A Phase 1 interventional study of Cohort 1: 1.0 x 10^8 PolyTregs and Cohort 2: 2.5x10^8 PolyTregs in Pemphigus Foliaceus and Pemphigus Vulgaris, sponsored by National Institute of Allergy and Infectious Diseases (NIAID). Terminated at 4 sites in United States. Open to participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2024-02-15.
Sponsored by National Institute of Allergy and Infectious Diseases (NIAID) · Phase 1, Interventional, and Treatment
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.
76 studies on the registry are indexed under Pemphigus; 18 are open to participants now.
This study's enrollment of 5 is below the median of 30 across 53 interventional studies indexed under Pemphigus.
Browse Pemphigus studies →National Institute of Allergy and Infectious Diseases (NIAID) is the lead sponsor of 2,446 studies on the registry; 179 are open to participants now.
Of its 405 completed or terminated interventional studies of FDA-regulated products, 302 (75%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Presence of:
Exclusion Criteria:
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:
At or within three months of screening:
Unwilling or unable to use reliable method(s) of :
For females of , from four weeks prior to Day 0 through
1 year after Treg dosing;
Concomitant medical condition that places the subject at risk by participating in this study, including but not limited to:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Vaccines made with a weakened form of the germ, such as measles–mumps–rubella (MMR), chickenpox and yellow fever. They can be risky when the immune system is weakened. Flu injections are not live; the nasal flu spray is.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Live vaccines
Vaccines made with a weakened form of the germ, such as measles–mumps–rubella (MMR), chickenpox and yellow fever. They can be risky when the immune system is weakened. Flu injections are not live; the nasal flu spray is.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A single intravenous infusion of 1.0 x 10\^8 PolyTregs will be administered.
Biological: Cohort 1: 1.0 x 10^8 PolyTregs
A single intravenous infusion of 2.5x10\^8 PolyTregs will be administered.
Biological: Cohort 2: 2.5x10^8 PolyTregs
Each participant will receive a target cell dose of 1.0 x 10\^8 polyclonal Tregs.
Also known as: Polyclonal Regulatory T Cells, autologous PolyTregs, CD4+CD127lo/negCD25+ PolyTregs
Each participant will receive a target cell dose of 2.5x10\^8 polyclonal Tregs.
Also known as: Polyclonal Regulatory T Cells, autologous PolyTregs, CD4+CD127lo/negCD25+ PolyTregs
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.
Time frame: Up to Week 52
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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
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
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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: Weeks 12, 26, 39, 52, 78, 104, 130, and 156
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.
| Milestone | Cohort 1: Polyclonal Treg Infusion (PolyTregs) Low Dose | Cohort 2: Polyclonal Treg Infusion (Poly Tregs) High Dose |
|---|---|---|
| Started | 5 | 0 |
| Completed | 5 | 0 |
| Not completed | 0 | 0 |
| Milestone | Cohort 1: Polyclonal Treg Infusion (PolyTregs) Low Dose | Cohort 2: Polyclonal Treg Infusion (Poly Tregs) High Dose |
|---|---|---|
| Started | 5 | 0 |
| Completed | 4 | 0 |
| Not completed | 1 | 0 |
| Withdrew: Baseline pemphigus disease area index (pdai) >12, thus ineligible for polytregs infusion. | 1 | 0 |
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.
| Number of Events | Cohort 1: Polyclonal Treg Infusion (PolyTregs) |
|---|---|
| Number of Significant Adverse Events Through Week 52 | 0 |
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.
| Number of Events | Polyclonal Treg Infusion |
|---|---|
| Number of Significant Adverse Events | 0 |
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.
| Number of Events | Polyclonal Treg Infusion |
|---|---|
| Number of All Adverse Events | 23 |
Adverse events Grade 3 or higher were graded according to the National Cancer Institute's Common Terminology Criteria for Adverse Events, Version 4.0.
| Number of Events | Polyclonal Treg Infusion |
|---|---|
| Number of All NCI-CTCAE Grade 3 or Higher Adverse Events | 0 |
Adverse events Grade 3 or higher were graded according to the National Cancer Institute's Common Terminology Criteria for Adverse Events, Version 4.0.
| Number of Events | Polyclonal Treg Infusion |
|---|---|
| Number of All NCI-CTCAE Grade 3 or Higher Adverse Events | 0 |
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.
