Pemphigus FoliaceusPhase 1NCT03239470
Polyclonal Regulatory T Cells (PolyTregs) for Pemphigus
A Phase 1 study testing Cohort 1: 1.0 x 10^8 PolyTregs and Cohort 2: 2.5x10^8 PolyTregs in people with Pemphigus Foliaceus and Pemphigus Vulgaris, run by National Institute of Allergy and Infectious Diseases (NIAID) at 4 sites in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Pemphigus Foliaceus, Pemphigus Vulgaris
- What is being tested?
- Cohort 1: 1.0 x 10^8 PolyTregs and Cohort 2: 2.5x10^8 PolyTregs
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 4 sitesUnited States
- Study dates
- 10 Oct 2017to 9 Jan 2023
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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:
- anti-Dsg3 antibodies (>20.0 U/ml) at screening visit consistent with diagnosis of pemphigus vulgaris or,
- anti-Dsg1 antibodies (>20.0 U/ml) at screening visit consistent with diagnosis of pemphigus foliaceus.
Show all 10Show fewer
- 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).
Who cannot join
- 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:
- methotrexate, mycophenolate mofetil, mycophenolic acid, azathioprine, cyclosporine or dapsone within the 6 weeks prior to screening or in the time between screening and study drug infusion,
- intravenous Immunoglobulin (IVIG) within 12 weeks prior to screening or in the time between screening and study drug infusion (subjects on IVIG must be on stable dose for at least 12 weeks prior to screening),
- treatment with cyclophosphamide within 12 weeks prior to screening or in the time between screening and study drug infusion.
- Doses of background medications at screening:
Show all 53Show fewer
- methotrexate > 25 mg/week,
- mycophenolate mofetil > 3000 mg/d,
- mycophenolic acid > 1080 mg/bid,
- azathioprine > 200 mg/d,
- cyclosporine > 2 mg/kg/d,
- dapsone >250 mg/d,or
- intravenous immunoglobulin (IVIG) > 4mg/kg monthly.
- 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:
- a positive QuantiFERON(R)-TB Gold test or positive purified protein derivative tuberculin skin test (PPD) [>5mm induration, regardless of Bacille Calmette Guerin (BCG) vaccine administration] unless completion of treatment has been documented for active Tuberculosis (TB),
- an indeterminate QuantiFERON (R)-TB Gold test unless followed by a subsequent negative PPD or negative QuantiFERON(R)-TB Gold test as well as a consultation with and clearance by local infectious disease (ID) department.
- 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:
- another severe, systemic autoimmune disease or condition (besides pemphigus) requiring systemic immunosuppressive therapy (e.g., rheumatoid arthritis, Systemic Lupus Erythematosus (SLE), systemic sclerosis, primary Sjogren's syndrome, primary vasculitis, psoriasis, multiple sclerosis, ankylosing spondylitis, and inflammatory bowel disease), or
- severe, progressive, or poorly controlled renal, hepatic, hematological, gastrointestinal, pulmonary, cardiac, or neurological disease, or
- history of significant infection or recurrent infection that, in the investigator's opinion, places the subject at risk by participating in this study, or
- any other concomitant medical condition that, in the investigator's opinion, places the subject at risk by participating in this study.
- 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.
Trial locations and contacts
4 sites in 1 country.
United States4 sites
University of Texas Southwestern Medical Center: Department of Dermatology
Dallas, Texas, 75390
Duke University Medical Center: Department of Dermatology
Durham, North Carolina, 27710
University of Iowa Health Care: Department of Dermatology
Iowa City, Iowa, 52242
University of California San Francisco School of Medicine: Department of Dermatology
San Francisco, California, 94115
About this study
Official title: A Phase I, Open-Label, Multicenter Trial Exploring the Safety and Tolerability of Autologous Polyclonal Regulatory T Cell Therapy in Adults With Active Pemphigus (APG01)
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.
Read the detailed description
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:
- 1.0 x 10^8 PolyTregs or
- 2.5 x 10^8 PolyTregs.
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.
