Clinical Research Directory

Deciphering the Genetic Architecture of Autoimmune Diseases

Background: Autoimmune diseases can be caused by genes people inherit from their parents. The gene changes that cause these diseases have been well studied in people with European and Asian ancestors. But some diseases behave differently in people who are native to North and South America. Researchers want to know more about the gene changes and other factors that may cause autoimmune diseases among these people. This project will be based in Peru. Objective: To study how gene changes can lead to autoimmune diseases in people native to Peru. Eligibility: People aged 18 years and older with an autoimmune disease. These may include systemic lupus erythematosus; Sjogren disease; scleroderma; rheumatoid arthritis; seronegative spondylo-arthropathies; and systemic vasculitis. Family members and healthy volunteers are also needed. Design: Participants will have 2 clinic visits; these will be 2 weeks apart. The clinics will be in Lima, Iquitos, and other sites in Peru. Visit 1: Participants will have a physical exam. They will answer questions about their health risks and habits. They will provide blood and urine samples. Visit 2: Participants will provide a second blood sample and a stool sample. They will talk about the results of their first clinical exam with researchers. The cost of travel to and from the clinics will be provided. Participants will get $30 per visit and a snack.

Treatment Strategies for IgG4-RD Patients With Superficial Organ Involvement

This study has been designed as a 12-month, open-label randomized controlled clinical trial. The study aims to compare the efficacy and safety of two treatment strategies in IgG4-RD patients with superficial organ involvement: prednisone plus iguratimod and prednisone plus leflunomide.

Proof of Concept Study Evaluating the Efficacy and Safety of ATH-063 Treatment in Patients With Relapsed/Refractory Moderately to Severely Active Ulcerative Colitis (UC)

The primary objective of this trial is to evaluate the clinical efficacy of ATH-063 in participants with biologic/advanced therapy relapsed/refractory moderately to severely active UC.

Allogeneic CD19/BCMA CAR-T for B Cell-Related Autoimmune Disease

This is an exploratory, open-label, single-arm Phase 1 clinical study designed to evaluate the safety, tolerability, and preliminary efficacy of QT-219C. QT-219C is a universal allogeneic chimeric antigen receptor T-cell (CAR-T) product targeting both CD19 and BCMA. The study targets subjects with refractory B-cell-related autoimmune diseases, including systemic lupus erythematosus (SLE), multi-drug resistant nephrotic syndrome (NS), IgA nephropathy (IgAN), systemic sclerosis (SSc), and ANCA-associated vasculitis (AAV) .The research is divided into two phases: a dose-escalation phase and a dose-expansion phase. Dose Escalation: Utilizes a standard "3+3" design to evaluate potential recommended dose(RD) and identify dose-limiting toxicities (DLTs) .Treatment Procedure: Eligible subjects will receive a lymphodepleting conditioning regimen followed by a single intravenous infusion of QT-219C .Primary Objectives: The primary goals are to evaluate the safety profile, including the incidence of cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS), and to assess clinical response rates at 90 days post-infusion .Follow-up: Subjects will be monitored for pharmacokinetics (cell expansion), pharmacodynamics (B-cell depletion), and long-term safety for up to two years .

A Study of mRNA Encoding CD19/CD3 T Cell Engager (ABO2203) in Patients With Refractory Autoimmune Diseases

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, as well as preliminary efficacy of mRNA encoding CD19/CD3 T cell engager (ABO2203) in patients with refractory autoimmune diseases who received inadequate response or relapsed from standard of care (SoC). The trial included dose escalation and dose expansion parts.

Cluster of Differentiation 19 (CD19)/B Cell Maturation Antigen (BCMA) Chimeric Antigen Receptor T-Cell (CAR-T) Therapy for Refractory Autoimmune Diseases

This study is an investigator-initiated single center, single arm clinical study with a target population of patients with refractory autoimmune diseases. It is an early exploratory clinical study of the safety, tolerability and initial efficacy of CD19/BCMA CAR-T in the treatment of refractory autoimmune diseases.

Clonal Hematopoiesis of Immunological Significance

Ambispective, national, multicenter observational cohort study aimed at characterizing the satellite dysimmune manifestations of clonal hematopoiesis, including Vexas (Vacuoles, E1 enzyme, X-linked, Autoinflammatory and Somatic) syndrome.

