Pemphigus

Researchers are evaluating the efficacy and safety of a drug called B007 in adults with pemphigus, a condition characterized by blistering of the skin and mucous membranes. This Phase IIIII clinical trial is designed to understand how well B007 works to achieve remission with minimal treatment and to monitor its safety in this patient population. Participants will receive B007 through subcutaneous injections administered on days 1 and 15. The study measures include the proportion of patients achieving complete remission, partial remission, changes in the Pemphigus Disease Area Index PDAI, frequency of disease relapses, duration of response, and incidence of treatment-emergent adverse events. The treatment period and follow-up assessments extend up to approximately one year. During the trial, participants will be closely monitored through scheduled visits to assess disease activity and treatment response. Outcomes such as remission rates and relapse frequency will be tracked, along with safety evaluations for any adverse effects. The total participation duration includes about one year of observation after treatment initiation to fully capture treatment effects and safety data.

Researchers are evaluating autologous Chimeric Antigen Receptor T cells CART cells in people with active pemphigus vulgaris PV, a condition causing painful blisters on skin and mucous membranes. This phase 12 open-label study includes two parts the main study focused on DSG3-CAART therapy for mucosal PV patients not well controlled by standard treatments, and a sub-study assessing CABA-201 therapy in mucosal-dominant and mucocutaneous PV patients. The study aims to find safe dosing and to understand if these therapies can control disease activity. The main study now closed to enrollment tested different doses and infusion schedules of DSG3-CAART, sometimes combined with other treatments. The open sub-study involves a single infusion of CABA-201 with varying preconditioning regimens using cyclophosphamide and fludarabine. Treatments are given intravenously, and the sub-study is currently enrolling participants. Participants will be monitored for adverse events up to 3 months for DSG3-CAART and up to 156 weeks for CABA-201. Researchers will measure safety, immune cell levels, antibody changes, disease activity scores, and remission status over up to 36 months. The study includes regular clinical assessments, laboratory tests, and ongoing safety follow-up to evaluate treatment effects and tolerability.

Researchers are evaluating the severity and impact of autoimmune blistering diseases, specifically pemphigus vulgaris, pemphigus foliaceus, and bullous pemphigoid. These diseases cause severe blistering and require new ways to measure disease severity accurately for use in clinical trials. The study aims to validate two key disease severity instruments, the Pemphigus Disease Area Index PDAI and the Bullous Pemphigoid Disease Area Index BPDAI, against other measures and to understand how these relate to patients quality of life. Participants will complete several questionnaires, including the PDAI, BPDAI, the Autoimmune Bullous Skin Disorder Intensity Score ABSIS, and various quality of life assessments such as the SF-36, Skindex-29, Dermatology Life Quality Index DLQI, Autoimmune Bullous Disease Quality of Life ABQOL, and Treatment of Autoimmune Bullous Disease Quality of Life TABQOL. Blood samples may also be donated to a blood bank for further immunologic and pathophysiologic studies. The study is observational and data is securely stored within a password-protected database. During the study, participants will be asked to rate their disease severity and complete quality of life questionnaires over a one-year period. The research team will use these assessments to compare disease severity scores and quality of life impacts. This study is conducted at the University of Pennsylvania and involves ongoing data collection to support future clinical trials for these conditions.

Researchers are evaluating autologous CD19 CAR-T cell therapy in adults with moderate-to-severe refractory pemphigus vulgaris, a condition that has not responded well to standard treatments. This exploratory, single-center study aims to assess the safety, tolerability, and early effectiveness of this patient-specific cellular therapy. The study also investigates how the therapy affects immune response and disease activity over time. Participants will first undergo leukapheresis to collect their own immune cells, which are then engineered outside the body to target CD19-positive B cells. Before receiving the CAR-T cells, participants will have lymphodepleting chemotherapy with fludarabine and cyclophosphamide to prepare their body for the infusion. They will then receive a single intravenous infusion of the manufactured CAR-T cells. Premedication may be given before infusion based on the investigators judgment. Throughout the study, participants will be monitored for safety concerns such as inflammatory reactions, infections, and organ function. Effectiveness will be measured through changes in disease severity scores and antibody levels. Additional tests will track immune cell changes and CAR-T cell persistence. Follow-up will continue for up to 96 weeks after infusion to assess long-term outcomes and safety.

This research aims to collect biological and clinical data from patients with autoimmune, dysimmune, or auto-inflammatory skin diseases. These conditions include a wide range of rare diseases that collectively affect a significant portion of the adult population, especially women. The study focuses on understanding the breakdown of self-tolerance and the complex mechanisms behind these diseases, as well as identifying patient subgroups based on clinical and biological profiles to improve diagnosis and treatment. Participants will provide biological samples such as blood, cerebrospinal fluid, saliva, stools, urine, tissue biopsies, and hair follicles during their normal diagnostic and follow-up care. The study will gather these samples and clinical data over an average period of one year. The goal is to build a collection that supports the identification of new autoantibodies, biomarkers related to disease severity, and the exploration of disease mechanisms. Samples will also be analyzed to compare blood cell populations before and after certain therapies. During the study, participants will be involved in providing biological specimens as part of their routine medical care. Researchers will monitor these samples and clinical data to assess disease markers, treatment responses, and underlying disease processes. The main outcome is the development of a detailed biological and clinical collection to support future research and therapeutic strategies. The study follows participants for about one year, with ongoing data collection throughout this period.