- Conditions
- Pemphigus Foliaceus, Pemphigus Vulgaris
- Being tested
- Cohort 1: 1.0 x 10^8 PolyTregs, Cohort 2: 2.5x10^8 PolyTregs
- Keywords
- autologous polyclonal regulatory T cell therapy, PolyTregs, open-label, Phase 1 (safety)
- Medical subject headings
- Pemphigus
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cohort 1: 1.0 x 10^8 PolyTregsBiological
Also called Polyclonal Regulatory T Cells, autologous PolyTregs, CD4+CD127lo/negCD25+ PolyTregs
Each participant will receive a target cell dose of 1.0 x 10^8 polyclonal Tregs.
Cohort 2: 2.5x10^8 PolyTregsBiological
Also called Polyclonal Regulatory T Cells, autologous PolyTregs, CD4+CD127lo/negCD25+ PolyTregs
Each participant will receive a target cell dose of 2.5x10^8 polyclonal Tregs.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1: 1.0 x 10^8 PolyTregs | Experimental | A single intravenous infusion of 1.0 x 10^8 PolyTregs will be administered. Receives: Biological: Cohort 1: 1.0 x 10^8 PolyTregs |
| Cohort 2: 2.5x10^8 PolyTregs | Experimental | A single intravenous infusion of 2.5x10^8 PolyTregs will be administered. Receives: Biological: Cohort 2: 2.5x10^8 PolyTregs |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Up to Week 52
Secondary outcomes (12)
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Weeks 12, 26, 39, 52, 78, 104, 130, and 156
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Aug 2017
- Study start10 Oct 2017
- Primary completion10 Dec 2020
- Study completion9 Jan 2023
Why it stopped: Lack of recruitment, ongoing and new feasibility issues, and the impact of the coronavirus infectious disease 19 (COVID-19) pandemic
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Pemphigus FoliaceusStudies of the same condition, open and under way.
- More trials run by National Institute of Allergy and Infectious Diseases (NIAID)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Collaborators
- Autoimmunity Centers of Excellence, Rho Federal Systems Division, Inc.
- Responsible party
- Sponsor
- Investigators
- Haley Naik, MD,MHSc, Study chair, University of California San Francisco School of Medicine: Department of DermatologyAnna Haemel, MD, Study chair, University of California San Francisco School of Medicine: Department of DermatologyMichael Rosenblum, MD, Ph.D., Study chair, University of California San Francisco School of Medicine: Department of DermatologyJeffrey Bluestone, Ph.D., Study chair, UCSF School of Medicine: UCSF Diabetes ClinicDavid Wofsy, M.D., Study chair, University of California San Francisco School of Medicine: Lupus Clinic and Rheumatology Clinical Research Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03239470
- Sponsor's protocol ID
- DAIT APG01
- Other IDs
- APG01, NIAID CRMS ID#: 37534
- Status last verified
- Feb 2024
- First submitted
- 2 Aug 2017
- Last updated
- 15 Feb 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Study at a glance
From the registry record, last updated by the sponsor on 15 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I, Open-Label, Multicenter Trial Exploring the Safety and Tolerability of Autologous Polyclonal Regulatory T Cell Therapy in Adults With Active Pemphigus (APG01)
- ClinicalTrials.gov ID
- NCT03239470
- Acronym / sponsor ID
- DAIT APG01
- Conditions
- Pemphigus Foliaceus, Pemphigus Vulgaris
- Keywords
- autologous polyclonal regulatory T cell therapy, PolyTregs, open-label, Phase 1 (safety)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Lack of recruitment, ongoing and new feasibility issues, and the impact of the coronavirus infectious disease 19 (COVID-19) pandemic
- Enrolment
- 5 participants (actual)
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)US National Institutes of Health
- Collaborators
- Autoimmunity Centers of Excellence, Rho Federal Systems Division, Inc.