A Phase 1 Study of Prulacabtagene Leucel (Prula-cel, Formerly ADI-001) in Autoimmune Disease

ADI-202300103 is a phase 1 multicenter, open label, dose finding and dose expansion, safety/efficacy study in patients with autoimmune disease. The study will consist of different periods including screening, lymphodepletion, treatment, and follow-up

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single- and Multiple-Ascending Doses and Food Effect of BGB-45035 in Healthy Participants and in Adults With Autoimmune Dermatological Diseases

This study is the first-in-human (FIH) study of BGB-45035. The study will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of BGB-45035 with both a single dose and multiple doses administered at different dose levels in healthy participants, followed by a Part E to evaluate the safety and tolerability of BGB-45035 in adults with autoimmune dermatological diseases like atopic dermatitis (AD) and prurigo nodularis (PN). An additional biomarker cohort will be evaluated in Part F. Study details include: * The study duration will be up to 24 months. * The treatment duration will be up to 14 days for Parts A-D, up to 12 weeks for Part E, and up to 3 weeks for Part F. * Safety follow-up 30 days after last dose of study drug.

Anti-CD19 Chimeric Antigen Receptor T Cells for Refractory Autoimmune Diseases

The goal of this study is to evaluate the safety and effi cacy of CD19 CAR T cells in the treatment of Refractory Autoimmune Diseases.

Exploratory Clinical Study on the Safety of STR-P004

This is an open-label, multiple-dose escalation IIT clinical study aimed at evaluating the safety, tolerability, PK, and preliminary efficacy of STR-P004 in adult patients with relapsed or refractory autoimmune diseases.

Clinical Study on Targeted CD19 or CD19-BCMA CAR-T Therapy for Autoimmune Diseases

This is an open clinical pharmacological translational Research Study, aiming to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of CD19 or CD19-BCMA CAR-T in patients with active SLE, SSc, AAV, IIM and pSS.

Study of S-4321 in Participants With an Autoimmune or Immune-mediated Disease

This is a multi-center, open-label Ph 1b basket study to assess safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), immunogenicity, biomarker response, and preliminary efficacy of S-4321 in adults with autoimmune or immune-mediated disease including rheumatoid arthritis (RA), psoriatic arthritis (PsA), psoriasis (PsO), cutaneous lupus erythematosus (CLE) with or without systemic manifestations, or atopic dermatitis (AD).

Clinical Study of AFN50 Injection in the Autoimmune Diseases

This is an investigator-initiated trial designed to evaluate the safety, tolerability and primary efficacy of AFN50 injection for the treatment of autoimmune diseases.

Study to Evaluate the Safety and Feasibility of Diamyd® in Individuals at Risk for Type 1 Diabetes

A 2-arm randomized Phase II Open Label Study to evaluate the safety and feasibility of intralymphatic administration of Diamyd® (Diamyd) also known as retogatein in individuals at risk of Type 1 diabetes carrying the HLA DR3-DQ2 haplotype.

Ovarian Tissue Cryopreservation for Fertility Preservation

The goal of this observational study is to learn about fertility preservation for pre-pubertal, peri-pubertal, and adult participants that are unable to pursue clinical standard of care fertility preservation such as egg (oocyte) and embryo cryopreservation. In addition, this study will provide research tissue for the following Specific Aims: 1. To optimize techniques for cryopreservation of ovarian tissues from patients at significant risk for infertility. 2. To investigate factors affecting successful maturation ovarian tissue. Participants will undergo a surgical procedure to remove an ovary (oophorectomy) to preserve their gonadal tissue for fertility preservation.

A Study to Investigate Safety and Effectiveness of Porcine Pancreatic Cells (OPF-310) in Patients With Type 1 Diabetes Mellitus

This study is First In Human study for Encapsulated Porcine Islet Cells for Xenotransplantation (OPF-310). The purpose of this study to assess the safety, tolerability, and efficacy of OPF-310 transplantation and to define the recommended Phase 2 dose (RP2D) in adult subjects with unstable Type 1 Diabetes Mellitus (T1DM) and a level 3 (severe) hypoglycemic episode at least three times within the 1 year prior to enrollment despite treatment with a closed loop system (CLS) for at least 6 months.

A Phase 1 Study of Anitocabtagene Autoleucel for the Treatment of Subjects With Non-oncology Plasma Cell-related Diseases

A Phase 1 dose-escalation study designed to evaluate the safety, tolerability, and preliminary efficacy of anito-cel in subjects with generalized myasthenia gravis (GMG). Anitocabtagene autoleucel (anito-cel) is a BCMA-directed CAR-T cell therapy.

Exploratory Clinical Study on the Safety and Efficacy of Anti- CD19/BCMA U CAR-T Cell Injection for the Treatment of Relapsed/Refractory Autoimmune Diseases

A single arm, open-label pilot study is designed to determine the safety and effectiveness of anti-CD19/BCMA U CAR T cells in patients with autoimmune diseases. 60 patients are planned to be enrolled in the dose-escalation trial.

