|
| From the start of investigational product infusion through Week 156. |
| Number of All Adverse Events | 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. | From the start of investigational product infusion through Week 156. |
| Number of All NCI-CTCAE Grade 3 or Higher Adverse Events | Adverse events Grade 3 or higher were graded according to the National Cancer Institute's Common Terminology Criteria for Adverse Events, Version 4.0. | From the start of investigational product infusion through Week 52. |
| Number of All NCI-CTCAE Grade 3 or Higher Adverse Events | Adverse events Grade 3 or higher were graded according to the National Cancer Institute's Common Terminology Criteria for Adverse Events, Version 4.0. | From the start of investigational product infusion through Week 156. |
| Number of All SAEs | 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. | From the start of investigational product infusion through Week 156. |
| Number of All Infection Related Events | 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. | From the start of investigational product infusion through Week 156. |
| Number of All Infusion Reactions | 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. | Within 24 hours of infusion |
| Change in Pemphigus Disease Area Index (PDAI) Score From Baseline | 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. | Weeks 1, 2, 8, 12, 26, 39, 52, 78, 104, 130, and 156 |
| Change in Desmoglein 1 and 3 Titers by ELISA From Baseline | 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. | Weeks 1, 2, 8, 12, 26, 39, 52, 78, 104, 130, and 156 |
| Number of Participants Experiencing a Relapse/Flare | 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. | From the start of investigational product infusion through Week 156. |
| Time to Relapse (Flare) | 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. | From the start of investigational product infusion through Week 156. |
| Number of Participants on Prednisone Dose ≤10 mg/Day | 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. | Weeks 12, 26, 39, 52, 78, 104, 130, and 156 |
| University of Iowa Health Care: Department of Dermatology |
| Iowa City |
| Iowa |
| 52242 |
| United States |
| Duke University Medical Center: Department of Dermatology | Durham | North Carolina | 27710 | United States |
| University of Texas Southwestern Medical Center: Department of Dermatology | Dallas | Texas | 75390 | United States |
| View source |
| NOT COMPLETED |
|
|
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Count of Participants | Participants |
|
| Type of Pemphigus | Summary of the type of diagnosed pemphigus at screening. To be eligible for the study, participants must have diagnosis of pemphigus vulgaris or pemphigus foliaceus. | Count of Participants | Participants |
|
| Age at Diagnosis of Pemphigus | Study participant's age in years at the time of pemphigus diagnosis. | Mean | Standard Deviation | years |
|
| Description |
|---|
| OG000 | Cohort 1: Polyclonal Treg Infusion (PolyTregs) | 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. |
|
|
| Secondary | Number of Significant Adverse Events | 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. | 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. | Posted | Number | Number of Events | From the start of investigational product infusion through Week 156. |
|
|
|
| Secondary | Number of All Adverse Events | 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. | 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. | Posted | Number | Number of Events | From the start of investigational product infusion through Week 156. |
|
|
|
| Secondary | Number of All NCI-CTCAE Grade 3 or Higher Adverse Events | Adverse events Grade 3 or higher were graded according to the National Cancer Institute's Common Terminology Criteria for Adverse Events, Version 4.0. | 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. | Posted | Number | Number of Events | From the start of investigational product infusion through Week 52. |
|
|
|
| Secondary | Number of All NCI-CTCAE Grade 3 or Higher Adverse Events | Adverse events Grade 3 or higher were graded according to the National Cancer Institute's Common Terminology Criteria for Adverse Events, Version 4.0. | 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. | Posted | Number | Number of Events | From the start of investigational product infusion through Week 156. |
|
|
|
| Secondary | Number of All SAEs | 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. | 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. | Posted | Number | Number of Events | From the start of investigational product infusion through Week 156. |
|
|
|
| Secondary | Number of All Infection Related Events | 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. | 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. | Posted | Number | Number of Events | From the start of investigational product infusion through Week 156. |
|
|
|
| Secondary | Number of All Infusion Reactions | 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. | 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. | Posted | Number | Number of Events | Within 24 hours of infusion |
|
|
|
| Secondary | Change in Pemphigus Disease Area Index (PDAI) Score From Baseline | 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. | 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. | Posted | Mean | Standard Deviation | score | Weeks 1, 2, 8, 12, 26, 39, 52, 78, 104, 130, and 156 |
|
|
|
| Secondary | Change in Desmoglein 1 and 3 Titers by ELISA From Baseline | 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. | 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. | Posted | Mean | Standard Deviation | U/mL | Weeks 1, 2, 8, 12, 26, 39, 52, 78, 104, 130, and 156 |
|
|
|
| Secondary | Number of Participants Experiencing a Relapse/Flare | 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. | 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. | Posted | Number | participants | From the start of investigational product infusion through Week 156. |
|
|
|
| Secondary | Time to Relapse (Flare) | 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. | Participants who initiated the investigational product infusion and experienced a flare between investigational product infusion and Week 156. | Posted | Mean | Standard Deviation | Days | From the start of investigational product infusion through Week 156. |
|
|
|
| Secondary | Number of Participants on Prednisone Dose ≤10 mg/Day | 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. | 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. | Posted | Number | participants | Weeks 12, 26, 39, 52, 78, 104, 130, and 156 |
|
|
|
| 0 |
| 4 |
| 0 |
| 4 |
| 4 |
| 4 |
| Neutropenia | Blood and lymphatic system disorders | 23.0 | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | 23.0 | Systematic Assessment |
|
| COVID-19 | Infections and infestations | 23.0 | Systematic Assessment |