| Number of Events | Polyclonal Treg Infusion |
|---|---|
| Number of All SAEs | 0 |
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.
| Number of Events | Polyclonal Treg Infusion |
|---|---|
| Number of All Infection Related Events | 7 |
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.
| Number of Events | Polyclonal Treg Infusion |
|---|---|
| Number of All Infusion Reactions | 5 |
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.
| score | Polyclonal Treg Infusion |
|---|---|
| PDAI Total Activity Score at Week 1 | -1.58 ± 1.179 |
| PDAI Total Activity Score at Week 2 | -1.15 ± 2.845 |
| PDAI Total Activity Score at Week 8 | -1.50 ± 3.032 |
| PDAI Total Activity Score at Week 12 | -0.33 ± 4.392 |
| PDAI Total Activity Score at Week 26 | -2.87 ± 3.092 |
| PDAI Total Activity Score at Week 39 | -1.23 ± 6.823 |
| PDAI Total Activity Score at Week 52 | -3.28 ± 3.323 |
| PDAI Total Activity Score at Week 78 | -5.08 ± 3.496 |
| PDAI Total Activity Score at Week 104 | -5.75 ± 4.070 |
| PDAI Total Activity Score at Week 130 | -3.98 ± 7.617 |
| PDAI Total Activity Score at Week 156 | -7.23 ± 2.700 |
| PDAI Total Damage Score at Week 1 | 0.000 ± 0.816 |
| PDAI Total Damage Score at Week 2 | 0.25 ± 1.258 |
| PDAI Total Damage Score at Week 8 | -1.00 ± 1.414 |
| PDAI Total Damage Score at Week 12 | -1.00 ± 1.414 |
| PDAI Total Damage Score at Week 26 | -0.67 ± 1.155 |
| PDAI Total Damage Score at Week 39 | -0.75 ± 0.957 |
| PDAI Total Damage Score at Week 52 | -0.50 ± 1.291 |
| PDAI Total Damage Score at Week 78 | -1.25 ± 1.893 |
| PDAI Total Damage Score at Week 104 | -1.50 ± 2.380 |
| PDAI Total Damage Score at Week 130 | -1.25 ± 2.630 |
| PDAI Total Damage Score at Week 156 | -1.50 ± 2.380 |
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.
| U/mL | Polyclonal Treg Infusion |
|---|---|
| Desmoglein 1 at Week 1 | 1.00 ± 1.414 |
| Desmoglein 1 at Week 2 | 1.50 ± 2.082 |
| Desmoglein 1 at Week 8 | -0.50 ± 3.873 |
| Desmoglein 1 at Week 12 | 3.50 ± 4.123 |
| Desmoglein 1 at Week 26 | -7.67 ± 17.786 |
| Desmoglein 1 at Week 39 | -8.67 ± 15.144 |
| Desmoglein 1 at Week 52 | -17.00 ± 33.237 |
| Desmoglein 1 at Week 78 | -20.75 ± 57.968 |
| Desmoglein 1 at Week 104 | -33.88 ± 68.940 |
| Desmoglein 1 at Week 130 | -28.75 ± 75.168 |
| Desmoglein 1 at Week 156 | -28.25 ± 79.943 |
| Desmoglein 3 at Week 1 | -1.75 ± 1.708 |
| Desmoglein 3 at Week 2 | -0.50 ± 4.123 |
| Desmoglein 3 at Week 8 | 3.00 ± 9.309 |
| Desmoglein 3 at Week 12 | 1.25 ± 15.457 |
| Desmoglein 3 at Week 26 | 13.33 ± 27.538 |
| Desmoglein 3 at Week 39 | -33.50 ± 36.919 |
| Desmoglein 3 at Week 52 | -30.75 ± 45.966 |
| Desmoglein 3 at Week 78 | -33.25 ± 49.789 |
| Desmoglein 3 at Week 104 | -38.88 ± 50.150 |
| Desmoglein 3 at Week 130 | -34.38 ± 51.406 |
| Desmoglein 3 at Week 156 | -42.25 ± 49.291 |
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 | Polyclonal Treg Infusion |
|---|---|
| Number of Participants Experiencing a Relapse/Flare | 2 |
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.