Biologics And Clinical Immunology Cohort at Sinai

The study team plans to establish a bioregistry of patients receiving biologic therapy as part of their standard treatment at the Mount Sinai Therapeutic Infusion Center and affiliated practices. The study team will to apply state-of-the-art approaches to assessing and predicting immunological and clinical responses associated with these standards and prescribed treatments. The approach is twofold. The first component is to establish a robust and flexible biorepository and database that includes demographic, immunologic, exposure and clinical records, and can facilitate research across disciplines, and across other registries affiliated with Mount Sinai. The second component is to address specific key research questions focused on using novel diagnostics to increase the effectiveness of biologic treatment. Most patients will be recruited from the Mount Sinai Therapeutic Infusion Center (TIC), although others receiving infusions elsewhere or at home will be recruited from outpatient Sinai affiliated clinical practices.

Natural History of Type 1 Interferonopathies: Insights From a European Cohort

Type I interferonopathies are rare autoinflammatory disorders caused by genetic defects and associated with significant morbidity and mortality. These diseases are refractory to conventional immunosuppressive therapies. They typically occur in childhood, although disease onset in adulthood has been observed. The clinical spectrum is wide and mainly involves the central nervous system. Joint involvement is also common, and more rarely, haematological features such as cytopenias or immunodeficiency may be observed. Nearly all patients show consistent over-activation of the type I IFN pathway, as evidenced, the expression of IFN-stimulated genes, the so-called 'interferon signature'. To date, the natural history of interferonopathies remains unclear. In this context, the establishment of a natural history of type I interferonopathy in patients is proposed to elucidate the pathophysiological mechanisms and identify biomarkers for diagnosis, prognosis, and disease activity, with the aim of better characterising the diversity of interferonopathies. The main objective is to characterise the evolution of the pathology in paediatric and adult patients with type I interferonopathies. The overall aim of this research is to propose therapeutic options tailored to patient phenotypes and to better define patient sub-groups in order to optimise the preparation of future clinical trials.

A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)

This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients with hematologic malignancies and other indications.

Exploratory Study on LNP-mRNA Drugs Targeting CD19/BCMA for the Treatment of Relapsed and Refractory Autoimmune Diseases

Autoimmune diseases are a type of chronic disabling disease, characterized by the immune system incorrectly recognizing and attacking the body's own tissues, leading to tissue damage and organ dysfunction, seriously threatening life and health. Although there are various treatment methods currently available, there are still many limitations to immune related diseases that aim for long-term remission, and further research and breakthroughs are urgently needed. Targeting and clearing B cells is one of the core strategies in the treatment of autoimmune diseases. Its mechanism mainly involves clearing abnormally activated B cells, reducing the production of autoantibodies, and regulating immune disorders. The therapy targeting CD19 has become an important research and development direction due to its ability to clear a wider range of B cell lineages (including plasma cells), which may achieve deeper levels of B cell depletion than CD20 targeted therapy. In addition, BCMA is highly expressed specifically on plasma cells, especially long-lived plasma cells, and is a key target for clearing the source of antibody production. Therefore, the CD19/BCMA dual target therapy aims to achieve more complete coverage of the antibody production pathway by simultaneously targeting B cells (and precursors) and plasma cells, which is expected to further improve the treatment response rate and achieve deeper and more persistent immune reset. The advent of COVID-19 vaccine has brought LNP mRNA technology into the public's view. After years of development, it not only shines brilliantly in COVID-19 vaccine, but also is widely used in the treatment and exploration of cancer, rare diseases and other fields. The core of LNP mRNA technology targeting CD19/BCMA is to encapsulate the mRNA encoding specific proteins (such as anti-CD19/BCMA related proteins) in lipid nanoparticles and deliver them to the body through intravenous or intramuscular injection. This experimental drug is a messenger ribonucleic acid (mRNA) therapeutic drug based on the dual targets of CD19 and BCMA, formed by loading mRNA encoding CD19/BCMA receptor related proteins onto lipid nanoparticles (LNP) for injection.

Integrating Vaccination Into Hospital Care Pathways for Vulnerable Patients

AMBU-VAX is a prospective, single-center observational study designed to develop and implement an organizational model for delivering recommended vaccinations within a hospital setting. The study targets adult and elderly patients with chronic diseases or immunocompromising conditions who are eligible for vaccination according to national immunization guidelines. Vaccination is actively proposed during outpatient visits, hospital admissions, or at discharge and, when accepted, administered within the hospital or coordinated with local public health vaccination services. The study aims to evaluate the feasibility, uptake, and completion of hospital-based vaccination pathways and to support integration between hospital and territorial prevention services for vulnerable populations.