Clinical Research Directory

Determine Effectiveness of Anifrolumab In SYstemic Sclerosis (DAISY)

The purpose of this study is to evaluate the efficacy and safety of treatment with subcutaneous anifrolumab versus placebo in adult participants with systemic sclerosis. The target population for this study includes patients who meet the 2013 American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) classification for systemic sclerosis, either limited or diffuse cutaneous subsets, with a disease duration of less than 6 years from first non-Raynaud's phenomenon symptom.

AlloNK®, an Allogeneic Non-genetically Modified, Cord Blood-derived NK Cell Therapy, in Combination With Rituximab, Studied in Relapsing Forms of B-cell Dependent Rheumatologic Diseases.

A Basket Trial of Refractory Rheumatoid Arthritis (RA), Sjögren's Disease (SjD), Idiopathic Inflammatory Myopathies (IIMs) and Systemic Sclerosis (SSc) subjects to evaluate the safety and efficacy of AlloNK, a non-genetically modified allogeneic NK cell, in combination with rituximab.

Skeletal Muscle Function in Interstitial Lung Disease

Dyspnea (i.e. breathlessness) and exercise intolerance are common symptoms for patients with interstitial lung disease (ILD), yet it is not known why. It has been suggested that muscle dysfunction may contribute to dyspnea and exercise intolerance in ILD. Our study aims to: i) examine differences in the structure and function of the leg muscles in ILD patients, ii) determine if leg muscle fatigue contributes to dyspnea and exercise limitation in patients with ILD, and iii) determine the effects of breathing extra oxygen on leg muscle fatigue, as well as ability to exercise in ILD patients.

RESET-SSc: An Open-Label Study to Evaluate the Safety and Efficacy of CABA-201, a CD19-CAR T Cell Therapy, in Subjects With Systemic Sclerosis

RESET-SSc: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201, a CD19-CAR T cell therapy, in Subjects with Systemic Sclerosis

Brentuximab Vedotin for Systemic Sclerosis

There is significant unmet need for effective treatment options for Diffuse Cutaneous Systemic Sclerosis (dcSSc). The present study will be a dose-escalation safety trial of brentuximab vedotin, a drug-antibody conjugate approved for the treatment of lymphoma and targeted to the protein CD30 molecule expressed on activated immune cells There is evidence for CD30 involvement in SSc. This study represents the first step in determining safety and tolerability of brentuximab vedotin in SSc.

MRI for Screening and Monitoring Scleroderma ILD

The purpose of this study is to test MRI methods for evaluating patients with Scleroderma-associated interstitial lung disease.

Elastography Ultrasound in Localized Scleroderma (Morphea) Study

Localized scleroderma (LS) is a skin disease that can cause complications such as disfigurement and limitation of mobility. Treatment for LS should be initiated early in the disease, as late-stage scleroderma does not respond well to treatment. Objective tools for diagnosis and follow-up of treatment for LS are not available clinically and are urgently needed. A new non-invasive ultrasound technique called "elastography" measures stiffness of tissues and holds potential for providing objective measures for follow-up of treatment of LS in children and adolescents. The team plans to conduct a multicenter study in North America to determine whether elastography ultrasound can show changes in skin stiffness for a period of 9 to 12 months after initiation of treatment for LS. This novel technology may add information to the existing imperfect clinical tools, thus improving the way therapy is offered to patients with LS.

Efficacy and Safety of HN2301 in Autoimmune Diseases（AIDs）

This is an open lable and single arm study, is designed to evaluate the safety and preliminary efficacy of HN2301 in Autoimmune Disease（AID）

Brain Boost Program to Improve Cognitive Function in People With Systemic Sclerosis

The purpose of this study is to examine whether an 8-week online educational group-based program tailored to people with systemic sclerosis can help improve cognitive function and well-being. The study team hypothesize that participants that receive the intervention will have better improvements immediately after treatment at week 8 in all cognitive function measures, non-cognitive symptoms, and self-management compared to those in the waitlist control.

Hyaluronidase Via LADD Scleroderma-induced Microstomia

Two treatment methods, ablative carbon dioxide (CO2) laser and intradermal hyaluronidase, will be combined to maximize the beneficial potential of each. Laser-assisted drug delivery (LADD) has been shown to be effective for the administration of medications in diverse skin diseases but not previously reported for the treatment of scleroderma-induced microstomia. By using this laser technique, the investigator can reduce the pain of typical hyaluronidase injections and reap the therapeutic benefit of the laser treatment itself. The investigator's goal is to create greater mouth mobility for chewing and speaking as well as improved oral hygiene, self-esteem, and overall quality of life. Each participant will undergo three separate laser sessions at 4 to 8-week intervals. Participants will also complete a follow-up visit three months after the last laser session to evaluate the response.

Clinico-biological Collection to Investigate the Physiopathology of Systemic Autoimmune Diseases

The aim of this project is to start a biological and clinical collection of patients presenting systemic autoimmune disease. This collection will provide appropriate biological samples to identify new biomarkers and to be accessible to the medical, scientific and industrial communities for the identification of new therapeutic strategies

Clinical Study of EVM18001 in the Treatment of Refractory Autoimmune Diseases

A FIH, single arm, open-label, Investigator Initiated Trial (IIT) study to evaluate the safety and tolerability of EVM18001 in the treatment of active refractory autoimmune diseases (SLE, MG, and SSc), and determine the recommended dose for subsequent treatment. At the same time, the PK/PD characteristics of EVM18001 will be evaluated, preliminary efficacy will be observed, and related biomarkers and immunogenicity will be explored.

Rheumatology Patient Registry and Biorepository

To facilitate clinical, basic science, and translational research projects involving the study of rheumatic diseases.

