Polyclonal Regulatory T Cells (PolyTregs) for Pemphigus (NCT03239470) | Clinical Trial Compass
TerminatedPhase 1
Polyclonal Regulatory T Cells (PolyTregs) for Pemphigus
Stopped: Lack of recruitment, ongoing and new feasibility issues, and the impact of the coronavirus infectious disease 19 (COVID-19) pandemic
United States5 participantsStarted 2017-10-10
Plain-language 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. 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.
Who can participate
Age range
18 Years – 75 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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:
* 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.
* 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. S…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of Significant Adverse Events Through Week 52
Timeframe: Up to Week 52
Trial details
NCT IDNCT03239470
SponsorNational Institute of Allergy and Infectious Diseases (NIAID)