- Study start
- 10 Oct 2017 (actual)
- Primary completion
- 10 Dec 2020 (actual)
- Study completion
- 9 Jan 2023 (actual)
- First posted
- 4 Aug 2017 (actual)
- Results
- Posted 22 Nov 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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:
- 1.0 x 10^8 PolyTregs or
- 2.5 x 10^8 PolyTregs.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort 1: 1.0 x 10^8 PolyTregs | Experimental | Biological: Cohort 1: 1.0 x 10^8 PolyTregsA single intravenous infusion of 1.0 x 10^8 PolyTregs will be administered. |
| Cohort 2: 2.5x10^8 PolyTregs | Experimental | Biological: Cohort 2: 2.5x10^8 PolyTregsA single intravenous infusion of 2.5x10^8 PolyTregs will be administered. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- 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;
Key exclusion criteria (53 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Cohort 1: 1.0 x 10^8 PolyTregsAlso: Polyclonal Regulatory T Cells, autologous PolyTregs, CD4+CD127lo/negCD25+ PolyTregsEach participant will receive a target cell dose of 1.0 x 10^8 polyclonal Tregs. | Biological | Cohort 1: 1.0 x 10^8 PolyTregs |
| Cohort 2: 2.5x10^8 PolyTregsAlso: Polyclonal Regulatory T Cells, autologous PolyTregs, CD4+CD127lo/negCD25+ PolyTregsEach participant will receive a target cell dose of 2.5x10^8 polyclonal Tregs. | Biological | Cohort 2: 2.5x10^8 PolyTregs |
Primary outcome
Number of Significant Adverse Events Through Week 52
Time frame: Up to 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.
12 secondary outcomes
Number of Significant Adverse Events
Time frame: From the start of investigational product infusion through Week 156.
Number of All Adverse Events
Time frame: From the start of investigational product infusion through Week 156.
Number of All NCI-CTCAE Grade 3 or Higher Adverse Events
Time frame: From the start of investigational product infusion through Week 52.
Number of All NCI-CTCAE Grade 3 or Higher Adverse Events
Time frame: From the start of investigational product infusion through Week 156.
Number of All SAEs
Time frame: From the start of investigational product infusion through Week 156.
Number of All Infection Related Events
Time frame: From the start of investigational product infusion through Week 156.
Number of All Infusion Reactions
Time frame: Within 24 hours of infusion
Change in Pemphigus Disease Area Index (PDAI) Score From Baseline
Time frame: Weeks 1, 2, 8, 12, 26, 39, 52, 78, 104, 130, and 156
Change in Desmoglein 1 and 3 Titers by ELISA From Baseline
Time frame: Weeks 1, 2, 8, 12, 26, 39, 52, 78, 104, 130, and 156
Number of Participants Experiencing a Relapse/Flare
Time frame: From the start of investigational product infusion through Week 156.
Time to Relapse (Flare)
Time frame: From the start of investigational product infusion through Week 156.
Number of Participants on Prednisone Dose ≤10 mg/Day
Time frame: Weeks 12, 26, 39, 52, 78, 104, 130, and 156
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Feb 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- National Institute of Allergy and Infectious Diseases (NIAID) websiteLink provided by the sponsor.
- Division of Allergy, Immunology, and Transplantation (DAIT) websiteLink provided by the sponsor.
- Autoimmunity Centers of Excellence (ACE) websiteLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. 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.
Related trials for Pemphigus Foliaceus
RecruitingObservational study
Autoimmune Blistering Diseases Study
Pemphigus Vulgaris, Pemphigus Foliaceus
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Haley Naik, MD,MHSc study chair
University of California San Francisco School of Medicine: Department of Dermatology
Anna Haemel, MD study chair
University of California San Francisco School of Medicine: Department of Dermatology
Michael Rosenblum, MD, Ph.D. study chair
University of California San Francisco School of Medicine: Department of Dermatology
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Cohort 1: 1.0 x 10^8 PolyTregs and Cohort 2: 2.5x10^8 PolyTregs in people with Pemphigus Foliaceus and Pemphigus Vulgaris, run by National Institute of Allergy and Infectious Diseases (NIAID) at 4 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 53 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 15 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 22 Nov 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Significant Adverse Events Through Week 52 (time frame: Up to Week 52). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Feb 2024.