Tibulizumab Systemic Sclerosis Understanding and Response Evaluation (TibuSURE)

The study is a Phase 2, multi-center, randomized, double-blind, placebo-controlled study to evaluate the effects of tibulizumab over 24 weeks (Period 1) in adult participants with systemic sclerosis, followed by an open-label extension period where all active participants will receive tibulizumab and will be evaluated for an additional 28 weeks (Period 2)

Platform Clinical Study for Conquering Scleroderma

The goal of this clinical trial is to test efficacy of different investigational products (IPs) compared with placebo on the change from baseline to the end of the treatment period at Week 52 in lung capacity in participants with Interstitial Lung Disease Secondary to Systemic Sclerosis.

Transcriptomic Study of Cutaneous Fibroblasts in Scleroderma

Scleroderma is a complex connective tissue disease involving three interconnected pathophysiological axes: vascular hyperactivity and remodelling, immune system dysfunction and over-activation of fibroblasts at the origin of the fibrosis process in the skin and organs. Given that this pathology occurs preferentially in mature subjects, it is possible to suggest a potential inductive role for senescent fibroblasts, which would be responsible for activating and/or maintaining the immune response and systemic inflammation. Our hypothesis is that fibroblasts play a predominant role in the genesis and maintenance of this pathology.

Maternal Autoimmune Disease Research Alliance (MADRA) Registry

This multi-site registry, centered at Duke University, will enroll pregnant women with autoimmune and rheumatologic diseases. The main goal of MADRA is to identify ways to improve the health of women with rheumatic diseases and their babies during pregnancy. Prior studies demonstrate the importance of increase inflammation prior to and during pregnancy on these outcomes. The future research will seek to better define these risk factors and to identify ways to may improve them.

Investigating the Effectiveness of the Biopsychosocial Model-Based Exercise Approach in Children and Adults Diagnosed With Scleroderma

This study aims to investigate the effectiveness of BETY, an exercise approach based on the biopsychosocial model, in children and adults diagnosed with scleroderma.

Evaluating Tools to Communicate Scleroderma Research Results to Patients - Trial #1

Sharing research results with patients is required by ethical regulations. Yet, most researchers do not share results from their studies with patients. The investigators plan to conduct a series of randomized controlled trials among people with scleroderma, a rare autoimmune disease, in a large international cohort, to identify the most effective methods for communicating study results with patients. The first trial in the series will compare a research dissemination tool (infographic) against a plain-language summary comparator. Participants will be randomly assigned to receive the dissemination tool or comparator. Study participants will rate communication tools for (1) information completeness; (2) understandability; and (3) ease of use of format. Our results can be used by researchers and patient organizations who disseminate research results so that they can tailor the way they disseminate results to patient needs.

Scleroderma Registry & Repository at the Hospital for Special Surgery

The overall objective of the Scleroderma Registry is to support and promote the basic science and clinical research of this complex rheumatic disease at the Hospital for Special Surgery (HSS). The registry facilitates our understanding of the clinical features, pathobiology, genetics of Scleroderma. This will ultimately lead to a potential treatment for this currently untreatable condition.

A Biospecimen Collection Study to Identify the Targets of Disease-Reactive T Cells in Patients With Autoimmune Disease

The most clinically meaningful way to discover new targets of T cells in autoimmune diseases is to study the tissues of patients with active autoimmune disease mediated organ inflammation. These tissues contain both cytotoxic and helper T cells that are driving their disease, and these T cells are being guided by TCRs that recognize tissue-specific targets. By collecting tissue when a patient has active inflammation, it is possible to determine which T cells are activated and undergoing clonal expansion in the patient's diseased organ. TScan has developed a genome-wide, high-throughput technology to determine the natural, physiological target of any TCR (Kula, 2019). The goal of this study is to isolate T cells from inflamed tissues and matched blood samples and/or matched normal tissues (for patients with inflammatory bowel diseases). T cell clones that are expanded in diseased tissues relative to blood or normal tissues will be selected and the targets of their TCRs will be defined using TScan's genome-wide, high-throughput target ID technology. The goal of this study is to discover a collection of peptide targets, along with their associated TCRs to be developed as new tolerogenic therapies for patients with autoimmune diseases.

Sildenafil for Early Pulmonary Vascular Disease in Scleroderma

This is a Phase II randomized, double-blind, placebo-controlled trial of sildenafil in men and women with Scleroderma with mildly elevated pulmonary pressures (SSc-MEP) to determine whether sildenafil may be an effective treatment for SSc-MEP.

Patients Diagnosed With Scleroderma and Their Chewing and Swallowing Performance

It is recommended to focus on the physical functionality, chewing, and swallowing performance of individuals diagnosed with scleroderma. There is a need to address children and adults diagnosed with scleroderma comprehensively and to evaluate them on a biopsychosocial basis to support their disease management. The aim was to assess the biopsychosocial characteristics of both children and adults diagnosed with scleroderma and to examine their chewing and swallowing performance. Additionally, this study aims to identify effective scales that can be used in future research to assess chewing and swallowing in individuals diagnosed with scleroderma.

Patients Diagnosed With Scleroderma: Physical Performance and Functionality

Physical functionality and performance are important for individuals diagnosed with scleroderma. There is a need to address children and adults diagnosed with scleroderma comprehensively and to evaluate them on a biopsychosocial basis to support their disease management. In light of all this literature, the aim was to evaluate the biopsychosocial characteristics of both pediatric and adult scleroderma patients and to examine their physical performance and functionality. Additionally, this study aims to identify effective tests that can be used in future research to assess physical performance and functionality in individuals diagnosed with scleroderma.