|
| Eye infection | Infections and infestations | 23.0 | Systematic Assessment |
|
| Herpes simplex | Infections and infestations | 23.0 | Systematic Assessment |
|
| Viral upper respiratory tract infection | Infections and infestations | 23.0 | Systematic Assessment |
|
| Fascial rupture | Injury, poisoning and procedural complications | 23.0 | Systematic Assessment |
|
| Infusion related reaction | Injury, poisoning and procedural complications | 23.0 | Systematic Assessment |
|
| Procedural pain | Injury, poisoning and procedural complications | 23.0 | Systematic Assessment |
|
| Exostosis | Musculoskeletal and connective tissue disorders | 23.0 | Systematic Assessment |
|
| Paraesthesia | Nervous system disorders | 23.0 | Systematic Assessment |
|
| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | 23.0 | Systematic Assessment |
|
| Upper-airway cough syndrome | Respiratory, thoracic and mediastinal disorders | 23.0 | Systematic Assessment |
|
| Pemphigus | Skin and subcutaneous tissue disorders | 23.0 | Systematic Assessment |
|
| Flushing | Vascular disorders | 23.0 | Systematic Assessment |
|
| Hypertension | Vascular disorders | 23.0 | Systematic Assessment |
|
| Hypotension | Vascular disorders | 23.0 | Systematic Assessment |
|
| Orthostatic hypotension | Vascular disorders | 23.0 | Systematic Assessment |
|
Not provided
Not provided
| D007154 | Immune System Diseases |
|
| PDAI Total Activity Score at Week 12 |
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| PDAI Total Activity Score at Week 26 |
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| PDAI Total Activity Score at Week 39 |
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| PDAI Total Activity Score at Week 52 |
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| PDAI Total Activity Score at Week 78 |
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| PDAI Total Activity Score at Week 104 |
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| PDAI Total Activity Score at Week 130 |
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| PDAI Total Activity Score at Week 156 |
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| PDAI Total Damage Score at Week 1 |
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| PDAI Total Damage Score at Week 2 |
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| PDAI Total Damage Score at Week 8 |
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| PDAI Total Damage Score at Week 12 |
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| PDAI Total Damage Score at Week 26 |
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| PDAI Total Damage Score at Week 39 |
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| PDAI Total Damage Score at Week 52 |
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| PDAI Total Damage Score at Week 78 |
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| PDAI Total Damage Score at Week 104 |
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| PDAI Total Damage Score at Week 130 |
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| PDAI Total Damage Score at Week 156 |
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| Title | Measurements |
|---|---|
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| Desmoglein 1 at Week 12 |
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| Desmoglein 1 at Week 26 |
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| Desmoglein 1 at Week 39 |
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| Desmoglein 1 at Week 52 |
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| Desmoglein 1 at Week 78 |
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| Desmoglein 1 at Week 104 |
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| Desmoglein 1 at Week 130 |
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| Desmoglein 1 at Week 156 |
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| Desmoglein 3 at Week 1 |
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| Desmoglein 3 at Week 2 |
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| Desmoglein 3 at Week 8 |
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| Desmoglein 3 at Week 12 |
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| Desmoglein 3 at Week 26 |
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| Desmoglein 3 at Week 39 |
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| Desmoglein 3 at Week 52 |
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| Desmoglein 3 at Week 78 |
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| Desmoglein 3 at Week 104 |
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| Desmoglein 3 at Week 130 |
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| Desmoglein 3 at Week 156 |
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| Title | Measurements |
|---|---|
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| Number taking 0 mg/day at Week 26 |
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| Number taking >0 mg/day and <= 10 mg/day at Week 26 |
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| Number taking >10 mg/day at Week 26 |
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| Number taking 0 mg/day at Week 39 |
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| Number taking >0 mg/day and <= 10 mg/day at Week 39 |
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| Number taking >10 mg/day at Week 39 |
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| Number taking 0 mg/day at Week 52 |
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| Number taking >0 mg/day and <= 10 mg/day at Week 52 |
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| Number taking >10 mg/day at Week 52 |
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| Number taking 0 mg/day at Week 78 |
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| Number taking >0 mg/day and <= 10 mg/day at Week 78 |
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| Number taking >10 mg/day at Week 78 |
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| Number taking 0 mg/day at Week 104 |
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| Number taking >0 mg/day and <= 10 mg/day at Week 104 |
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| Number taking >10 mg/day at Week 104 |
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| Number taking 0 mg/day at Week 130 |
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| Number taking >0 mg/day and <= 10 mg/day at Week 130 |
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| Number taking >10 mg/day at Week 130 |
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| Number taking 0 mg/day at Week 156 |
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| Number taking >0 mg/day and <= 10 mg/day at Week 156 |
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| Number taking >10 mg/day at Week 156 |
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