Pemphigus

This research aims to evaluate how full-arch implant-supported prostheses affect lip support in patients undergoing complex dental rehabilitation. The study focuses on how different clinical and laboratory decisions between diagnosis and prosthesis delivery may influence the prosthetic design and the patient's facial profile. Advanced 3D facial scanning and superimposition techniques allow precise measurement of vertical occlusion and lip support. Participants will undergo removal of their full-arch implant-supported prostheses, with digital facial images captured before and after this procedure using a facial scanner. This observational study leverages modern digital technology to measure changes objectively and does not involve experimental treatments. During the study, researchers will assess changes in the volume around the mouth area, distances between the lips and esthetic lines on the facial profile, and angles related to the nose and lips. These measurements occur on the same day as the prosthesis removal. The study helps improve understanding of facial changes related to dental prostheses and involves participants collaborating with the research protocol through imaging and clinical assessments.

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 II/III 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 1/2 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.

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 evaluating the safety, tolerability, and effectiveness of stem cell therapy for treating a variety of acute and chronic conditions. This multi-site, multi-arm study focuses on regenerative treatments using amniotic and umbilical cord stem cell rich tissue. The study covers conditions including autoimmune diseases, cardiovascular disorders, diabetes complications, integumentary diseases, musculoskeletal disorders, neurological and neurodegenerative disorders, pulmonary disorders, sexual dysfunction, urologic disorders, and viral illnesses. The goal is to measure improvements based on validated quality of life and condition-specific tools. Participants will receive injections of PrimePro™ or PrimeMSK™ stem cell products administered through condition-specific routes. Each study arm targets a particular group of conditions to compare outcomes against established research. The treatments are given once with follow-up visits planned to assess changes over time. No placebo or control group is used, and the study is non-randomized with open-label participation. During the study, participants will undergo various assessments such as the 36-Item Short Form Survey (SF-36) for quality of life, condition-specific questionnaires like the DASH for arm and shoulder disabilities, IIEF-5 for erectile function, clinical COPD questionnaire, MMSE for mental state, O'Leary/Sant questionnaire for interstitial cystitis, Oswestry Low Back Pain Disability Questionnaire, and WOMAC for osteoarthritis. Follow-up evaluations occur at 6 and 12 months after treatment to monitor progress. The study includes safety monitoring and requires participants to be available for follow-up visits over this time frame.