| Days | Polyclonal Treg Infusion |
|---|---|
| Time to Relapse (Flare) | 149.0 ± 181.02 |
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 | Polyclonal Treg Infusion |
|---|---|
| Number taking 0 mg/day at Week 12 | 2 |
| Number taking >0 mg/day and <= 10 mg/day at Week 12 | 2 |
| Number taking >10 mg/day at Week 12 | 0 |
| Number taking 0 mg/day at Week 26 | 2 |
| Number taking >0 mg/day and <= 10 mg/day at Week 26 | 2 |
| Number taking >10 mg/day at Week 26 | 0 |
| Number taking 0 mg/day at Week 39 | 2 |
| Number taking >0 mg/day and <= 10 mg/day at Week 39 | 2 |
| Number taking >10 mg/day at Week 39 | 0 |
| Number taking 0 mg/day at Week 52 | 2 |
| Number taking >0 mg/day and <= 10 mg/day at Week 52 | 2 |
| Number taking >10 mg/day at Week 52 | 0 |
| Number taking 0 mg/day at Week 78 | 2 |
| Number taking >0 mg/day and <= 10 mg/day at Week 78 | 2 |
| Number taking >10 mg/day at Week 78 | 0 |
| Number taking 0 mg/day at Week 104 | 2 |
| Number taking >0 mg/day and <= 10 mg/day at Week 104 | 2 |
| Number taking >10 mg/day at Week 104 | 0 |
| Number taking 0 mg/day at Week 130 | 2 |
| Number taking >0 mg/day and <= 10 mg/day at Week 130 | 2 |
| Number taking >10 mg/day at Week 130 | 0 |
| Number taking 0 mg/day at Week 156 | 2 |
| Number taking >0 mg/day and <= 10 mg/day at Week 156 | 2 |
| Number taking >10 mg/day at Week 156 | 0 |
Collected over 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. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Polyclonal Treg Infusion | 0/4 (0%) | 0/4 (0%) | 4/4 (100%) |
| Event | Polyclonal Treg Infusion |
|---|---|
| COVID-19Infections and infestations | 2/4 |
| Viral upper respiratory tract infectionInfections and infestations | 2/4 |
| Infusion related reactionInjury, poisoning and procedural complications | 2/4 |
| LeukopeniaBlood and lymphatic system disorders | 1/4 |
| NeutropeniaBlood and lymphatic system disorders | 1/4 |
| NauseaGastrointestinal disorders | 1/4 |
| Eye infectionInfections and infestations | 1/4 |
| Herpes simplexInfections and infestations | 1/4 |
| Fascial ruptureInjury, poisoning and procedural complications | 1/4 |
| Procedural painInjury, poisoning and procedural complications | 1/4 |
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.
| Age, Continuous(years) | Cohort 1: Polyclonal Treg Infusion (PolyTregs) Low Dose |
|---|---|
| Mean | 38.8 ± 14.2 |
| Sex: Female, Male(Participants) | Cohort 1: Polyclonal Treg Infusion (PolyTregs) Low Dose |
|---|---|
| Female | 2 |
| Male | 2 |
| Ethnicity (NIH/OMB)(Participants) | Cohort 1: Polyclonal Treg Infusion (PolyTregs) Low Dose |
|---|---|
| Hispanic or Latino | 2 |
| Not Hispanic or Latino | 2 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | Cohort 1: Polyclonal Treg Infusion (PolyTregs) Low Dose |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 4 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
| Region of Enrollment(Participants) | Cohort 1: Polyclonal Treg Infusion (PolyTregs) Low Dose |
|---|---|
| United States | 4 |
| Type of Pemphigus(Participants) | Cohort 1: Polyclonal Treg Infusion (PolyTregs) Low Dose |
|---|---|
| Pemphigus Vulgaris | 3 |
| Pemphigus Foliaceus | 1 |
| Age at Diagnosis of Pemphigus(years) | Cohort 1: Polyclonal Treg Infusion (PolyTregs) Low Dose |
|---|---|
| Mean | 33.5 ± 16.9 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: 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.
Time frame: On average, within 24 months after database lock for the trial.
Access criteria: Open access.
Data sharing details ↗ · www.immport.org
Ran from Oct 2017 to primary completion in Dec 2020, 38 months.
Results were first posted in Nov 2021, 11 months after primary completion.
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