Researchers are evaluating the coagulation and fibrinolysis profiles in people with autoimmune skin diseases, such as bullous pemphigoid, pemphigus vulgaris, mucous membrane pemphigoid, cutaneous lupus erythematosus, and dermatomyositis. The study compares blood samples from participants with active or poorly controlled immune-mediated skin diseases and those with mild or well-controlled conditions. A control group of general dermatology outpatients without immune-mediated skin diseases or certain skin cancers will also provide samples. This observational study aims to help guide future research on how these blood profiles relate to autoimmune skin conditions. Participants will have a one-time blood draw to measure various coagulation and inflammatory markers. Tests include standard coagulation assays such as activated partial thromboplastin time aPTT and international normalized ratio INR, as well as viscoelastic coagulation profiles like rotational thromboelastometry and thromboelastography, including tPA-challenged versions. Blood samples from both groups will be analyzed and compared to understand differences in coagulation and fibrinolysis related to immune-mediated skin diseases. During the study, participants will provide detailed medical history and have their blood collected once for analysis. Researchers will measure primary outcomes including thromboelastography, tPA-challenged thromboelastography, aPTT, and INR over a two-year period to track coagulation function. This study includes adult participants aged 19 and older and involves healthy volunteers as controls. The total participation involves one visit for blood sample collection and history review.

Pemphigus diseases are serious, chronic autoimmune conditions that cause blistering due to the immune system attacking certain skin proteins. The two main types studied here are pemphigus vulgaris and pemphigus foliaceus. Researchers are evaluating a new personalized maintenance therapy involving rituximab infusions guided by antibody levels to see if this approach can better prevent disease relapses compared to the standard treatment. This is a phase 4 randomized study conducted by the University Hospital, Rouen, building on previous rituximab trials that led to regulatory approvals. Participants in the trial will receive initial treatment with rituximab and oral prednisone following French guidelines, with rituximab given at Day 1, Day 14, Month 12, and Month 18. One group will receive this standard regimen, while the experimental group will have their treatment adapted based on anti-desmoglein antibody levels. Additional rituximab infusions may be given at Month 6 or later if antibody levels remain high or increase, with up to two extra infusions per year allowed during the study. During the study, participants will be regularly monitored through blood tests for antibody levels and blood cells every three months for up to 7.5 years. Researchers will assess the number of disease relapses, time to relapse, duration of remission, quality of life, and rituximab exposure. The study will also track the total dose of rituximab and record safety outcomes. This long follow-up aims to understand if personalized maintenance with rituximab can reduce relapses and the need for corticosteroid treatment.

Researchers are evaluating a zinc oxide adhesive securement device to help reduce complications related to the fixation of midline and peripherally inserted central catheters PICC in hospitalized adult patients. The study aims to compare this zinc oxide device with a standard acrylic adhesive device to see if it lowers issues such as catheter displacement, skin injuries, phlebitis, and pain. The trial is a randomized clinical study designed to improve care decisions for vascular access devices by reducing common complications. Participants will be divided into two groups after catheter insertion using a modified micro-Seldinger technique. One group will receive the standard care using an acrylic adhesive securement device along with a clear polyurethane dressing and cyanoacrylate glue. The intervention group will receive the same care except their catheter will be fixed with a zinc oxide adhesive securement device instead of the acrylic adhesive. Ultrasound guidance is used for all catheter insertions to improve accuracy. During the study, researchers will observe participants for seven days following catheter placement to monitor for treatment-related adverse events, skin changes under the fixation device, catheter displacement, phlebitis using a visual rating scale, and pain in the affected limb measured by a pain scale. Participants will be monitored closely for any complications related to the catheter fixation. The total participation involves care and assessments over this week-long period to evaluate the safety and efficacy of the zinc oxide adhesive device compared to the standard adhesive.

This observational study aims to prospectively evaluate the individual burden of nine rare skin diseases by assessing various aspects of disability, including psychological, social, economic, and physical impacts on patients and their families. The goal is to gain a comprehensive understanding of how these conditions affect daily life and care needs. The study uses two main indicators first, an individual burden score derived from a specially designed questionnaire that patients or their families complete as a self-assessment to track changes in care and lifestyle second, a descriptive analysis of all medical and non-medical resources used by the family to manage the disease. These tools help capture the broad impact of the diseases over time. Participants will be followed for an average of five years, during which researchers will collect data through surveys completed by patients and parents, monitor quality-of-life changes, validate clinical severity scores where needed, and analyze socioeconomic and healthcare costs. The study focuses on detailed evaluations of burden scores and their association with disease severity, aiming to provide a deep insight into the challenges faced by individuals affected by these rare skin diseases.

Researchers are studying chronic inflammatory skin diseases, which include conditions affecting the skin and other organs such as joints and muscles. These diseases vary widely in how they progress and respond to treatments. The goal is to discover new cellular or molecular markers that can predict how well patients respond to systemic treatments after one year. This research also aims to improve understanding of these diseases, identify markers linked to clinical status over ten years, and find predictors of drug side effects. Participants include patients with moderate to severe chronic inflammatory skin diseases like atopic dermatitis, psoriasis, and others, as well as healthy controls who have undergone plastic surgery. The study involves collecting various biological samples such as blood, skin biopsies, skin swabs, hair follicles, and stool. These samples will be collected at different times, including at the start, four months, and one year, to analyze molecular, cellular, and microbiological markers. During the study, participants will undergo evaluations involving these biological sample collections to monitor immune signaling pathways, blood T cell populations, and skin gene activity. Researchers will also assess treatment responses, quality of life, and drug side effects at multiple points from inclusion up to ten years. This long-term follow-up allows detailed tracking of disease progression, treatment effectiveness, and safety over time.